Amicus Therapeutics (NASDAQ:FOLD) is a global biotherapeutics company focused on rare genetic devastating diseases. The company has advanced its precision medicine, Galafold (migalastat), in treating patients in Europe with Fabry disease (alpha galactosidase A deficiency), a rare X-linked genetic lysosomal disorder in which sphingolipids are not metabolized properly. Galafold, an orally administered drug, is the first medicine approved (EMA but not yet FDA) for treatment of Fabry disease. Other goals for 2017 include submitting a J-NDA (Japan) for migalastat, establishing a clinical plan for ATB200/AT2221 in Pompe disease, completion of phase 3 clinical trial in epidermolysis bullosa.

FOLD announced its regulatory plan with FDA for U.S. treatment with Galafold in advancing it's Fabry disease program including two phase 3 trials in late 2016. As previously mentioned, the EMA approved use of Galafold for treatment of Fabry disease. The company published data from its pivotal trial in the New England Journal of Medicine. A statistically significant benefit was conferred by Galafold in 50 patients with treatable mutant galactosidase alleles. However, a closer look at the trial endpoints reveals that the study failed to reach its primary endpoints including greater than 50% reduction in GL-3 inclusions per kidney, and if all 67 patients were included in the study (including mutant alleles that are not expected to benefit by Galafold). More studies are needed for FDA going forward. It could be well worth the company's investment, as the market for Fabry's disease is estimated to be in excess of $1.2 billion by 2024.

December 2016, FOLD announced positive early phase 1/2 data for Pompe disease, a rare genetic disorder leading to the buildup of glycogen in the body, particularly the muscles, which become impaired in function. The study showed a positive safety profile with no serious adverse events and generally showed musculatoprotection as shown by biomarkers of muscle damage. The study is divided into three cohorts: non-ambulatory ERT-switch, ERT-switch, and ERT-naive. Creatine kinase, alanine aminotransferase, and aspartate aminotransferase levels showed a trend towards improvement in half the patients and were stable in all. ATB200/AT2221 has a unique mechanism of treatment that uses ATB200, a recombinant functional alpha-glucosidase enzyme carrying mannose-6 phosphate moieties designed to increase uptake. AT2221 is a pharmacological chaperone co-treated to stabilize the compound. The company believes that its Pompe program (with market of $1.2 billion) will be a driver in its growth with 12% CAGR as a world's leading rare disease company. Key study readout dates include Q2 and Q3 2017.

SD-101 is currently in phase 3 studies as a topical for Epidermolysis Bullosa, and FOLD believes it will be the first-to-market therapy for the rare indication. The inherited disease is characterized by blistering of keratinized outer skin, wet skin (such as mouth), and internal organs. Serious complications include infection, pain, and even death. The company was granted FDA Breakthrough Therapy Designation in 2013 based on results from its phase 2a study, having demonstrated wound closure in all disease types. Strong Bio has previously written about the impacts of FDA Breakthrough Therapy Designation on biotechnology stocks, and if you are an investor and have not perused the article, now might be a good time, as it is a part of the investment plan for FOLD. Phase 3 top-line data for the 160 patient study in which 95% of the patients elected to continue the open-label extension is due Q3 2017. This statement could be the company's way of saying they find it likely it will have clinical benefit for patients. Since it has been observed that FDA breakthrough therapy status stocks get volatile late in stage 3, any sharp drops may indicate stock manipulation that interested investors might jump on with a small position. Strong Bio regards FOLD as a nice investment prospect for any unexplainable late Q2 early Q3 pullbacks. With significant market potential of $1 billion, severe symptoms, and 30,000 sufferers in the U.S. alone, its worth watching for that pullback. One competitor, RegeneRx (OTCQB:RGRX) has initiated phase 3 trials for RGX-137 (active ingredient thymosin beta 4 wound healing gel) in the condition as well.

Cash burn was $55 million in first quarter 2017. Cash on hand at end Q1 was $280 million. The current runway is expected to last through the second half of 2018. Seven analysts average about $12 per share for FOLD, which is currently trading at about $8, which may be a slight pullback from fair value. Strong Bio will look for dips in price below $6 for no a brainer initial position. If all three drugs get FDA approval, this stock could be off to the races. Strong Bio has learned a lesson from Amicus. Rare diseases may have surprisingly large markets!

