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BOSTON, Jan. 22, 2020 /PRNewswire/ --Proteostasis Therapeutics, Inc.,(NASDAQ:PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF) and other diseases caused by dysfunctional protein processing, today announced the presentation of results from an ex vivo study of the Company's proprietary cystic fibrosis transmembrane conductance regulator (CFTR) modulators in organoids from individuals with CF who are ineligible for the current standard of care CFTR modulator therapies due to their genotype, a population of approximately 2,300 adults in Europe alone. The results, outlined in a poster entitled "Intestinal Organoid Models as a Path for Personalized Therapy Development in Cystic Fibrosis," will be presented at the Keystone Symposia on Tissue Organoids taking place on January 19-23, 2020 in Vancouver, BC, Canada.

The study remains on track for collecting tissue samples from up to 500 CF patients with less common genotypes by the end of Q1 2020 for assaying as organoids and for testing responsiveness to investigational CFTR modulators, including Proteostasis' CFTR potentiator, corrector and amplifier, dirocaftor (DIR), posenacaftor (POS) and nesolicaftor (NES), respectively. Data from the organoid study will be used to select a subset of patients for a confirmatory clinical trial, known as the CHOICES trial (Crossover trial based on Human Organoid Individual response in CF - Efficacy Study). This organoid program is a strategic initiative funded by the European Commission, which has invited a select number of drug developers and leading researchers in CF to build a roadmap for personalized therapeutics in CF. Based on the outcome of the study, this transition from precision to personalized medicine for the treatment of CF could begin in patients with less common mutations.

The organoid study seeks to measure the ex vivo responsiveness to the PTI CFTR modulators in tissue samples collected via a rectal suction procedure. The rectal tissue is developed into an organoid or a miniaturized organ that is genetically identical to the patient donor and shares the same micro-anatomy as the organ from which they were derived. Organoid cultures frommore than370 adult CF patients have been established to date. Based on initial genotype analysis, approximately 85% of enrolled patients carry genotypes that lead to CFTR protein synthesis making them eligible for ex vivo study with DIR, POS and NES. Data from the organoid study will be used to select a subset of patients for the confirmatory CHOICES clinical trial. The poster outlining these results is available on the Company's website at proteostasis.com.

"With advancements in models such asorganoid testing used to predict the effectiveness of CFTR modulator treatments, the transition from precision to personalized medicine in this disease is an inevitability," saidGeoffrey Gilmartin, M.D., M.M.Sc., Chief Medical Officer ofProteostasis Therapeutics. "Essential to this transition is the introduction of more therapeutic options that expand the treatment choices for patients and physicians. As the only company in the HIT-CF consortium with a proprietary combination of novel CFTR modulators that have demonstrated positive Phase 2 data, we remain very enthusiastic about its progress and the translation of these results to the clinic in the CHOICES trial."

"Access to CFTR modulators in Europe is challenged by either the ineligibility of patients for approved drugs due to their genotype, or by the lack of reimbursement for approved drugs due to their high cost," saidCornelis K. van der Ent, Professor at the Department of Pediatric Pulmonology of the Wilhelmina Children's Hospital at the University Medical Center in Utrecht, the Netherlands and HIT CF Project Coordinator. "An organoid-assay-based personalized medicine approach offers the potential of a new decision-supporting technology to inform clinical decisionsand provide each patient with CFTR modulators that lead to their highest possible benefit."

CHOICES, which is expected to initiate in mid-2020, will be the first ever personalized medicine-based study in CF. Fully funded by the HIT-CF, this trial is a placebo controlled, double blind, crossover study with an 8-week treatment period and 6 months of uninterrupted dosing. The CHOICES trial will complement the MORE trial (Modulator Options to RestorE CFTR study), a global, Phase 3, randomized, placebo-controlled study in CF subjects with the common F508del homozygous mutation, which is designed to confirm the positive efficacy and tolerability results from a recently completed Phase 2 study of the Proteostasis CFTR modulator triple combination.

About HIT-CF Europe

HIT-CF Europe is a research project which aims to provide better treatment and better lives for people with cystic fibrosis (CF) and rare mutations. To achieve this, drug candidates are first tested on patient-derived organoids in qualified laboratories across Europe. Subsequently, based on the measured signal in the organoids, a smaller group of patients will be invited to participate in a clinical trial. All participating centers are part of the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN). The project has received funding from the European Union's Horizon 2020 research and innovation program under grant agreement number 755021. For more information, visit http://www.hitcf.org.

About Proteostasis Therapeutics, Inc.

Proteostasis Therapeutics, Inc. is a clinical stage biopharmaceutical company developing small molecule therapeutics to treat cystic fibrosis and other diseases caused by dysfunctional protein processing. Headquartered in Boston, MA, the Proteostasis Therapeutics team focuses on identifying therapies that restore protein function. For more information, visit http://www.proteostasis.com.

