Category Archives: Progress

Russian and U.S. state flags fly near a factory in Vsevolozhsk, Leningrad Region, Russia March 27, 2019. REUTERS/Anton Vaganov/File Photo

MOSCOW, Nov 6 (Reuters) - Russia on Saturday said it does not expect progress in talks in the coming weeks with the United States on issues including visas and the size and functioning of their embassies, Russian news agencies reported.

With ties already at post-Cold War lows, Moscow and Washington are in a dispute over the number of diplomats they can post to each other's countries, though Russia has said it was willing to lift restrictions imposed in recent years.

Deputy Foreign Minister Sergei Ryabkov on Saturday said recent contacts with Washington had simply involved the two sides repeating their previously stated positions, but that a new round of talks in a third country would take place in the coming weeks.

"There is no progress and, it seems, none is expected," Interfax cited Ryabkov as saying.

Russia and the United States failed to make major headway on the embassies dispute during a visit to Moscow last month by U.S. Under Secretary of State Victoria Nuland. read more

The U.S. State Department is getting to the point of being able to maintain only a "caretaker presence" in Russia, a senior department official said in late October, with just 120 staff at the embassy in Moscow. read more

Russia has just over 400 diplomats in the United States, including its delegation to the United Nations in New York, the U.S. official said.

"The Americans need to simply increase the number of their staff in Russia to restore normal consular and visa services," Ryabkov said on Saturday.

TASS cited Ryabkov as saying that the next round of U.S.-Russia talks on strategic stability could take place in January.

Reporting by Alexander MarrowEditing by Ros Russell

Our Standards: The Thomson Reuters Trust Principles.

Originally posted here:

Russia not expecting progress at talks with U.S. on visas, diplomats -agencies - Reuters

SYDNEY, Nov. 9, 2021 /PRNewswire/ -- Clinical stage drug development company Pharmaxis Ltd (ASX: PXS) today announced that an Investigational New Drug application (IND) for a trial of PXS-5505 in hepatocellular carcinoma (HCC) patients has been cleared by the United States Food and Drug Administration (FDA). The IND was submitted by the University of Rochester Medical Center, New York State, following the positive pre-clinical results reported in August 2021 at the Americas Hepato-Pancreato-Biliary Association conference in Miami, USA. The trial design approved by the FDA calls for PXS-5505 to be added to current chemotherapy standard of care; combination of a PD-L1 inhibitor and an anti-VEGF drug as first line therapy in newly diagnosed patients with unresectable HCC carcinoma.

Primary liver malignancies have doubled in incidence over the last two decades. These malignancies are now the 4th leading cause of cancer-related mortality worldwide with a 19.6% 5-year relative survival rates. Currently, just 20-30% HCC are resectable at presentation with many patients relying on chemotherapy. A prominent feature of HCC is the presence of highly fibrotic tissue that increases tumour stiffness, and decreases access of drugs into the tumour. Under the guidance of Dr. Roberto Hernandez-Alejandro, MD (Chief Division of Transplantation / Hepatobiliary Surgery), the research team at the University of Rochester Medical Center, New York State, have been investigating the role of lysyl oxidase enzymes in liver cancer and whether Pharmaxis' cancer drug PXS-5505 can improve the efficacy of current chemotherapy drugs by inhibiting these enzymes.

Dr. Roberto Hernandez-Alejandro said, "At the University of Rochester Wilmot Cancer Center, we are excited about the prospect of combining PXS-5505 with standard first line therapy for our unresectable hepatocellular carcinoma patients. The incidence of hepatocellular carcinoma is rising in part due to increasing incidence of cirrhosis and non-alcoholic steatohepatitis. Beyond resection, effective systemic therapies for this disease are lacking, thus new treatment regimens are of significant clinical need."

Dr. Nabeel Badri, (Wilmot Cancer Institute, University of Rochester) added, "PXS-5505 is a potent inhibitor of lysyl oxidase, a key enzyme in collagen crosslinking. By inhibiting the formation of fibrotic tissue in the tumor we hope to improve delivery and effectiveness of immunotherapy drugs which have so far had a limited impact of the survival of our patients. Through preclinical testing and translational research, we have developed a promising clinical trial design that has the potential to benefit these patients and improve our understanding of hepatocellular carcinoma."

The IND submitted by Rochester referenced the previous successful IND lodged by Pharmaxis for the ongoing phase 2 trial of PXS-5505 in myelofibrosis. The approved trial design envisages a dose escalation stage where the safety of PXS-5505 in combination with a PD-L1 inhibitor and an anti-VEGF drug will be assessed at several different doses as well as measures designed to explore the impact of PXS-5505 on fibrosis and drug perfusion. This will be followed by a 6-month trial of the selected dose with both safety and efficacy endpoints.

Pharmaxis CEO Gary Phillips said, "We highly value our collaboration with the research team at University of Rochester. The rapid progression from the compelling pre-clinical work presented for the first time in August to a successful IND submission is very encouraging and we look forward to concluding arrangements for the commencement of the dose escalation study in 2022. PXS-5505 has recently progressed to a phase 2 clinical trial looking for evidence of disease modifying effects in the bone cancer myelofibrosis as a monotherapy so exploring its additional potential to address cancers where fibrosis is limiting the clinical benefit of current chemotherapy- such as liver and pancreatic cancer- would be significantly value adding."

AUTHORISED FOR RELEASE TO ASX BY:

Pharmaxis Ltd Disclosure Committee. Contact: David McGarvey, Chief Financial Officer and Company Secretary: T +61 2 9454 7203, E [emailprotected]

CONTACT:

Media: Felicity Moffatt: T +61 418 677 701, E [emailprotected]Investor relations:Rudi Michelson (Monsoon Communications) T +61 411 402 737, E [emailprotected]

Join the Pharmaxis mailing listhere

Follow us: LinkedIn/Twitter

About Pharmaxis

Pharmaxis Ltd is an Australian clinical stage drug development company developing drugs for inflammatory and fibrotic diseases, with a focus on myelofibrosis. The company has a highly productive drug discovery engine built on its expertise in the chemistry of amine oxidase inhibitors, with drug candidates in clinical trials. Pharmaxis has also developed two respiratory products which are approved and supplied in global markets, generating ongoing revenue.

Pharmaxis is developing its drug PXS-5505 for the bone marrow cancer myelofibrosis which causes a build up of scar tissue that leads to loss of production of red and white blood cells and platelets. The US Food and Drug Administration has granted Orphan Drug Designation to PXS-5055 for the treatment of myelofibrosis and permission under an Investigational Drug Application (IND) to progress a phase 1c/2 clinical trial that began recruitment in Q1 2021. PXS5505 is also being investigated as a potential treatment for other cancers such as liver and pancreatic cancer.