Risk factors for FOLD could include dilution (which may be yet another entry opportunity). Risks also include FDA approval and/or regulatory delays for all three major indications. Clinical trial design will be key, because FDA wants a clear metric upon which to agree with FOLD to approve these rare disease therapies. Large scale GMP-compliant manufacturing for U.S. application will also be a significant but manageable obstacle. Because the company has a market cap over $1.1 billion dollars, SD-101 key readout will be very important in terms of valuation for FOLD stock. If approved by FDA, the stock could easily triple in value over the next year. With ATB200/AT2221 being regarded as a key driver in value by the company, 2017 is going to be a pivotal year for FOLD. With three candidates all on the verge of pivotal data, this is a must-watch!

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Read this article:
Amicus Therapeutics: A Rare Find In Biotechnology - Seeking Alpha

Wednesday was a good day for stocks, and the Dow Jones Industrials and S&P 500 both climbed through milestone levels. Most market participants attributed the positive sentiment to the Federal Reserve, which released the minutes of its latest monetary policy meeting during the afternoon. The central bank revealed plans to clamp down on the size of its balance sheet, which it initially expanded in the aftermath of the financial crisis to provide liquidity to the bond market and additional stimulus to the U.S. economy. Investors were pleased that the Fed believes that it's no longer necessary to extend that level of monetary accommodation to the economy. In addition, some individual companies had extremely good news, and Abercrombie & Fitch (NYSE:ANF), Triumph Group (NYSE:TGI), and Puma Biotechnology (NASDAQ:PBYI) were among the best performers on the day. Below, we'll look more closely at these stocks to tell you why they did so well.

Shares of Abercrombie & Fitch climbed 6% in the wake of reports that the teen retailer might receive an acquisition bid from a consortium of investors. According to The Wall Street Journal(subscription required), industry peer American Eagle Outfitters (NYSE:AEO) and private equity company Cerberus Capital Management are looking at putting together a potential buyout offer for Abercrombie & Fitch, following speculation that other players in the industry might also be interested in consolidation. A&F has been dealing with takeover speculation for a while, and it has typically noted that any discussions wouldn't necessary translate into actual offers. Yet with Abercrombie set to release its first-quarter financial results Thursday, investors will want to see signs that the company can take care of its challenges on its own -- or else they'll start clamoring more loudly for a buyout to take place.

Image source: Getty Images.

Triumph Group stock soared over 30% after the company announced its fiscal fourth-quarter financial results and resolved a dispute with aircraft manufacturer Bombardier. The aerospace components and systems specialist said that sales fell 13% from year-ago levels, and it posted a GAAP loss of $126.8 million. With challenges in its aerospace structures business, Triumph has focused on amending contracts and addressing operational and financial challenges, and the company's transformation plan has led to improving free cash flow and cost savings. Investors were also happy that Triumph reached a settlement of all of its disputes with Bombardier. Triumph said that the agreement "resets the commercial relationship between [Triumph] and Bombardier and allows each of them to better achieve their business objectives going forward."

Finally, shares of Puma Biotechnology jumped 30%. The biopharmaceutical company earned a hoped-for approval from the U.S. Food and Drug Administration advisory panel looking at its neratinib candidate treatment for breast cancer. The panel voted 12 to 4 in favor of recommending the drug to the FDA, and although panelists expressed some thoughts about potentially limiting the size of the group of women eligible to use the drug, investors nevertheless took the news as a positive. The FDA still needs to make its own decision about Puma's drug, and it isn't bound by the opinion of the advisory panel. Nevertheless, today's recommendation moves Puma one step further to getting a big win under its belt, and shareholders recognized that fact with the second big move in the stock this week.

Dan Caplinger has no position in any stocks mentioned. The Motley Fool has no position in any of the stocks mentioned. The Motley Fool has a disclosure policy.

Read the original post:
Why Abercrombie & Fitch, Triumph Group, and Puma Biotechnology Jumped Today - Motley Fool

The Technical Chart For Puma Biotechnology, Inc. (PBYI) Is Very Revealing Today NY Stock News The technicals for Puma Biotechnology, Inc. (PBYI) has spoken via its technical chart and the message is loud and clear. Based on that message, this is the relevant information necessary to make sense of that current setup. Often the difference between ... and more »

Original post:
The Technical Chart For Puma Biotechnology, Inc. (PBYI) Is Very Revealing Today - NY Stock News

While 2017 has been a pretty good year overall for biotechs - the two largest ETFs in the space, the iShares Nasdaq Biotechnology ETF (NASDAQ:IBB) and the SPDR S&P Biotech ETF (NYSEARCA:XBI), are up 10% and 17%, respectively - it's tough to forget that both of these funds are still about 25% off of their 2015 highs. IBB, which is heavily influenced by the largest biotech names, has been impacted by 50% drops in Gilead (NASDAQ:GILD) and Biogen (NASDAQ:BIIB). XBI has a much more diversified all-cap mix but has experienced similar results.