Safe Harbor

To the extent that statements in this release are not historical facts, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Words such as "aim," "may," "will," "expect," "anticipate," "estimate," "intend," and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Examples of forward-looking statements made in this release include, without limitation, statements regarding the potential of our proprietary combination therapies for the treatment of CF, the potential benefit to patients of our proprietary combination therapies, expected timing of patient enrollment in our clinical studies and cohorts for our clinical programs, including our planned Phase 3 programs and initiation of registrational or pivotal studies. Forward-looking statements made in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved. Such risks and uncertainties include, without limitation, the possibility final or future results from our drug candidate trials (including, without limitation, longer duration studies) do not achieve positive results or are materially and negatively different from or not indicative of the preliminary results reported by the Company (noting that these results are based on a small number of patients and small data set), uncertainties inherent in the execution and completion of clinical trials (including, without limitation, the possibility that FDA or other regulatory agency comments delay, change or do not permit trial commencement, or intended label, or the FDA or other regulatory agency requires us to run cohorts sequentially or conduct additional cohorts or pre-clinical or clinical studies), in the enrollment of CF patients in our clinical trials in a competitive clinical environment, in the timing of availability of trial data, in the results of the clinical trials, in possible adverse events from our trials, in the actions of regulatory agencies, in the endorsement, if any, by therapeutic development arms of CF patient advocacy groups (and the maintenance thereof), and those set forth in our Annual Report on Form 10-K for the year ended December 31, 2018, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2019 and our other SEC filings. We assume no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

CONTACTS:

Investors:David Pitts / Claudia StyslingerArgot Partners212.600.1902david@argotpartners.com/ claudia@argotpartners.com

Media:David RosenArgot Partners212.600.1902david.rosen@argotpartners.com

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SOURCE Proteostasis Therapeutics, Inc.

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Proteostasis Therapeutics Announces Keystone Symposia Presentation of Organoid Study from More than 300 Adult CF Patients - BioSpace

CF Industries Holdings, Inc. [NYSE: CF] stock went down by -3.48% or -1.54 points down from its previous closing price of $44.25. The stock reached $42.71 during the last trading session. During the course of the last 5 trading session in the week, CF share price went up during 2 out of 5 days, that way losing -5.97% in the period of the last 7 days.

CF had two major price movements with the latest trading session as the price jumped to a high of $44.32, at one point touching $41.92. The latest high is set lower that the high price recorded in the period of the last 52 weeks, set at $44.32. The 52-week high currently stands at $55.15 distance from the present share price, noting recovery 2.03% after the recent low of $38.90.

Its stock price has been found in the range of 38.90 to 55.15. This is compared to its latest closing price of $44.25.

Keep looking out for the next-scheduled quarterly financial results that this company is expected to put out, which is slated to occur on Wed 12 Feb (In 21 Days).

Now lets turn to look at profitability: with a current Operating Margin for CF Industries Holdings, Inc. [CF] sitting at +15.67 and its Gross Margin at +20.46, this companys Net Margin is now 10.40%. These metrics indicate that this company is not generating as much profit, after accounting for expenses, compared to its market peers.

This companys Return on Total Capital is 6.37, and its Return on Invested Capital has reached 8.70%. Its Return on Equity is 8.87, and its Return on Assets is 2.16. These metrics show a mixed bag, which means that this investments attractiveness can be quickly increased or decreased in the short term, depending on future updates CF financial performance.

Turning to investigate this organizations capital structure, CF Industries Holdings, Inc. [CF] has generated a Total

What about valuation? This companys Enterprise Value to EBITDA is 8.96 and its Total Debt to EBITDA Value is 3.00. The Enterprise Value to Sales for this firm is now 3.00, and its Total Debt to Enterprise Value stands at 0.28. CF Industries Holdings, Inc. [CF] has a Price to Book Ratio of 3.28, a Price to Cash Flow Ratio of 6.75 and P/E Ratio of 19.58. These metrics all suggest that CF Industries Holdings, Inc. is more likely to generate a positive ROI.

Shifting the focus to workforce efficiency, CF Industries Holdings, Inc. [CF] earns $1,476,333 for each employee under its payroll. Similarly, this companys Receivables Turnover is 15.71 and its Total Asset Turnover is 0.33. This publicly-traded organizations liquidity data is also interesting: its Quick Ratio is 1.37 and its Current Ratio is 1.81. This company, considering these metrics, has a healthy ratio between its short-term liquid assets and its short-term liabilities, making it a less risky investment.