Other drug candidates being developed from Pharmaxis' amine oxidase chemistry platform are targeting fibrotic diseases such as kidney fibrosis, NASH, pulmonary fibrosis and cardiac fibrosis; fibrotic scarring from burns and other trauma; and inflammatory diseases such as Duchenne Muscular Dystrophy.

Pharmaxis has developed two products from its proprietary spray drying technology that are manufactured and exported from its Sydney facility; Bronchitol for cystic fibrosis, which is approved and marketed in the United States, Europe, Russia and Australia; and Aridol for the assessment of asthma, which is approved and marketed in the United States, Europe, Australia and Asia.

Pharmaxis is listed on the Australian Securities Exchange (PXS). Its head office, manufacturing and research facilities are in Sydney, Australia. http://www.pharmaxis.com.au

About PXS-5505

PXS-5505 is an orally taken drug that inhibits the lysyl oxidase family of enzymes, two members LOX and LOXL2 are strongly upregulated in human myelofibrosis. In pre-clinical models of myelofibrosis PXS-5505 reversed the bone marrow fibrosis that drives morbidity and mortality in myelofibrosis and reduced many of the abnormalities associated with this disease. It has already received IND approval and Orphan Drug Designation from the FDA.

Forward-looking statements

Forwardlooking statements in this media release include statements regarding our expectations, beliefs, hopes, goals, intentions, initiatives or strategies, including statements regarding the potential of products and drug candidates. All forward-looking statements included in this media release are based upon information available to us as of the date hereof. Actual results, performance or achievements could be significantly different from those expressed in, or implied by, these forward-looking statements. These forward-looking statements are not guarantees or predictions of future results, levels of performance, and involve known and unknown risks, uncertainties and other factors, many of which are beyond our control, and which may cause actual results to differ materially from those expressed in the statements contained in this document. For example, despite our efforts there is no certainty that we will be successful in developing or partnering any of the products in our pipeline on commercially acceptable terms, in a timely fashion or at all. Except as required by law we undertake no obligation to update these forward-looking statements as a result of new information, future events or otherwise.

SOURCE Pharmaxis Limited

Originally posted here:

FDA Clears Pharmaxis Cancer Drug to Progress to Phase 2 Study in Liver Cancer - PRNewswire

We are 10 games into the 2021-22 NHL season so it seems like a good time to take a look back at what we have learned about these Pittsburgh Penguins so far.

The early record of 4-3-3 is not great, and there have been a couple of points given away with third period breakdowns (specifically against Florida and Minnesota where multi goal leads disappeared) but I think there is a lot of reason to like the way the Penguins have played for the most part.

Their underlying numbers are strong, and among the best in the league.

They are on the positive side of the shot attempt differential (51.5 percent shot attempt share during 5-on-5 play) and in the top-five when it comes to expected goals (fifth place at 54.9 percent) and high-danger scoring chance (fourth place at 56.4 percent) differentials.

They are also outscoring teams by a 24-19 margin in 5-on-5 situations (very good!) and have one of the leagues best penalty killing units (also very good!).

Basically, I have not seen anything from this team so far that changes my preseason prediction that they are still going to be playoff team. They are right there. They just need to get their players back. Speaking of that.

Because of injury and COVID-19 protocols the Penguins top players have been sidelined for most of the season so far. Sidney Crosby, Evgeni Malkin, Kris Letang, Jeff Carter, Bryan Rust, Zach Aston-Reese, Brian Dumoulin, and Marcus Pettersson have combined to miss 40 man games so far this season. Crosby and Malkin have played in a total of one game (Crosby played one game), while Letang has missed four games and Rust has missed seven games.

Add in head coach Mike Sullivan being out for the past two games and who knows how many more in the future and that is a lot to overcome.

In all honesty it kind of makes the overall record and the underlying numbers even more impressive. They have at least stayed in it and given themselves a chance to remain competitive until they get their best players back in the lineup.

Overall I think Jarry has played fine, and the numbers back that up. So far he has rebounded nicely from that ugly Stanley Cup Playoff showing and has rewarded Penguins management for its confidence in him. But it is also still a small sampling, and at times it does seem like his whole performance is a house of cards. I did not like his game against Minnesota on Saturday, and his shootout performances have been dreadful so far, stopping just two out of seven shots in two losses to Dallas and Minnesota. He is still a huge wild card for the Penguins and is going to play a significant role in what this team does this season.

Specifically Danton Heinen and Brock McGinn, Heinen has been especially impressive as he currently is the teams leading scorer after signing for just over one million dollars against the salary cap in the offseason. With Brandon Tanev and Jared McCann leaving this offseason they were going to need to rebuild some depth, and the depth players have more than held their own this season so far. Heinen has been strong, McGinn has six points in 10 games, and even players like Drew OConnor and Evan Rodrigues are making contributions. It is a good, positive development to have secondary players contributing because when the stars get back in the lineup you know you can still have a competent group of complementary players around them.

We can chalk some of this up to the absence of players like Crosby, Malkin, Rust, and Letang for a significant portion of the season. But you would still like to see them convert on more than 12 percent of their chances so far. They have missed some big opportunities to put games away (or get back into games) and have not been a game-changing unit at any point this season. That continues a weakness from the 2020-21 season. The fact the Penguins have been so good with 5-on-5 scoring is encouraging, but you still want to get something from the power play unit.

If you like chaos, Mike Matheson is your player. He is among the Penguins leaders in expected goals for when he is on the ice, and one of the worst players in expected goals against. That is the Mike Matheson experience. He scored one of the best goals of the season against Toronto, and was guilty of one of the most glaring turnovers resulting in a late game-tying goal against the Philadelphia Flyers.

At least he has been to me. Prior to leaving the lineup recently I thought he has been one of the Penguins best all-around players, and certainly one of their best defensemen in all phases of the game. He and John Marino are important players for the Penguins defense because of their long-term salary and salary cap commitments, and also because of their age and the fact they should be just now entering the prime years of their careers. If they play to their abilities that is the makings of a great second defensive pair to go with Kris Letang and Brian Dumoulin.

They can overcome the early record and still make the Stanley Cup Playoffs (I again believe they will) but they do not want to waste any time. There are no weaknesses or off nights in the division with this season, with even the Columbus Blue Jackets and New Jersey Devils looking strong.

See the original post:

Progress report: What we know about the Penguins through 10 games - PensBurgh

In implementing an entirely new defensive system upon his arrival to the Boston Celtics, first-year head coach Ime Udoka knew that his team would likely need some time to adjust.

Such was confirmed through the first five games of the regular season, as the Cs struggled out of the gates with defensive rating of 110.6, which placed them 23rd in the NBA in that department.

However, over the last four games, Boston has begun to show steady improvement, posting a defensive rating of 98.5, which ranks fourth in the league during that span.

Some things that we were asking them to do early were probably not natural for all of them, so we knew it was going to take some time, Udoka said after Saturday mornings shootaround in Dallas. But you just gotta keep working.

And so, they did.