But not all news coming out of the sector is bad. In fact, one biotech ETF has been downright ripping it since its launch at the end of 2014. The BioShares Biotechnology Products ETF (NASDAQ:BBP), which invests in companies that have at least one primary product that's received FDA approval, is up roughly 42% since its inception at the end of 2014 compared to a loss of 4% for IBB during the same time frame.

IBB Total Return Price data by YCharts

As fund advisor Virtus says on its website, companies that the fund invests in are "typically more established companies with much clinical trial failure risk behind them. They have already successfully completed multiple human clinical trials and have received FDA approval to sell and market a drug." That sounds a lot like IBB so what's the big differentiator between the two funds? It's BBP's focus on small- and micro-cap biotechs. Nearly 60% of fund assets are dedicated to this space whereas IBB has nearly 40% of the portfolio alone invested in the big five of Regeneron (NASDAQ:REGN), Biogen , Celgene (NASDAQ:CELG), Amgen (NASDAQ:AMGN) and Gilead .

So what did BBP have going for it that IBB didn't over the past year or so? I think it's a combination of portfolio construction and M&A.

BBP has a portfolio that has performed almost as well as can be expected, especially in 2017. Take a look at this chart with the year-to-date performance of the fund's biggest components.

That's exactly what you want to see out of your ETFs - the largest holdings performing the best. Part of that is due to the fact that the fund is equal-weighted and rebalanced semiannually (the last rebalance was done on December 15th). Still, that's a lot of companies whose stocks have risen by 20% or more.

Among the top 10 holdings, all have posted double-digit gains with seven components delivering 25%+ gains.

The other advantage the fund has is that a number of its holdings are in the sweet spot of being developed enough to generate meaningful revenue from their approved product line yet being small enough that they can be potential takeover targets. We've seen that within the fund multiple times recently. Relypsa (RYLP) has a top holding when it got bought out by Galenica (OTC:GNHAY). Not an M&A deal, but the fund's stake in Progenics (NASDAQ:PGNX) spiked when its partnership with Valeant (NYSE:VRX) was announced. Current holdings such as Exelixis (NASDAQ:EXEL) and Acadia (NASDAQ:ACAD) have been rumored as potential takeover targets for a while so further action could be in store for the fund in the near future as well.

Conclusion

Equal weighting the mature biotech players has been a strategy that's paid off for investors in the last year. The relatively limited exposure to the likes of Celgene and Gilead, which are still nearly 50% off of recent highs, has made the fund more attractive than its more well-known counterpart.

This fund will underperform when the mega-cap biotech names begin to rally again, but over the long-term this ETF should hold up well to IBB given its more diversified portfolio.

----------

If you found this article of value and are interested in more ETF analysis and investing strategies, please consider following me by clicking on the "Follow" button at the top of this article next to my name. Even if you don't, thanks for taking the time to read!

----------

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Link:
BBP: A Diversified Biotech ETF - Seeking Alpha

WALTHAM, Mass.--(BUSINESS WIRE)--Bioverativ Inc. (NASDAQ: BIVV), a global biotechnology company focused on the discovery, development and commercialization of innovative therapies for hemophilia and other rare blood disorders, today announced that it has entered into a definitive agreement to acquire South San Francisco-based True North Therapeutics, a privately-held, clinical-stage rare disease biotechnology company, for an upfront payment of $400 million plus assumed cash. True North investors are also eligible to receive additional payments of up to $425 million contingent on the achievement of future development, regulatory and sales milestones.

As part of the acquisition, Bioverativ will obtain worldwide rights to True Norths lead candidate, TNT009, a first-in-class monoclonal antibody in development to treat cold agglutinin disease (CAD). CAD is a rare and chronic hemolytic condition that often leads to severe anemia, requiring numerous transfusions, and can result in life-threatening thrombotic events. There are no approved therapies for CAD, which occurs in approximately 16 people per million globally, including an estimated 5,000 people in the United States.

In May 2017, the U.S. Food and Drug Administration (FDA) granted TNT009 breakthrough therapy designation for the treatment of hemolysis in patients with primary CAD, and plans for the full clinical development program, including a registrational program, are underway. Breakthrough therapy designation was created by the FDA to expedite the development and review of medicines that target serious or life-threatening conditions and have shown preliminary evidence of potential clinical benefit.