CF Industries Holdings, Inc. [CF] has 216.30M shares outstanding, amounting to a total market cap of $9.24B. Its stock price has been found in the range of 38.90 to 55.15. At its current price, it has moved down by -22.56% from its 52-week high, and it has moved up 9.79% from its 52-week low.

This stocks Beta value is currently 1.04, which indicates that it is 2.98% more volatile that the wider market. This stocks Relative Strength Index (RSI) is at 31.90. This RSI score is good, suggesting this stock is neither overbought or oversold.

Shares of CF Industries Holdings, Inc. [CF], on the whole, present investors with both positive and negative signals. Wall Street analysts have mixed reviews when it comes to the 12-month price outlook, and this companys financials show a combination of strengths and weaknesses. Based on the price performance, this investment is somewhat risky while presenting reasonable potential for ROI.

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CF Industries Holdings, Inc. [CF] Analyzing the CF using Fundamentals and Trend Analysis - The Dwinnex

The global cystic fibrosis therapeutics market is expected to witness significant growth during the forecast period majorly due to availability of personalized drug therapy & molecular prosthetics for the treatment of cystic fibrosis. Furthermore, raise in prevalence of cystic fibrosis and surge in awareness about cystic fibrosis across the globe boost the cystic fibrosis market trend. For instance, according to the Cystic Fibrosis Foundation Patient Registry, in the U.S. more than 30,000 people are living with cystic fibrosis and more than 70,000 worldwide.

In addition, approximately 1,000 new cases of CF are diagnosed each year and more than 75 % of people with Cystic fibrosis F are diagnosed by age 2. Moreover, increase in R&D investments made by government in the healthcare sector drives the cystic fibrosis therapeutics market growth. However, high cost of the cystic fibrosis treatment and introduction of generic drugs hinder the growth of the market. Developing nations such as countries of Asia-Pacific region are not so aware reading the cystic fibrosis diagnosis of the newborns. In various nations, new born are not tested for CF during birth and national registries are lacking in data, thereby hampering the cystic fibrosis market growth.

The global CF therapeutics market size was estimated at USD 3,920.5 million in 2018 and is expected to grow at a CAGR of 16.7% from 2018 to 2025

To request a sample copy or view summary of this report, click the link below:

http://www.digitsnmarkets.com/sample/4306-cystic-fibrosis-(cf)-therapeutics-market

Based on cystic fibrosis therapeutics market analysis, the CFTR modulators segment dominated the market in 2018 and is anticipated to continue its dominance during the forecast period. This is attributed to increased usage of these drugs for the treatment of cystic fibrosis. This segment is also estimated to grow at the fastest rate during the forecast period. Further, the introduction of CFTR drugs, Kalydeco, Orkambi and Symdeko is one such innovation. These drugs are now widely being used for the treatment of cystic fibrosis and many other CFTR drugs are under clinical trials. The main target of these drugs is to increase the life expectancy of the patients. Moreover, advances in the diagnosis and therapeutics have significantly increased the survival of patients.

Europe constitutes the highest market share in the global cystic fibrosis therapeutics market in 2018. North America is owing to the increase in incidence of cystic fibrosis. Moreover, increase in governmental support for innovation and rise in funding of projects for R&D of various cystic fibrosis drugs further fuel the market growth in this region. UK is the highest revenue contributor due to increase in prevalence of cystic fibrosis in this region. Moreover, surge in national newborn screening program in the UK propels the market growth. In addition, many companies are involved in developing drugs for individuals who do not get benefitted with CFTR drugs. However, North America is projected to grow at the highest CAGR during the forecast period.

Key players operating in cystic fibrosis therapeutic markets are Genentech, Inc., Novartis AG, Gilead Sciences, Inc., Vertex Pharmaceuticals Incorporated, AbbVie Inc., GlaxoSmithKline Plc., Johnson & Johnson, Allergan plc, Pharmaxis Ltd., and Mylan N.V.

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Global Cystic Fibrosis (CF) Therapeutics Market , Trends, Analysis, Opportunities, Share and Forecast 2017-2025 - Dagoretti News

SCOTTSDALE, Ariz., Jan. 21, 2020 /PRNewswire/ --MC Companies San Antonio has won the Cystic Fibrosis Foundation's 'Rookie Fundraiser Team of the Year' award for their presence as a national sponsor and powerful impact at San Antonio Great Strides. Through combined efforts of their employees at The Place at Castle Hills and The Place at Oak Hills, both located in the Castle Hills neighborhood, and the MC Companies Sharing the Good Life Foundation, MC was able to build a team of 22 Great Strides Walkers and donate a total of $4,478 to the San Antonio Great Strides Walk.