After losing to the Washington Wizards 116-107 in regulation on Oct. 27, the Cs bounced back defensively two nights later by holding Washington to 115 in a double-overtime loss on the road. Then on Monday night, they outscored a one-loss Chicago team 94-75 through the first 33 minutes of action at TD Garden, before easing up and allowing the Bulls to fight back for a 128-114 win.

Despite those two losses, the team felt that it was at least making progress on the defensive end. Al Horford told the team after the Chicago game that the wins would come if they kept bringing pressure on a consistent basis.

After the game we lost, [Al] sat us down and he told us, were gonna eat, Rob Williams recalled. He just told us to embrace it, embrace the loss, embrace the mess-ups and just keep pushing.

Sure enough, Bostons persistent effort turned into wins during this weeks two-game trip to Florida. First, the Cs made the Orlando Magic disappear with a 92-79 victory. And then they torched the first-place Miami Heat with a 95-78 win.

It marked the first time since April of 2012 that Boston held two consecutive opponents to fewer than 80 points with the latter instance coming against the NBAs top-ranked offense, nonetheless.

The thing that's really been impressive is the team defense, Udoka said looking back on the past week-plus of improvement. The rotations behind it have really stepped up, us showing the crowd, shifting, keeping guys from the rim, and then everybody covering each other's back. So when you watch the film you've seen over the last five games, we've been number eight in defense. Even in the games we lost, we played really well and just had some breakdowns offensively. So we feel good about it even in the losses, and then it just translated in those last two games. But the effort, understanding, and accountability with each other has been great in those games.

Team defense is the foundation of Udokas system. The switching style that he has implemented requires Bostons defenders to often change/switch onto different defensive assignments mid-play in order to disrupt the flow of an opposing offense.

Transitioning into such a system also requires collective patience to get on the same page. Though, the team is embracing the adjustment process.

There really wasnt a lot of switching my rookie year, said Williams. Its something I talked to all of my coaches and teammates about its something I work on and I like the challenge. I like taking on the challenge of switching onto guards.

Theres also the challenge of knowing exactly when to switch and when to stay on ones initial assignment.

We don't need to switch on certain things, and they're getting a better understanding of that, Udoka said. And then, when a guy gets put in a bad spot, we have each other's back. And thats what we've been doing great. They'll try to take advantage of mismatches and we have the counters behind that. So I think we've kind of been on point with recognition of that, looking like second nature out there.

The same could not have been said during the first week-plus of the season, but the Celtics have come a long way since then. They look far more comfortable on the defensive end, theyre working better as a team, their understanding of Udokas system has improved, and the results are beginning to show. It's an upward trend which the C's hope to continue Saturday night against the Mavericks, as they look to bring their record up to 5-5 in the final matchup of their three-game road trip.

More here:

Cs Showing Progress Adjusting to New Defensive System - Celtics.com

Return to ITER Power Facts Main Page

By Steven B. KrivitNov. 9, 2021

Two U.S. Department of Energy scientists with the Advanced Research Projects Agency-Energy (ARPA-E) plan to give a presentation on progress in nuclear fusion research at the annual meeting of the American Physical Society on Wednesday.

Samuel E. Wurzel serves as a technology-to-market advisor for ARPA-E. Scott C. Hsu serves as a program director for the agency.

Samuel E. Wurzel (l) and Scott C. Hsu (r)

On May 23, 2021, Wurzel and Hsu published a preprint of peak triple-product values from many fusion reactor types. The caption of their graph explains that the displayed values are those that set a record for a given [reactor] concept vs. year achieved.

New Energy Times examined only the experiments in the tokamak reactor design category. The graph below isolates only experiments from that category.

However, although Wurzel and Hsu displayed values for non-tokamak concepts through the year 2017, they omitted all data in the tokamak category from 1995 onward.

Table 2 from Version 1 of their pre-print, which appears as Table VI in Version 3 of their pre-print, provides an extensive list of fusion experiments performed in tokamak reactors. Wurzel and Hsu excluded all tokamak experiments beyond 1995.

For a more accurate and honest display of progress in nuclear fusion in the last 70 years, New Energy Times published a report for readers and investors called When Will We Get Energy From Nuclear Fusion?

The latter part of that report presents a preliminary discussion of the awkward fact that one of the two required fuel sources for fusion tritium does not exist as a natural resource on Earth. This fact casts a dark cloud over the long-running sensationalist claims that the fuel for fusion is virtually unlimited.

A subsequent New Energy Times report provides the full discussion of the fusion fuel limitation.

See the original post:

DOE Scientists Will Give Presentation on Fusion Energy Progress - New Energy Times

Phase 1/2/3a and 3b study of ARCT-154 COVID-19 vaccine candidate completed enrollment with over 17,000 participants

Initiated ~2,000 participant ARCT-154 Phase 3c sub-study to compare immunogenicity noninferiority to AstraZeneca COVID-19 vaccine; enrollment to be completed this week

Preparing to file Emergency Use Authorization (EUA) application for ARCT-154, pending interim study results, with the Vietnam Ministry of Health in December 2021; potential for EUA approval in Q1 2022

Investor conference call at 4:30 p.m. ET today

SAN DIEGO, November 08, 2021--(BUSINESS WIRE)--Arcturus Therapeutics Holdings Inc. (the "Company", "Arcturus", Nasdaq: ARCT), a leading clinical-stage messenger RNA medicines company focused on the development of infectious disease vaccines and significant opportunities within liver and respiratory rare diseases, today announced its financial results for the third quarter ended September 30, 2021 and provided corporate updates.

"We are extremely pleased to have rapidly completed full enrollment of the Phase 1/2/3a and 3b cohorts and are looking forward to completing enrollment in the Phase 3c sub-study of the ARCT-154 pivotal trial this week, in collaboration with Vinbiotech. Assuming favorable data, we look forward to filing for Emergency Use Authorization as soon as December of this year," said Joseph Payne, President and CEO of Arcturus Therapeutics. "ARCT-154 has an attractive and differentiated profile as a low dose, self-amplifying mRNA vaccine candidate that targets multiple SARS-CoV2 variants of concern, and we are working diligently with our global manufacturing partners to make it available as soon as possible."

Recent Corporate Highlights

In August, Arcturus, together with Vinbiotech, advanced ARCT-154 into a Phase 1/2/3 study in Vietnam.

In October, Arcturus received approval to proceed with enrollment of the Phase 3b cohort of the study following a review by the Vietnam Ministry of Health of early safety data from the initial 1,000 participants enrolled in the Phase 1/2/3a cohorts.

In November, the Phase 3b cohort of this randomized, placebo-controlled portion of the trial was modified to enroll approximately 16,000 participants with a Phase 3c sub-study of approximately 2,000 participants added to evaluate immunogenicity noninferiority compared to AstraZeneca COVID-19 vaccine. This sub-study provides an additional path to potential full approval.

The Phase 3b cohort is now fully enrolled. Enrollment in the Phase 3c sub-study is underway and expected to be completed this week.