One of our strategic priorities is to invest thoughtfully in business development with a focus on building our pipeline in areas where we believe we can make a real difference for patients, said John Cox, Chief Executive Officer of Bioverativ. This acquisition of True North is aligned with those goals and with our vision to become the leading rare disease company focused on blood disorders. It strengthens our pipeline with a potential first-in-class therapy to treat CAD, a rare blood disorder with a high unmet patient need.

People living with CAD currently have no approved treatment options and suffer with a significant disease burden including crippling fatigue, frequent transfusions and an increased risk of life-threatening thrombotic events such as pulmonary embolism and stroke, Mr. Cox continued. By combining True Norths industry-leading science in complement biology with Bioverativs expertise in hematology and robust clinical and commercial capabilities, we believe we can bring meaningful progress to CAD patients. In addition, this transaction is consistent with our capital allocation philosophy, and we expect it to create significant value for our shareholders over the long term."

Todays announcement and TNT009s breakthrough therapy designation are testaments to the innovative science underpinning our lead candidate and the strength of the True North team. We are delighted to have progressed our pipeline to this stage, said Nancy Stagliano, PhD, Chief Executive Officer of True North. Bioverativ is well positioned to advance the development and commercialization of TNT009 on behalf of CAD patients who are greatly in need of safe and effective treatments.

The FDA granted TNT009 breakthrough therapy designation based on data from a Phase 1b trial, which showed that TNT009 normalized hemoglobin levels in six of six study participants with primary CAD, increasing hemoglobin by an average of 4g/dL and resolving their anemia. All participants were transfusion free while on treatment. TNT009 was generally well tolerated. As of December 21, 2016, five of six participants (83.3%) with primary CAD experienced at least one adverse event; all were mild to moderate in severity and no adverse event was assessed as related to TNT009 by the investigator.

TNT009 is designed to selectively inhibit the classical complement pathway by targeting C1s and directly impacting the central mechanism of CAD. The only therapy in development that targets C1s, TNT009 has received orphan drug designation from the FDA and the European Medicines Agency. A number of other rare, complement-mediated diseases share the basic underlying pathological mechanism involving C1s that could potentially be addressed by TNT009.

True Norths second molecule, TNT020, is a discovery-stage, follow-on monoclonal antibody that targets activated C1s with the potential for less-frequent dosing and subcutaneous administration.

The acquisition will be financed through a combination of cash on hand and debt. It is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott-Rodino Antitrust Act of 1976 in the United States, and is expected to close in mid-2017.

Conference Call and Webcast Bioverativ will hold a brief conference call and webcast today at 8:30 a.m. ET to discuss the transaction. The conference call can be accessed through the Investors section of Bioverativs homepage, investors.bioverativ.com, and an audio replay will subsequently be available on the website for at least one month.

About Cold Agglutinin Disease (CAD) CAD is a debilitating autoimmune hemolytic anemia in which autoantibodies target red blood cells, leading to red blood cell destruction via complement activation initiated by the C1 complex, causing chronic anemia, severe fatigue and potentially fatal thrombotic events. CAD occurs in approximately 16 people per million, affecting an estimated 5,000 people in the United States. Typically, symptom onset begins around age 60. There are currently no approved therapies for CAD. Treatment is aimed at normalizing hemoglobin levels through blood transfusions, steroids or off-label immunotoxic therapy; however, current treatment options are often intensive, incomplete or nondurable, leaving patients dependent upon frequent transfusions, which can lead to chronic iron overload.

About TNT009 TNT009 is a first-in-class, humanized, monoclonal antibody that is designed to selectively inhibit the classical complement pathway by targeting C1s, a serine protease within the C1-complex in the complement pathway of the immune system. With a unique mechanism of action and high target specificity, TNT009 is designed to selectively inhibit disease processes in the classical complement pathway while maintaining activity of the alternative complement pathway and lectin complement pathway, which are important for immune surveillance and other functions.

About True North Therapeutics True North Therapeutics is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of first-in-class product candidates for complement-mediated diseases. The companys lead product candidate, TNT009, is designed to selectively inhibit a target in the classical complement pathway. True Norths antibody drug development is focused on the treatment of certain rare diseases mediated by the complement system. True North Therapeutics is located in South San Francisco, California. For more information, please visit http://www.truenorthrx.com.