MC is extremely honored by this award, but even more so by the impact they were able to make on the national stage. In 2019 MC had Great Strides walk teams in five cities, officially designating them as a National Sponsor. Throughout the course of 2019, MC Companies participated in 7 different Cystic Fibrosis walks across three states with 118 walkers, and raised an impressive $39,000. Combined with MC Companies' employee driven Sharing the Good Life Foundation donation of $63,000, MC Companies was able to donate a grand total of $100,000 the Cystic Fibrosis Foundation in 2019.

MC Companies is no stranger to the Cystic Fibrosis Foundation and is a long-time supporter of the Great Strides Walk.However, their philanthropic ambitions do not end with CF. Giving back it as the core of MC, which is why they created their employee driven Sharing the Good Life Foundation which encourages and gives employees countless opportunities to volunteer, get involved, and give back to our local communities.

2019 was an incredible year for MC's contributions to the CF community, and they have no intention of slowing down. A new year marks a new opportunity to give back and better our communities. MC is invigorated by the challenge to surpass their 2019 goals and make 2020 another landmark year in their support for the CF community.

You can donate to our 2020 walk teams and learn more about MC's efforts by visiting the MC Companies Great Strides National Team Page. Find MC at these 2020 Great Strides walks.

Build your walk team, find and join other walk teams, and learn how you can get involved at the Cystic Fibrosis Foundation Great Strides website.

About MC Companies: MC Companies http://www.mccompanies.com is a real estate investment, development, construction, and management company specializing in the multifamily properties and commercial markets. MC Companies has completed over $750 million in multi-family and commercial value-added transactions since 1985.

Photos:https://www.prlog.org/12806931

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MC Companies Wins 'Rookie Fundraiser Team of the Year' by the Cystic Fibrosis Foundation For National Sponsorship Impact - PRNewswire

Stay Ahead of Wall Street. Find out what is happening to CF Industries Holdings, Inc. (NYSE:CF) stock today? Its price is nose-diving -1.72 points, trading at $42.53 levels, and is down -3.9% from its previous close of $44.25. The shares seem to have an active trading volume day with a reported 1420231 contracts so far this session. CF shares had a relatively better volume day versus average trading capacity of 1.98 million shares, but with a 0.22 billion float and a -2.4% run over a week, its definitely worth keeping an eye on. The one year price forecast for CF stock indicates that the average analyst price target is $52.1 per share. This means the stock has a potential increase of 22.5% from where the CF share price has been trading recently.

During the recent trading session for CF Industries Holdings, Inc. (NYSE:CF), the company witnessed their stock drop by $-2.84 over a week and tumble down $-5.7 from the price 20 days ago. When compared to their established 52-week high of $55.15, the high they recorded in their recent session happens to be higher. Their established 52-week high was attained by the company on 01/08/19. The recent low of $38.9 stood for a -22.89% since 08/03/19, a data which is good for most investors who are looking to take advantage of the stocks recent rise. A beta of 1.04 is also allocated to the stock. Since the beta is greater than one, it implies that the stock is more volatile than the market, a data that traders are keeping close attention to.

Looking at the current readings for CF Industries Holdings, Inc., the two-week RSI stands at 31.24. This figure suggests that CF stock, for now, is neutral, meaning that the shares are stable in terms of price movement. The stochastic readings, on the other hand, based on the current CF readings is similarly very revealing as it has a stochastic reading of 15.47% at this stage. This figure means that CF share price today is being overbought.

Technical chart claims that CF Industries Holdings, Inc. (CF) would settle between $45.34/share to $46.42/share level. However, if the stock price goes below the $43.69 mark, then the market for CF Industries Holdings, Inc. becomes much weaker. If that happens, the stock price might even plunge as low as $43.12 for its downside target. The stock is currently in the red zone of MACD, with the indicator reading -0.75. Traders are always alerted for the move of a stock above or below the zero line due to the fact that the reading is an indicator of the position of the short-term average relative to the long-term average. If the MACD is above the zero line, then the short-term average relative is above that of the long-term average, thus implying an upward momentum. Vice versa is the case if the MACD is below the zero line.

Analysts at Bernstein lowered their recommendation on shares of CF from Mkt Perform to Underperform in their opinion released on December 10. BofA/Merrill analysts bumped their rating on CF Industries Holdings, Inc. (NYSE:CF) stock from Neutral to Buy in a separate flash note issued to investors on November 25. Analysts at Cowen are sticking to their Outperform stance. However, on September 26, they lifted price target for these shares to $62 from $59.

CF equity has an average rating of 2.92, with the figure leaning towards a bullish end. 16 analysts who tracked the company were contacted by Reuters. Amongst them, 8 rated the stock as a hold while the remaining 8 were split even though not equally. Some analysts rate the stock as a buy or a strong buy while others rated it as a sell. 7 analysts rated CF Industries Holdings, Inc. (NYSE:CF) as a buy or a strong buy while 1 advised that investors should desist from purchasing the stock or sell them if they already own the companys stock.