All phases (1, 2, 3a, 3b and 3c) of the ARCT-154 clinical trial in Vietnam are sponsored and funded by Arcturus partner Vinbiotech.

Arcturus and Vinbiotech continue to make progress towards completion of a manufacturing facility in Hanoi capable of producing 200 million doses per year, and technology transfer for commercial manufacturing is in process.

In August, Arcturus announced approval from the Singapore Health Sciences Authority (HSA) to advance ARCT-154 and ARCT-165 into a Phase 1/2 clinical trial to evaluate the vaccines both as a primary vaccination series and as a booster following initial vaccination with Comirnaty. This study was also approved by U.S. Food and Drug Administration (FDA). The Comirnaty booster cohort (with ARCT-021, ARCT-154, ARCT-165) in this study is now fully enrolled. Previously disclosed preclinical data demonstrate that ARCT-154 elicits strong neutralizing immunogenicity in non-human primates to SARS-CoV-2 Alpha, Beta, Gamma, and Delta variants.

ARCT-021 has been selected by a global entity for inclusion in a multinational Phase 3 vaccine trial against COVID-19.

In October, we successfully achieved a milestone in our ongoing LUNAR-HBV program licensed by Janssen Pharmaceuticals (a subsidiary of Johnson and Johnson) and anticipate receiving one million dollars in the fourth quarter.

In July, Arcturus announced approval from the UK Health Research Authority to advance ARCT-810, a novel mRNA-based therapeutic candidate for Ornithine Transcarbamylase (OTC) Deficiency, into a multi-dose Phase 2 clinical study. The ARCT-810 Phase 2 study is a randomized, double-blind, placebo-controlled, nested single and multiple ascending dose study designed to enroll 24 adolescents and adults with OTC deficiency. We anticipate dosing will commence in the first quarter of 2022 and remain on track for interim data in the second half of 2022.

ARCT-032, our mRNA therapeutic candidate for cystic fibrosis, is on track for a Clinical Trial Application (CTA) in first half of 2022.

The LUNAR-FLU mRNA vaccine candidate targeting influenza is on track for a CTA in the second half of 2022.

Financial Results for the Quarter Ended September 30, 2021

Story continues

Revenues in conjunction with strategic alliances and collaborations: Arcturus primary sources of revenues were from license fees and collaborative payments received from research and development arrangements with pharmaceutical and biotechnology partners. For the three months ended September 30, 2021, the Company reported revenue of $2.4 million, compared with $2.3 million in the three months ended September 30, 2020 and $2.0 million for the three months ended June 30, 2021.

Operating expenses: Total operating expenses for the three months ended September 30, 2021, were $56.3 million compared with $23.3 million for the three months ended September 30, 2020 and $55.7 million for the three months ended June 30, 2021.

Research and development expenses increased to $45.4 million year over year compared to $17.7 million from the third quarter of 2020. Research and development expenses were relatively consistent compared to $45.7 million in the second quarter of 2021.

For the three months ended September 30, 2021, Arcturus reported a net loss of approximately $54.1 million, or ($2.05) per basic and diluted share, compared with a net loss of $21 million, or ($0.92) per basic and diluted share in the three months ended September 30, 2020 and a net loss of $54.6 million, or ($2.07) per basic and diluted share in the three months ended June 30, 2021.

The Companys cash balance totaled $413.9 million as of September 30, 2021, compared to a cash balance of $433.6 million at June 30, 2021 and $462.9 million at December 31, 2020. The cash balance includes $40 million received from Vinbiotech Research and Manufacture Joint Stock Company, of which $30 million was received during the current quarter ended September 30, 2021. Based on the current pipeline, the Companys cash position is expected to be sufficient to support operations for two years.

About Arcturus Therapeutics

Founded in 2013 and based in San Diego, California, Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT) is a clinical-stage mRNA medicines and vaccines company with enabling technologies: (i) LUNAR lipid-mediated delivery, (ii) STARR mRNA Technology and (iii) mRNA drug substance along with drug product manufacturing expertise. Arcturus diverse pipeline of RNA therapeutic and vaccine candidates includes mRNA vaccine programs for SARS-CoV-2 (COVID-19) and Influenza, and other programs to potentially treat Ornithine Transcarbamylase (OTC) Deficiency, and Cystic Fibrosis along with partnered programs including Glycogen Storage Disease Type 3, Hepatitis B Virus, and non-alcoholic steatohepatitis (NASH). Arcturus versatile RNA therapeutics platforms can be applied toward multiple types of nucleic acid medicines including messenger RNA, small interfering RNA, replicon RNA, antisense RNA, microRNA, DNA, and gene editing therapeutics. Arcturus technologies are covered by its extensive patent portfolio (with patents and patent applications, issued and filed in the U.S., Europe, Japan, China and other countries). Arcturus commitment to the development of novel RNA therapeutics has led to collaborations with Janssen Pharmaceuticals, Inc., part of the Janssen Pharmaceutical Companies of Johnson & Johnson, Ultragenyx Pharmaceutical, Inc., Takeda Pharmaceutical Company Limited, CureVac AG, Duke-NUS Medical School, and the Cystic Fibrosis Foundation. For more information visit http://www.ArcturusRx.com. In addition, please connect with us on Twitter and LinkedIn.

Forward Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact included in this press release, are forward-looking statements, including those regarding strategy, future operations, the expectations for or likelihood of success of any collaborations (including with respect to LUNAR-HBV), the likelihood of success (including safety and efficacy) of the Companys pipeline (including LUNAR-FLU, ARCT-021, ARCT-032, ARCT-154, ARCT-165 and ARCT-810), anticipated sponsorship and/or funding of clinical trials of the Companys candidates, the Companys efforts to develop a vaccine against COVID-19 and therapeutic potential thereof based on the Companys mRNA therapeutics, the planned initiation, design or completion of clinical trials, the likelihood that the Company will obtain clearance from regulatory authorities to proceed with future planned clinical trials, the likelihood that preclinical or clinical data will be predictive of future clinical results (including with respect to safety, immunogenicity and efficacy), the ability to enroll, and timing for enrollment of, subjects in clinical trials, the timing and nature of any study results, the likelihood that clinical data will be sufficient for regulatory approval or completed in time to submit an application for regulatory approval within a particular timeframe, the likelihood or timing of any regulatory approval, the Companys manufacturing plans or technologies (including with its partner, Vinbiotech), the likelihood that a patent will issue from any patent application, its current cash position and adequacy of its capital to support future operations and the impact of general business and economic conditions. Arcturus may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in any forward-looking statements such as the foregoing and you should not place undue reliance on such forward-looking statements. These statements are only current predictions or expectations, and are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industrys actual results, levels of activity, performance or achievements to be materially different from those anticipated by the forward-looking statements, including those discussed under the heading "Risk Factors" in Arcturus most recent Annual Report on Form 10-K and in subsequent filings with, or submissions to, the SEC, which are available on the SECs website at http://www.sec.gov. Except as otherwise required by law, Arcturus disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.