About Bioverativ Bioverativ is a global biotechnology company dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development and commercialization of innovative therapies. Launched in 2017 following separation from Biogen Inc., Bioverativ builds upon a strong heritage of scientific innovation and is committed to actively working with the blood disorders community. The companys mission is to create progress for patients where they need it most and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. For more information, visit http://www.bioverativ.comor follow@bioverativ on Twitter.

Safe Harbor This press release contains forward-looking statements, including statements relating to: the completion of the transaction with True North and timing thereof; the expected benefits of the transaction, including the potential to bring meaningful progress to CAD patients and create shareholder value; and anticipated clinical trials and the timing thereof. These forward-looking statements may be accompanied by such words as anticipate, believe, could, estimate, expect, forecast, intend, may, plan, potential, project, target, will and other words and terms of similar meaning. You should not place undue reliance on these statements.

These forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: risks that the transaction will be completed in a timely manner or at all; risks of failure to satisfy the conditions precedent to completion of the transaction, including the ability to secure regulatory approvals in a timely manner or at all; uncertainty of whether Bioverativ can achieve the expected benefits from the transaction and successfully integrate the operations of True North; risks of unexpected costs, liabilities or delays; integration difficulties, including the ability to retain key personnel; risks that Bioverativ will be able to obtain financing on satisfactory terms, if at all; risks relating to the protection of intellectual property; risks and uncertainties relating to product development, clinical trials, regulatory process and approvals, and commercialization that may impact or alter Bioverativs anticipated business plans, strategies and objectives; and other risks and uncertainties described in the Risk Factors section of Bioverativ's quarterly and annual filings with the Securities and Exchange Commission.

These statements are based on our current beliefs and expectations and speak only as of the date of this press release. We do not undertake any obligation to publicly update any forward-looking statements, whether as a result of new information, future developments or otherwise.

Read more from the original source:
Bioverativ to Acquire Clinical-Stage Rare Disease Biotechnology ... - Business Wire (press release)

By Nicolas Chahine, InvestorPlace Contributor|May 23, 2017, 1:28 pm EDT

The iShares Nasdaq Biotechnology Index (ETF) (NASDAQ:IBB) is technically tight and a move is coming. While the direction of which is uncertain I am willing to cautiously bet that it will be upwards.

After all, this is an uber-bullish equity market and it will take a really negative headline to cause a selloff, so betting long is easier than shorting. Even when we do sell off, buyers are buying every dip.

Originally I was looking to trade several biotech tickers, but the list got so long that I decided to cast a net over the sector rather than fish with a hook. This will also make managing the risk more streamlined. It came down to a toss-up between IBB and theSPDR S&P Biotech (ETF) (NYSEARCA:XBI).

Click to Enlarge I chose the IBB because it offers me bigger absolute returns with clear levels.

Other names I considered included large components of the ETF so the logic is the same but the vessel has a different ticker. Besides, selling risk on an ETF reduces the chances of a single stock headline which is common among biotech companies.

Fundamentally, the IBB carries a high ticket price but is not currently over-inflated. Four of its top six largest components have shed a lot of froth recently so they shouldnt have far to go without a change in the macro. At almost $300 per share its a tall order to buy outright for most investors but using spreads delivers profits without much risk as this April trade did for me.

The weekly chart shows clear levels where markets avoid, one above $315 and the other below $250.

The Trade: Sell the IBB Dec $245 put and collect $5.50 per contract. Here I have a 90% theoretical chance of keeping my maximum gains. If price falls below $239.50 then I accrue losses there and lower.

Usually I like to balance my trades by selling opposing risk. But since my thesis today revolves around a directional move, I will opt out from selling call risk. Instead, I could even add to the potential profits by adding more positive bias. But I will delay entry for now.

The Juice (Optional): Buy the IBB June 30 $295/$300 debit call spread for $2.30. If price rallies past my spread I stand to double my money.

While adding the optional calls may seem aggressive but its not. I dont even need a rally to profit. As long as IBB stays above my sold puts, any premium I recapture from selling my debit calls spread would be pure profit.

Selling puts is risky business so I only risk what I can afford to lose.

Learn how to generate income from options here. Nicolas Chahine is the managing director of SellSpreads.com. As of this writing, he did not hold a position in any of the aforementioned securities. You can follow him on Twitter at @racernicand stocktwits at@racernic.

Article printed from InvestorPlace Media, http://investorplace.com/2017/05/ishares-nasdaq-biotechnology-index-etf-ibb-etf-board/.

2017 InvestorPlace Media, LLC

Read the rest here:
The Move in iShares Nasdaq Biotechnology Index (ETF) (IBB) ETF Is Coming -- Get on Board - Investorplace.com