Moving on, CF stock price is currently trading at 17.7X forward 12-month Consensus EPS estimates, and its P/E ratio is 20.3 while for the average stock in the same group, the multiple is 19.6. CF Industries Holdings, Inc. current P/B ratio of 3.2 means it is trading at a premium against its industrys 1.3.

CF Industries Holdings, Inc. (CF)s current-quarter revenues are projected to climb by nearly -7.7% to hit $1.04 billion, based on current consensus estimate. The firms full-year revenues are expected to expand by over 4.6% from $4.43 billion to a noteworthy $4.63 billion. At the other end of the current quarter income statement, CF Industries Holdings, Inc. is expected to see its adjusted earnings surge by roughly 38.1% to hit $0.29 per share. For the fiscal year, CFs earnings are projected to climb by roughly 77.4% to hit $2.2 per share.

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How Far CF Industries Holdings, Inc. (CF) Will Fall Today - Find News

HUDSON, Wis.-- The Hudson girls hockey team ran into a hot goalie in a 2-1 Big Rivers Conference loss at home to Chippewa Falls/Menomonie Tuesday night, Jan. 14.

The Raiders fired 43 shots at Sabre goalie Haley Frank, including 20 in the second period, but the only one that found the back of the net came off the stick of Leah Parker in the first period.

Parkers unassisted goal 10 minutes, 43 seconds into the game gave the Raiders a 1-0 lead, but the only scoring the rest of the way came from the Sabres. Ella Ausman tied it up at the 12:02 mark of the second period and Sidney Polzin netted the game-winner just over a minute into the third to give Chippewa Falls/Menomonie its first BRC victory of the season.

Hudson goalie Alayna Kunshier finished with 15 saves as the Raiders slipped to 4-9-1 overall, 1-2-0 in conference play.

Hudson had its nonconference game at Onalaska scheduled for Saturday, Jan. 18, cancelled by weather. After visiting the sixth-ranked St. Croix Valley Fusion (11-5-0) in River Falls Tuesday, Jan. 21, the Raiders will host University School of Milwaukee (3-7-4) Friday, Jan. 24, at 7 p.m.

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Hudson girls stifled by hot CF/M goalie - RiverTowns

The Toronto Blue Jays have a collection of outfielders from which t hey have to select their center fielder. it doesnt look as though theyre looking to pull the trigger on a big deal to land a Starling Marte, so theyll head to Spring Training with their crop of in house options and hope someone takes the job and runs with it.

None of the options the club has is currently what we would call ideal. They guys like Anthony Alford and Jonathan Davis who are more what youd think of when you think of a natural CF. But, they have not been able to show they can handle big league pitching, at least not to the point where they should be given everyday at bats. Lourdes Gurriel Jr.has found a home in left, even if some think it would be worth trying him in CF. Teoscar Hernandez has speed and saw over 600 innings there last season. But, his -7 DRS and UZR/150 of -13.2 look worse than his -1 OAA. In fact, many are ready to limit him to being the DH in 2020.

All of this leads me to be more and more comfortable with Randal Grichuk in CF. Firstly, the guy is good for 25-30 home runs. I mean, he had better be since his 2019 OBP was .280. Maybe Cavan Biggio can work with him on that. Regardless, Grichuk gives you the power and that is difficult to ignore.

The defensive side of things is what really has me thinking that Grichuk would be just fine in CF. In nearly 2000 career innings, he has put up 14 DRS in that position, including 2 last season. His career UZR/150 is -1.2, but -0.6 in 2019. He put up 2 of his 6 OAA in 2019 in CF. Grichuk is a solid defender. Hes not likely to win a Gold Glove any time soon, but he can be relied upon for the bulk of the playing time.

Grichuk is also not going to be Superman out there. Fangraphs tells us that he made zero Impossible or Remote plays, 20% of the Unlikely, but 100% of the Even, 100% of the Likely and 99.1% of the routine plays. Hes not flashy and wont end up on highlight reels, but he is dependable.

None of this is to suggest that Randal Grichuk is someone I would choose in CF if I were building a team. In a perfect world, he would be playing in right field for the Blue Jays. However, in the context of the 2020 season, considering the options available to manager, Charlie Montoyo, it looks like Grichuk will get the nod for the most playing time. Since there is no pressure to win right now, the club can afford to use what they have until they can find a better option.

Maybe moving Biggio to CF makes the overall club better. Doubtful, but maybe. Maybe Gurriel can make the transition look easy. Maybe. Maybe Hernandez will prove to be more than a CF back up. Maybe Alford finds his bat in 2020. Maybe Davis does. Time will tell. Until then, the Blue Jays will go with Randal Grichuk in CF and that is OK with me.