Trademark Acknowledgements

The Arcturus logo and other trademarks of Arcturus appearing in this announcement, including LUNAR and STARR, are the property of Arcturus. All other trademarks, services marks, and trade names in this announcement are the property of their respective owners.

ARCTURUS THERAPEUTICS HOLDINGS INC. AND ITS SUBSIDIARIES

CONDENSED CONSOLIDATED BALANCE SHEETS

(in thousands, except par value information)

September 30,2021

June 30,2021

December 31,2020

(unaudited)

(unaudited)

Assets

Current assets:

Cash and cash equivalents

$

413,880

$

433,574

$

462,895

Accounts receivable

2,015

2,163

2,125

Prepaid expenses and other current assets

5,071

2,301

2,769

Total current assets

420,966

438,038

467,789

Property and equipment, net

4,843

3,407

3,378

Operating lease right-of-use asset, net

5,983

6,341

5,182

Equity-method investment

670

920

Non-current restricted cash

2,074

107

107

Total assets

$

434,536

$

448,813

$

476,456

Liabilities and stockholders equity

Current liabilities:

Accounts payable

$

8,265

$

10,084

$

10,774

Accrued liabilities

52,358

42,614

20,639

Deferred revenue

57,616

18,071

Read more:

Arcturus Therapeutics Announces Third Quarter 2021 Financial Update and Pipeline Progress - Yahoo Finance

Nov 2, 2021

Tim BontempsESPN

Monty McCutchen, the NBA's vice president of referee development and training, said Tuesday that he's pleased with the league's progress toward eliminating foul calls on non-basketball moves.

He added that his officials are still working to strike the right balance between not calling fouls on those non-basketball moves and missing some defensive fouls, and that the league is still committed to offensive players not being held and grabbed -- what the league refers to as "freedom of movement."

"The referees have done an excellent job," McCutchen said on a conference call with a handful of reporters after a meeting of the league's competition committee earlier Tuesday. "I think in any of this, there's some adjustment period on the shooting foul itself, as to what constitutes the things we're asking.

"There've been a few instances, nothing that's raised to a significant level, where we would still want a defensive foul where it's getting lumped into a non-basketball move. We're in the middle of that adjustment period with the staff. We have staff calls at a higher cadence than we would when we're not implementing something as significant as this, and we're showing them examples so that we can adjust in real-time to meet the demands of the league.

1 Related

"But to be clear: We love where the game has been from a freedom of movement perspective over the past few years, and we do not want to give that up in any way, and the directive will be, and will continue to adjust on my end, that we continue to keep that freedom of movement in place."

Before the season began, the NBA -- and specifically McCutchen -- made it clear in a series of presentations that the league's emphasis this year was to eliminate "non-basketball moves" -- ones the league has deemed to be "abnormal, abrupt or overt" -- from the game. The result has been a decrease in scoring, fouls and free throws per game and a near elimination of those kinds of plays being called as fouls through the opening two weeks of the season.

At the same time, there's also been a perceived increase in physicality on the court -- one that players have, at various points, voiced frustration over. League officials, however, said the data doesn't support the players' argument.

"I'll just say that from a data perspective we're not seeing any evidence of that increased physicality that we're hearing about," said Evan Wasch, the NBA's executive vice president of strategy and analytics. "So, for example, the number of personal, non-shooting fouls is actually up slightly. That's inclusive of plays on the perimeter, handchecks, etc. And we're not seeing an increase in incorrect non-calls, or so-called missed calls, in our game review data, which you would expect to see if defenders were committing more fouls that were going uncalled.

"So again, we're monitoring it, and listening to feedback, but we're not seeing any direct evidence in the data of that so-called increase in physicality."

Overall, though, the message from the league -- and one it said was echoed by the members of the competition committee, which is made up of owners, executives, coaches and players -- was that it is pleased with the way the non-basketball moves have been called, and the way the officiating, in the league's eyes, positively changed the way the game is viewed.

"It's free flowing in one way, the actual ability for a guy to pump fake and go through a normal shooting motion thereafter is getting back to kind of the essence of the game," said Byron Spruell, the NBA's president of league operations. "And we're getting away from the gimmicks. Not to mention names there, but getting away from the gimmicks, and I think that's part of what we intended to set out to do here.

"We focused on non-basketball moves because we want to see basketball moves," Wasch added. "So the notion that you can have these abnormal, overt actions and consider them non-basketball moves by definition means they're not what we want to see in the game."

But while the league pushed back on the idea that there is an increase in physicality on the court, McCutchen said that he's already working with the referees to try to take in the feedback from the players to both address their concerns, and make sure nothing becomes a problem that isn't already.

"I think one of the first things we do when we hear that criticism is players are playing the game," McCutchen said. "To discount what they're telling us would not only be foolish, it would be at a point where we aren't doing our job properly. So we always want to listen. The first place we look is the data.

"The data right now, there's no evidence of that. But that doesn't mean that just because the number of fouls aren't there, doesn't mean a different type of play isn't starting to come along, and we need to monitor that. ... We're monitoring that. We're looking at drives to the hoop to make sure we're within our guidelines. We're looking at freedom of movement to see we're within our guidelines. Then when we do that with our expertise, and compare what we're seeing through our expertise with the data, we have a fuller picture of whether we need to address that."

Spruell added that there has been minimal interaction about the new basketball, with Wilson replacing Spalding as the league's official partner this season. He said outside of some brief discussion about the way the balls are wearing, there has been little feedback.

Sources also confirmed to ESPN that on the competition committee call Tuesday, there was a discussion about the increase in "take" fouls -- the practice of grabbing a player intentionally to stop a fast break. Already a fairly common practice, it has been even more so this year. As a result, the committee agreed to attempt to come up with a potential rule change to eliminate the tactic. While such a change wouldn't happen this season, it could happen as soon as next season.