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Shaun Doyle is a long time Blue Jays fan and writer! He decided to put those things together and create Jays From the Couch. Shaun is the host of Jays From the Couch Radio, which is highly ranked in iTunes, and he has appeared on TV and radio spots.

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Why Blue Jays Would Be OK with Grichuk in CF - Jays From the Couch

Omar P. Haqqani, for the Daily News

Newly approved cystic fibrosis treatment offers promise

On October 21, the U.S. Food and Drug Administration (FDA) approved a new therapy that could improve the lives of thousands of people living with cystic fibrosis (CF). The first triple combination therapy has been made available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients twelve years and older with cystic fibrosis that have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. There are approximately 2,000 known mutations of the CFTR gene, but the most common mutation is the F508del mutation. It is estimated to represent 90% of the cystic fibrosis population. Trikafta is very effective on this mutation. The Cystic Fibrosis Foundation (CFF) calls it, "the single greatest therapeutic advancement in the history of CF."

What is cystic fibrosis?

Cystic fibrosis mutations prevent CFTR proteins to become functional and that prevents the movement of chloride to the cell surface in the body. Chloride is an element of salt and when its movement is hindered, mucus in various organs becomes thick and sticky. The improperly developed mucus causes germs and bacteria to remain in the lungs. This results in infection, inflammation and other complications, including respiratory failure.

CF also presents a danger in the pancreas. The mucus causes digestive enzymes not to function properly, resulting in malnutrition and poor growth, due to the prevention of the release of digestive enzymes. Mucus can also cause liver disease by blocking the bile duct. It may also alter the ability for men to father children.

In the U.S., more than 30,000 people are living with cystic fibrosis and more than 70,000 are diagnosed worldwide. More than 50% of them are age 18 or older.

Symptoms of cystic fibrosis

Symptoms of CF appear in proportion to damage to particular organs. The most common and noticeable symptoms occur when the lungs become damaged. Lung infections such as pneumonia and bronchitis, along with wheezing, or shortness of breath are frequent symptoms. Additionally, persistent coughing may occur and often produce phlegm.

When CF affects other organs, symptoms may include salty tasting skin, poor weight gain, despite a good appetite, and infertility. Other symptoms may be bulky stools or problems with bowel movement.

Diagnosis and treatment

CF is a genetic disease. 75% of cases are diagnosed before a child is two years old. As with the symptoms, treatment is focused on the organs exhibiting the most impact from the mutation. In looking at specific symptoms, a physician may prescribe airway clearance, pancreatic enzyme supplements and individualized physical fitness programs. These therapies have often proven effective.

Nasal inhalers that include antibiotics to keep nasal passages clear are used in airway clearance therapy. To improve the absorption of various nutrients, pancreatic enzyme supplement capsules may be prescribed, along with multivitamins.

The available therapies that target the defective protein are treatment options for some patients with cystic fibrosis, but many patients have mutations that are ineligible for treatment. The new therapy is a combination of three drugs that target the defective CFTR protein. It helps the protein made by the CFTR gene mutation function more effectively.

The prescribing information for Trikafta includes warnings related to elevated liver function tests (transaminases and bilirubin), the use at the same time with other products that are inducers or inhibitors of another liver enzyme called Cytochrome P450 3A4 (CYP3A), and the risk of cataracts. Patients and their caregivers should speak with a health care professional about these risks and any medicines they take before starting treatment.

Patients with cystic fibrosis should also speak with their physicians about having tests performed to understand which gene mutations they have.

To learn more about cystic fibrosis, its symptoms and treatment, log on to vascularhealthclinics.org.

Ask Dr. Haqqani

If you have questions about your cardiovascular health, including heart, blood pressure, stroke lifestyle and other issues, we want to answer them. Please submit your questions to Dr. Haqqani by e-mail at questions@vascularhealthclinics.org.

Dr. Omar P. Haqqani is the chief of Vascular and Endovascular Surgery at Vascular Health Clinics in Midland: http://www.vascularhealthclinics.org

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Newly approved cystic fibrosis treatment offers promise - Midland Daily News

Periodically, Crowdfund Insider revisits the Reg CF sector of online capital formation. Reg CF or Regulation Crowdfunding may have garnered most of the attention from popular media but really there are three individual crowdfunding exemptions including Reg A+ and Reg D 506c.

Under Reg A+ you must file an extensive offering circular with the entire offering process costing around $300,000, according to one estimate. But Reg A+ enables an issuer to raise up to $50 million from both accredited and non-accredited investors.

Under Reg D 506c, you may raise an unlimited amount of money but only from accredited investors. This is the most popular crowdfunding exemption and Reg D (5o6c and 5o06b) is a trillion-dollar market.

Issuers using Reg CF may only raise $1.07 million and must utilize a FINRA regulated Funding Portal or a broker-dealer. Due to the low cap on funding, frequently issuers will do a side-by-side Reg D 506c offering to circumvent the extremely low amount you may raise.

Last time CI revisited the number of approved Funding Portals was in July. Since that time, several new funding portals have joined the approved list and several have exited.

Regarding Reg CF funding portal exits two more have departed this sector of crowdfunding.

EquityBender based in Charleston, South Carolina, is no more. The domain just indicates a private site.

Seeding VR is the other exit. As the name indicates, Seeding VR was targeting the virtual reality sector. Apparently, it even attempted to launch a crowdfunding platform in the UK. Today, both domains simply time out.

This brings the total of funding portal exits to 12 with at least two the direct result of some time of enforcement action: UFP LLC and DreamFunding Marketplace.

The three additions to the list bring the total to 50 FINRA approved funding portals with one in question as Fundpaas has long been on the suspended list and is expected to join the exits.

The three recent additions include:

Fundopolis has yet to list its first offering but appears ready to launch its first issuer. According to its website, Fundopolis expects to also enable issuers to raise capital under both Reg A+ and Reg D as well.

Infrashares is described as follows:

InfraShares is a crowdfunding platform that pools investment from individuals into large sums of development capital for critical infrastructure projects (roads, bridges, airports, mass transit, water systems, renewable energy and schools).

This platform is utilizing Reg D as well. Currently, there are two issuers posted on the site -both under Reg D 506c.

Prospect Equity does not appear to have a live site as of yet.

As Crowdfund Insider reported in October, Reg CF has raised over $300 million in securities offerings since the exemption became actionable in May of 2016, providing capital to over 2000 campaigns. This is according to a report by Crowdfund Capital Advisors.

Overall, Reg CF can be called a success as it has helped smaller companies raise much-needed growth capital while creating new jobs. But multiple shortcomings hobble the exemption thus undermining its potential success.

First, the fund cap is widely recognized as far too low.

Average seed rounds in the US stand at about $2.2 million typically using Reg D. A good number raise much more.

In the UK, the most robust crowdfunding market in the world, issuers may raise as much money as they want. A prospectus requirement at 8 million creates a virtual speedbump for issuers looking to raise more than that amount.

The most successful Reg CF Funding Portal, Wefunder, stated earlier this year that limitations to the exemption may make the exemption an option of last resort.

Republic, another leader in the Reg CF sector, published a letter providing the perspective from issuers that have utilized Reg CF to raise growth capital. The letter, signed by 23 different founders and CEOs of early-stage firms, indicated that Reg CF has its benefits but suffers from serious limitations.

Every industry participant has voiced their concerns to both Congress and the Securities and Exchange Commission (SEC). While some policymakers have supported common-sense updates, since 2016, little has been accomplished.

And it is not just the funding cap that undermines Reg CF.

A report by the SEC from this past summer touched upon many of the issues.

Common sense changes that allow for special purpose vehicles (SPVs) that safeguard smaller investors while making it simpler to gain access to higher-quality deals is a no brainer.

There is also the 12g trap that may compel a company to become a reporting company once it has 500 investors. A problem that is antithetical to investment crowdfunding.

The Association of Online Investment Platforms (AOIP), an advocacy group for online capital formation, has posted a position paper with its goals.

So is Reg CF ready to scale? Probably not without some changes to the rules. Many of the larger platforms now have broker dealer licenses and offer other services. Doing enough $1 million deals in a year to cover your costs can be quite difficult.

The SEC is currently going through a regulatory review, as defined by a concept release, which seeks to improve the exemption ecosystem while harmonizing the alphabet soup of rules. This may be the best opportunity the industry has to see some impactful improvements. If not, it will be up to Congress to step up and do the job. Dont hold your breath.

Read more from the original source:

Reg CF Funding Portals: 50 in Total with Several Exits and Several Additions. Is Reg CF Ready to Scale? - Crowdfund Insider

BLACKFOOT Bingham County has shown itself as a community that rallies behind special causes and special needs whenever they arise.

The fight against cystic fibrosis (CF) is one of those causes. Its a a hereditary disease that affects the lungs and digestive system, with the body producing thick, sticky mucus that can clog the lungs and obstruct the pancreas. CF can be life-threatening, and people with the condition tend to have a shorter-than-normal life span.

Its a disease that has reached around the county. Its a disease that brings a rallying cry every July with the Wolverine Canyon Marathon, which raises awareness of CF and helps raise money for research into ultimately finding a cure, highlighting one person each year in the county who battles CF.

This year, with help from donors and local participants including sponsors and the runners themselves, the marathon raised $14,863.44. The VanOrden family in the Thomas area received a check for $7,431.42. They plan to donate a portion of of that to cystic fibrosis-related causes, use some of the funds to pay off medical expenses for their 4-year-old son, Taylor, who has CF, and put the remaining money in a medical fund for Taylor to use for future medical expenses.