Go here to read the rest:

NBA pleased with progress in officiating non-basketball moves, sees no evidence of increased physicality - ESPN

Country

United States of AmericaUS Virgin IslandsUnited States Minor Outlying IslandsCanadaMexico, United Mexican StatesBahamas, Commonwealth of theCuba, Republic ofDominican RepublicHaiti, Republic ofJamaicaAfghanistanAlbania, People's Socialist Republic ofAlgeria, People's Democratic Republic ofAmerican SamoaAndorra, Principality ofAngola, Republic ofAnguillaAntarctica (the territory South of 60 deg S)Antigua and BarbudaArgentina, Argentine RepublicArmeniaArubaAustralia, Commonwealth ofAustria, Republic ofAzerbaijan, Republic ofBahrain, Kingdom ofBangladesh, People's Republic ofBarbadosBelarusBelgium, Kingdom ofBelizeBenin, People's Republic ofBermudaBhutan, Kingdom ofBolivia, Republic ofBosnia and HerzegovinaBotswana, Republic ofBouvet Island (Bouvetoya)Brazil, Federative Republic ofBritish Indian Ocean Territory (Chagos Archipelago)British Virgin IslandsBrunei DarussalamBulgaria, People's Republic ofBurkina FasoBurundi, Republic ofCambodia, Kingdom ofCameroon, United Republic ofCape Verde, Republic ofCayman IslandsCentral African RepublicChad, Republic ofChile, Republic ofChina, People's Republic ofChristmas IslandCocos (Keeling) IslandsColombia, Republic ofComoros, Union of theCongo, Democratic Republic ofCongo, People's Republic ofCook IslandsCosta Rica, Republic ofCote D'Ivoire, Ivory Coast, Republic of theCyprus, Republic ofCzech RepublicDenmark, Kingdom ofDjibouti, Republic ofDominica, Commonwealth ofEcuador, Republic ofEgypt, Arab Republic ofEl Salvador, Republic ofEquatorial Guinea, Republic ofEritreaEstoniaEthiopiaFaeroe IslandsFalkland Islands (Malvinas)Fiji, Republic of the Fiji IslandsFinland, Republic ofFrance, French RepublicFrench GuianaFrench PolynesiaFrench Southern TerritoriesGabon, Gabonese RepublicGambia, Republic of theGeorgiaGermanyGhana, Republic ofGibraltarGreece, Hellenic RepublicGreenlandGrenadaGuadaloupeGuamGuatemala, Republic ofGuinea, RevolutionaryPeople's Rep'c ofGuinea-Bissau, Republic ofGuyana, Republic ofHeard and McDonald IslandsHoly See (Vatican City State)Honduras, Republic ofHong Kong, Special Administrative Region of ChinaHrvatska (Croatia)Hungary, Hungarian People's RepublicIceland, Republic ofIndia, Republic ofIndonesia, Republic ofIran, Islamic Republic ofIraq, Republic ofIrelandIsrael, State ofItaly, Italian RepublicJapanJordan, Hashemite Kingdom ofKazakhstan, Republic ofKenya, Republic ofKiribati, Republic ofKorea, Democratic People's Republic ofKorea, Republic ofKuwait, State ofKyrgyz RepublicLao People's Democratic RepublicLatviaLebanon, Lebanese RepublicLesotho, Kingdom ofLiberia, Republic ofLibyan Arab JamahiriyaLiechtenstein, Principality ofLithuaniaLuxembourg, Grand Duchy ofMacao, Special Administrative Region of ChinaMacedonia, the former Yugoslav Republic ofMadagascar, Republic ofMalawi, Republic ofMalaysiaMaldives, Republic ofMali, Republic ofMalta, Republic ofMarshall IslandsMartiniqueMauritania, Islamic Republic ofMauritiusMayotteMicronesia, Federated States ofMoldova, Republic ofMonaco, Principality ofMongolia, Mongolian People's RepublicMontserratMorocco, Kingdom ofMozambique, People's Republic ofMyanmarNamibiaNauru, Republic ofNepal, Kingdom ofNetherlands AntillesNetherlands, Kingdom of theNew CaledoniaNew ZealandNicaragua, Republic ofNiger, Republic of theNigeria, Federal Republic ofNiue, Republic ofNorfolk IslandNorthern Mariana IslandsNorway, Kingdom ofOman, Sultanate ofPakistan, Islamic Republic ofPalauPalestinian Territory, OccupiedPanama, Republic ofPapua New GuineaParaguay, Republic ofPeru, Republic ofPhilippines, Republic of thePitcairn IslandPoland, Polish People's RepublicPortugal, Portuguese RepublicPuerto RicoQatar, State ofReunionRomania, Socialist Republic ofRussian FederationRwanda, Rwandese RepublicSamoa, Independent State ofSan Marino, Republic ofSao Tome and Principe, Democratic Republic ofSaudi Arabia, Kingdom ofSenegal, Republic ofSerbia and MontenegroSeychelles, Republic ofSierra Leone, Republic ofSingapore, Republic ofSlovakia (Slovak Republic)SloveniaSolomon IslandsSomalia, Somali RepublicSouth Africa, Republic ofSouth Georgia and the South Sandwich IslandsSpain, Spanish StateSri Lanka, Democratic Socialist Republic ofSt. HelenaSt. Kitts and NevisSt. LuciaSt. Pierre and MiquelonSt. Vincent and the GrenadinesSudan, Democratic Republic of theSuriname, Republic ofSvalbard & Jan Mayen IslandsSwaziland, Kingdom ofSweden, Kingdom ofSwitzerland, Swiss ConfederationSyrian Arab RepublicTaiwan, Province of ChinaTajikistanTanzania, United Republic ofThailand, Kingdom ofTimor-Leste, Democratic Republic ofTogo, Togolese RepublicTokelau (Tokelau Islands)Tonga, Kingdom ofTrinidad and Tobago, Republic ofTunisia, Republic ofTurkey, Republic ofTurkmenistanTurks and Caicos IslandsTuvaluUganda, Republic ofUkraineUnited Arab EmiratesUnited Kingdom of Great Britain & N. IrelandUruguay, Eastern Republic ofUzbekistanVanuatuVenezuela, Bolivarian Republic ofViet Nam, Socialist Republic ofWallis and Futuna IslandsWestern SaharaYemenZambia, Republic ofZimbabwe

View original post here:

Editorial: An attempt to reverse progress on the Madison River - Bozeman Daily Chronicle

Country

United States of AmericaUS Virgin IslandsUnited States Minor Outlying IslandsCanadaMexico, United Mexican StatesBahamas, Commonwealth of theCuba, Republic ofDominican RepublicHaiti, Republic ofJamaicaAfghanistanAlbania, People's Socialist Republic ofAlgeria, People's Democratic Republic ofAmerican SamoaAndorra, Principality ofAngola, Republic ofAnguillaAntarctica (the territory South of 60 deg S)Antigua and BarbudaArgentina, Argentine RepublicArmeniaArubaAustralia, Commonwealth ofAustria, Republic ofAzerbaijan, Republic ofBahrain, Kingdom ofBangladesh, People's Republic ofBarbadosBelarusBelgium, Kingdom ofBelizeBenin, People's Republic ofBermudaBhutan, Kingdom ofBolivia, Republic ofBosnia and HerzegovinaBotswana, Republic ofBouvet Island (Bouvetoya)Brazil, Federative Republic ofBritish Indian Ocean Territory (Chagos Archipelago)British Virgin IslandsBrunei DarussalamBulgaria, People's Republic ofBurkina FasoBurundi, Republic ofCambodia, Kingdom ofCameroon, United Republic ofCape Verde, Republic ofCayman IslandsCentral African RepublicChad, Republic ofChile, Republic ofChina, People's Republic ofChristmas IslandCocos (Keeling) IslandsColombia, Republic ofComoros, Union of theCongo, Democratic Republic ofCongo, People's Republic ofCook IslandsCosta Rica, Republic ofCote D'Ivoire, Ivory Coast, Republic of theCyprus, Republic ofCzech RepublicDenmark, Kingdom ofDjibouti, Republic ofDominica, Commonwealth ofEcuador, Republic ofEgypt, Arab Republic ofEl Salvador, Republic ofEquatorial Guinea, Republic ofEritreaEstoniaEthiopiaFaeroe IslandsFalkland Islands (Malvinas)Fiji, Republic of the Fiji IslandsFinland, Republic ofFrance, French RepublicFrench GuianaFrench PolynesiaFrench Southern TerritoriesGabon, Gabonese RepublicGambia, Republic of theGeorgiaGermanyGhana, Republic ofGibraltarGreece, Hellenic RepublicGreenlandGrenadaGuadaloupeGuamGuatemala, Republic ofGuinea, RevolutionaryPeople's Rep'c ofGuinea-Bissau, Republic ofGuyana, Republic ofHeard and McDonald IslandsHoly See (Vatican City State)Honduras, Republic ofHong Kong, Special Administrative Region of ChinaHrvatska (Croatia)Hungary, Hungarian People's RepublicIceland, Republic ofIndia, Republic ofIndonesia, Republic ofIran, Islamic Republic ofIraq, Republic ofIrelandIsrael, State ofItaly, Italian RepublicJapanJordan, Hashemite Kingdom ofKazakhstan, Republic ofKenya, Republic ofKiribati, Republic ofKorea, Democratic People's Republic ofKorea, Republic ofKuwait, State ofKyrgyz RepublicLao People's Democratic RepublicLatviaLebanon, Lebanese RepublicLesotho, Kingdom ofLiberia, Republic ofLibyan Arab JamahiriyaLiechtenstein, Principality ofLithuaniaLuxembourg, Grand Duchy ofMacao, Special Administrative Region of ChinaMacedonia, the former Yugoslav Republic ofMadagascar, Republic ofMalawi, Republic ofMalaysiaMaldives, Republic ofMali, Republic ofMalta, Republic ofMarshall IslandsMartiniqueMauritania, Islamic Republic ofMauritiusMayotteMicronesia, Federated States ofMoldova, Republic ofMonaco, Principality ofMongolia, Mongolian People's RepublicMontserratMorocco, Kingdom ofMozambique, People's Republic ofMyanmarNamibiaNauru, Republic ofNepal, Kingdom ofNetherlands AntillesNetherlands, Kingdom of theNew CaledoniaNew ZealandNicaragua, Republic ofNiger, Republic of theNigeria, Federal Republic ofNiue, Republic ofNorfolk IslandNorthern Mariana IslandsNorway, Kingdom ofOman, Sultanate ofPakistan, Islamic Republic ofPalauPalestinian Territory, OccupiedPanama, Republic ofPapua New GuineaParaguay, Republic ofPeru, Republic ofPhilippines, Republic of thePitcairn IslandPoland, Polish People's RepublicPortugal, Portuguese RepublicPuerto RicoQatar, State ofReunionRomania, Socialist Republic ofRussian FederationRwanda, Rwandese RepublicSamoa, Independent State ofSan Marino, Republic ofSao Tome and Principe, Democratic Republic ofSaudi Arabia, Kingdom ofSenegal, Republic ofSerbia and MontenegroSeychelles, Republic ofSierra Leone, Republic ofSingapore, Republic ofSlovakia (Slovak Republic)SloveniaSolomon IslandsSomalia, Somali RepublicSouth Africa, Republic ofSouth Georgia and the South Sandwich IslandsSpain, Spanish StateSri Lanka, Democratic Socialist Republic ofSt. HelenaSt. Kitts and NevisSt. LuciaSt. Pierre and MiquelonSt. Vincent and the GrenadinesSudan, Democratic Republic of theSuriname, Republic ofSvalbard & Jan Mayen IslandsSwaziland, Kingdom ofSweden, Kingdom ofSwitzerland, Swiss ConfederationSyrian Arab RepublicTaiwan, Province of ChinaTajikistanTanzania, United Republic ofThailand, Kingdom ofTimor-Leste, Democratic Republic ofTogo, Togolese RepublicTokelau (Tokelau Islands)Tonga, Kingdom ofTrinidad and Tobago, Republic ofTunisia, Republic ofTurkey, Republic ofTurkmenistanTurks and Caicos IslandsTuvaluUganda, Republic ofUkraineUnited Arab EmiratesUnited Kingdom of Great Britain & N. IrelandUruguay, Eastern Republic ofUzbekistanVanuatuVenezuela, Bolivarian Republic ofViet Nam, Socialist Republic ofWallis and Futuna IslandsWestern SaharaYemenZambia, Republic ofZimbabwe

Excerpt from:

McKinley Bradshaw continues to progress for Cowgirls | University of Wyoming | wyomingnews.com - Wyoming Tribune

- Phase 3 pivotal trial evaluating apitegromab in patients with non-ambulatory Type 2 and Type 3 SMA on track to initiate by year-end 2021

- Update from Part A of the DRAGON Phase 1 trial evaluating SRK-181s ability to overcome primary resistance to checkpoint inhibitors being presented at the Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting

- Initiated Part B dose expansion portion of the DRAGON trial

- U.S. patent issued providing additional product protection for SRK-181 to May 2040

CAMBRIDGE, Mass., November 09, 2021--(BUSINESS WIRE)--Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today reported financial results for the third quarter ended September 30, 2021, and highlighted recent progress and upcoming milestones for its pipeline programs.

"The DRAGON trial addresses two key questions; first, can the selectivity of SRK-181 for TGF1 increase the therapeutic window for inhibition of this signaling pathway thereby enabling higher doses safely, and second, does SRK-181 exhibit anti-tumor activity effective in overcoming resistance to checkpoint inhibitors in solid tumors. We are pleased with the results of Part A, as will be provided this week at the Society for Immunotherapy of Cancer (SITC) Annual Meeting, and we look forward to exploring the efficacy dimension of our therapeutic hypothesis in Part B," said Nagesh Mahanthappa, Ph.D., Interim CEO of Scholar Rock. "Building on the momentum from the first half of this year, we are also excited about the progress with apitegromab and are on track to initiate by year-end the Phase 3 pivotal trial evaluating its efficacy in patients with non-ambulatory Type 2 and Type 3 spinal muscular atrophy (SMA)."

Company Updates and Upcoming Milestones

Apitegromab is a selective inhibitor of myostatin activation being developed as the potential first muscle-directed therapy for the treatment of spinal muscular atrophy (SMA).