In 2017, Frannie Baumgartner Crumley of Blackfoot was highlighted, turning 40 that year, having a double lung transplant to extend her life. Emersyn Drollinger, a young Shelley girl, was highlighted in 2018. She was diagnosed with cystic fibrosis as an infant. Taylor VanOrden started showing signs of the disease just a couple of days after being born at Eastern Idaho Regional Medical Center.

Frannie daughter of Alice and Gary Baumgartner lost a sister, Julia, to the disease.

The disease and the daily treatment that goes with it is all that Taylor has ever known, aside from the love and support of his family and friends. Hes become so accustomed to the daily ritual, months short of his fifth birthday, that there are things he does on his own to help himself, such as strapping on his own percussion vest which is needed at least twice a day, used to relieve the mucus that can build up in the body and become thick and sludgy much like overused motor oil in a cars engine, with life-threatening results if left untreated.

CF AWARENESS

We were in ignorant bliss for the first 36 hours after Taylor was born, his father Jeff says.

A couple of days after he was born, Jeff and Taylors mother, Lyndsey, knew there were complications.

That was when he threw up greenish mucus. He hadnt had a bowel movement in 36 hours, finding blockage in the small intestine. On day two after being born, Taylor was Life Flighted to Primary Childrens Medical Center in Salt Lake City.

At the time, doctors were unsure whether it was CF or an intestinal disorder. Then came the family history. Neither parent had known there had been CF in the family until Lyndsey was told that her father had a cousin who died of CF at age 18, an average lifespan for people with CF back then.

There are no outward symptoms at all. Its known as an invisible disease, and its a disease he will likely fight his entire life. But there is still hope that positive steps in funding and research will improve quality of life if not upholding the mantra of the Cystic Fibrosis Foundation (https://www.cff.org/): We will not rest until we find a cure.

Its a mantra the VanOrden family lives with every day for Taylor.

According to the CFF, in people with the disease, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. When the protein is not working correctly, its unable to help move chloride a component of salt to the cell surface. Without the chloride to attract water to the cell surface, the mucus in various organs becomes thick and sticky.

In the lungs, the mucus clogs the airways and traps germs, like bacteria, leading to infections, inflammation, respiratory failure, and other complications. Minimizing contact with germs is a top concern for people with CF.

In the VanOrden home, having two school-age children in Taylors sister Emma, 9, and brother Tommy, 7, who are exposed to germs on a daily basis means extra caution on a daily basis is required, along with avoiding germs from anywhere. Emma and Tommy have both been tested for CF and are clear.

According to the CFF, signs of CF can include very salty-tasting skin; persistent coughing, at times with phlegm; frequent lung infections including pneumonia or bronchitis; wheezing or shortness of breath; poor growth or weight gain in spite of a good appetite; frequent greasy, bulky stools or difficulty with bowel movements; and male infertility.

A CLOSE-KNIT UNIT

Frannie Baumgartner Crumley calls younger CF fighters {span}Cysters and fiBros, Shes hoped for a cure to be found, so that one day the fighters will be {span}able to take one pill and be cured of CF.{/span}{/span}

{span}{span}Frannie stands as an inspiration to families of those dealing with CF, and there is a tight bond between those families. Its there for support, to keep up the fight and the drive.{/span}{/span}

{span}{span}Theres quite a few families around here who have it, said Lyndsey, who Jeff says is highly involved in a CF advisory board that meets every week. The support theyve seen with the marathon has been a very humbling experience, they said, seeing the community rally together.{/span}{/span}

{span}{span}Frannie is incredible. Shes lived into her 40s, Lyndsey added. Frannie reminds me of what Taylor will be. She has been a role model. Shes given us hope.{/span}{/span}

The goal that Frannies had of getting down to a one-pill cure sounds much better than the 13 maintenance medications Taylor must take daily now, with some of them being adult-sized tablets. One of those medications, Orkambi an oral granule costs around $300,000 per year. Taylor has to take that twice a day, and its delivered in a box keeping them supplied four weeks at a time.

With the medical coverage the family is lucky to have, the family has a copay of $45 a month for Orkambi.

It does cause anxiety, Jeff said.

The VanOrdens say they have a good medical plan, but premiums keep rising substantially. They meet their yearly deductible by the end of January. The government provides no funding for research, which leaves donations through the foundation and efforts such as the Wolverine Canyon Marathon.

The foundation is pushing hard for a cure, Lyndsey said. Its making substantial progress.

Theres something about the CF world. There are a lot of passionate people.

Follow this link:

The 'CF World': Fighters work toward a cure - Post Register