Story continues

Additional Exploratory Responder Analyses and Pharmacologic Data from the TOPAZ Phase 2 Trial were Presented at Various Medical Congresses. In September 2021, two posters were presented at the World Muscle Society Virtual Congress, including a late-breaker poster featuring an exploratory analysis evaluating time to achieving various thresholds of improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) scores, which are consistent with the observed dose response in clinical efficacy. In October 2021, a poster presented at the 25th World Congress of Neurology (WCN) described the positive correlation between the magnitude of target engagement and motor function improvements following apitegromab treatment. At the 50th Child Neurology Society Annual Meeting, additional exploratory responder analyses on Hammersmith scale scores were presented, including time to achieve different thresholds of improvement in HFMSE scores.

Phase 3 Trial Evaluating Apitegromab in Patients with Non-Ambulatory Type 2 and 3 Patients Remains on Track to Initiate by Year-End 2021. Scholar Rock is preparing to announce the design of the Phase 3 pivotal study later this month. The company is on track to initiate by the end of 2021 the randomized, double-blind, placebo-controlled Phase 3 trial evaluating apitegromab as add-on therapy for patients on either nusinersen or risdiplam with non-ambulatory Type 2 and Type 3 SMA. This patient population comprises an estimated two-thirds of the overall prevalence of SMA, and the greatest improvements in motor function (as measured by HFMSE) observed in the TOPAZ Phase 2 trial were in patients with non-ambulatory Type 2 and Type 3 SMA receiving apitegromab as add-on therapy to background nusinersen.

SRK-181 is a selective inhibitor of latent TGF1 activation being developed with the aim of overcoming primary resistance to and increasing the number of patients who may benefit from checkpoint inhibitor therapy.

Update from Part A of the DRAGON Phase 1 Trial and Part B Dose to be Presented at the Society for Immunotherapy of Cancer (SITC) 36th Annual Meeting. A poster titled, "First-in-Human Phase 1 Trial of SRK-181: A Latent TGF1 inhibitor, Alone or in Combination with Anti-PD-(L)1 Treatment in Patients with Advanced Solid Tumors (DRAGON trial)" will be presented at the SITC meeting on November 12, 2021. The poster will include initial clinical data from Part A of the DRAGON trial as well as the rationale for the identified Part B dose.

The Company has initiated the Part B dose expansion portion of the trial, which consists of multiple cohorts, each enrolling up to 40 patients with various solid tumors who have demonstrated primary resistance to anti-PD-(L)1 therapy.

U.S. Patent Issued Providing Composition of Matter Product Protection for SRK-181. In September 2021, the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 11,130,803 with an expiry of May 2040, including 313 days of Patent Term Adjustment (PTA). The European counterpart has also been granted.

Third Quarter 2021 Financial Results

For the quarter ended September 30, 2021, net loss was $37.5 million or $1.02 per share compared to a net loss of $23.6 million or $0.79 per share for the quarter ended September 30, 2020.

Revenue was $5.5 million for the quarter ended September 30, 2021, compared to $3.0 million for the quarter ended September 30, 2020 and was related to the Gilead Collaboration Agreement that was executed in December 2018.

Research and development expense was $31.3 million for the quarter ended September 30, 2021, compared to $18.4 million for the quarter ended September 30, 2020. The increase year-over-year primarily reflects manufacturing costs associated with apitegromab, clinical trial costs for SRK-181, and additional personnel and facility-related costs.

General and administrative expense was $11.3 million for the quarter ended September 30, 2021, compared to $8.3 million for the quarter ended September 30, 2020. The increase year-over-year was primarily attributed to additional personnel, professional fees, and facility-related costs.

"As we approach the end of 2021, I am incredibly proud of the execution by the entire Scholar Rock team this year. Not only have we initiated Part B of the DRAGON trial, but were also very close to initiating our Phase 3 pivotal trial for apitegromab," said Ted Myles, CFO and Head of Business Operations of Scholar Rock. "We ended the third quarter with approximately $246 million in cash and cash equivalents and are well-funded to continue executing on our development programs while continuing to invest in our robust discovery platform."

About Scholar Rock

Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, and fibrosis. Scholar Rocks approach to targeting the molecular mechanisms of growth factor activation enabled it to develop a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level. By developing product candidates that act in the disease microenvironment, the Company intends to avoid the historical challenges associated with inhibiting growth factors for therapeutic effect. Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path. For more information, please visit http://www.ScholarRock.com or follow Scholar Rock on Twitter (@ScholarRock) and LinkedIn (https://www.linkedin.com/company/scholar-rock/).

Scholar Rock is a registered trademark of Scholar Rock, Inc.

Forward-Looking Statements

This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rocks future expectations, plans and prospects, including without limitation, Scholar Rocks expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab, SRK-181, and other product candidates and indication selection and development timing, its cash runway, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as "may," "might," "will," "should," "expect," "plan," "anticipate," "believe," "estimate," "project," "intend," "future," "potential," or "continue," and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include preclinical and clinical data, including the results from the Phase 2 trial of apitegromab or Part A of the Phase 1 trial of SRK-181, are not predictive of, are inconsistent with, or more favorable than, data generated from future clinical trials of the same product candidate, including the planned Phase 3 trial of apitegromab in SMA and Part B of the Phase 1 trial of SRK-181, respectively, Scholar Rocks ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline, the data generated from Scholar Rocks nonclinical and preclinical studies and clinical trials, information provided or decisions made by regulatory authorities, competition from third parties that are developing products for similar uses, Scholar Rocks ability to obtain, maintain and protect its intellectual property, the success of Scholar Rocks current and potential future collaborations, including its collaboration with Gilead, Scholar Rocks dependence on third parties for development and manufacture of product candidates including to supply any clinical trials, Scholar Rocks ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and the impacts of public health pandemics such as COVID-19 on business operations and expectations, as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rocks Quarterly Report on Form 10-Q for the quarter ended September 30, 2021, as well as discussions of potential risks, uncertainties, and other important factors in Scholar Rocks subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rocks views only as of today and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of the release, and Scholar Rock undertakes no duty to update this information unless required by law.

Scholar Rock Holding Corporation

Condensed Consolidated Statements of Operations

(unaudited)

(in thousands, except share and per share data)

Three Months Ended September 30

Nine Months Ended September 30

2021

2020

2021

2020

Revenue

$

5,464

$

3,037

$

14,767

$

11,967

Operating expenses

Research and development

31,265

18,383

79,417

52,282

General and administrative

11,276

8,272

29,907

20,459

Total operating expenses

42,541

26,655

109,324

72,741

Loss from operations

(37,077

)

(23,618

)

(94,557

)

(60,774

)

Other income (expense), net

(430

)

57

(1,328

)

862

Net loss

$

(37,507

)

$

(23,561

)

$

(95,885

)

$

(59,912

)

Net loss per share, basic and diluted

$

(1.02

)

$

(0.79

)

$

(2.62

)

Continued here:

Scholar Rock Reports Third Quarter 2021 Financial Results and Highlights Business Progress - Yahoo Finance