Patient deaths, testing concerns and “weak arguments.” Those are just a few of the issues the FDA had with Sarepta Therapeutics’s recent drug application, according to new documents released this week.

Sarepta (SRPT) exon-53 skipping DMD drug, Vyondys 53, gets approval in the United States following a CRL in August.

Frustrated with the slow pace of drug discovery and desperate to help their loved ones, untrained family members take the work into their own hands by starting biopharma companies. But few such companies have been successful.

– Biopsy results from the third patient dosed at 2E14 vg/kg in the SGT-001 IGNITE DMD clinical trial provide further support for continued development – – Solid.

Solid Biosciences Inc. (SLDB) today announced changes to its organizational structure to create a leaner company focused on advancing SGT-001, a gene transfer candidate for the treatment of Duchenne muscular dystrophy (Duchenne). In December 2019, the company reported biomarker data from two patients that provide evidence that SGT-001 is biologically active with differentiated properties which Solid believes warrants further evaluation. “We believe SGT-001 holds great potential for the treatment of Duchenne, and in order to effectively evaluate its potential for patients, we made some difficult choices to focus our resources and help extend our cash runway,” said Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences.

The Insider Monkey team has completed processing the quarterly 13F filings for the September quarter submitted by the hedge funds and other money managers included in our extensive database. Most hedge fund investors experienced strong gains on the back of a strong market performance, which certainly propelled them to adjust their equity holdings so as […]

Chardan Capital analyst Gbola Amusa reiterated a Buy rating on Solid Biosciences (NASDAQ:SLDB) Llc on Saturday, setting a price target of $5, which is approximately 104.08% above the present share price of $2.45.

Investors need to pay close attention to Solid Biosciences (SLDB) stock based on the movements in the options market lately.

Virtual oral presentation reviews interim clinical biomarker data for SGT-001 and provides data on expression and co-localization of microdystrophin and dystrophin associated.

Sarepta (SRPT) grants ex-U.S. marketing rights of its gene therapy DMD candidate, SRP-9001, to Roche for an upfront payment of $1.15 billion. Sarepta is also eligible to receive $1.7 billion in milestone payments.

Key highlights of the past week include regulatory and pipeline updates along with collaboration deals.

Solid Biosciences Inc. (SLDB) today announced that, due to the public health impact of the novel coronavirus (COVID-19) outbreak and governmental restrictions limiting the number of people who may gather together, its 2020 Annual Meeting of Stockholders (the “Annual Meeting”), to be held on Tuesday, June 16, 2020 at 8:00 a.m. Eastern Time, has been changed to a virtual-only meeting. The virtual-only Annual Meeting is expected to provide stockholders with the same rights and opportunities to participate as they would have at an in-person meeting. Stockholders are entitled to participate in and vote at the Annual Meeting if they were a stockholder at the close of business on April 20, 2020, the record date, or hold a legal proxy for the meeting provided by the stockholder’s bank, broker or nominee as of such record date.

Solid Biosciences (SLDB) has reported weaker-than-expected 1Q20 results, with FY GAAP EPS of -$2.91 missing Street consensus by $0.09. However the update for the SGT-001 phase I/II clinical program in Duchenne muscular dystrophy (DMD) was the real point of interest for investors.Disappointingly, as Solid revealed, the program remains on clinical hold at the FDA. “We share the FDA’s commitment to patient safety and are working collaboratively with the agency to resolve the clinical hold,” said Ilan Ganot, CEO of Solid Biosciences. “We consider patient safety the utmost priority and believe the clinical development of SGT-001 could offer meaningful benefits to patients with this devastating disease.”In April 2020, Solid submitted a response to the FDA that included changes to the clinical protocol designed to enhance patient safety, as well as information related to improvements to its manufacturing process.The FDA has now requested further data and analyses relating to this manufacturing process. According to Solid, the company is currently generating this data and expects to submit it to the FDA before 4Q20.Chardan Capital’s Gbola Amusa is staying on side despite the delay. He notes that the FDA is not requesting any further pre-clinical data, and writes: “We rate Solid Biosciences Buy based on the potential for Solid to help address what we believe are capacity constraints existing among AAV-based gene therapy (GT) players targeting the vast market of Duchenne muscular dystrophy” he says.As the analyst points out, Solid has an extremely modest valuation (>$100 mm market cap) relative to a >$50 bn global DMD AAV-based GT market opportunity. Indeed, Amusa’s $5 price target now indicates potential upside of 99% from current levels, with shares trading down 43% year-to-date.The risks surrounding SGT-001 are well-known to the market, says Amusa, which means that any improvements in the market’s view of SGT-001 safety and/or efficacy could lead to significant share price momentum with >5x or even >10x share price upside.Nonetheless he warns that SLDB is a particularly risky pick, as it “is more likely to fail to get its product to market than to succeed.” Notably, Evercore ISI analyst Ravi Mehrotra downgraded Solid from buy to hold on May 7. (See Solid Biosciences stock analysis on TipRanks).The company ended 1Q20 with $53.6 mm in cash and equivalents, down from $83.5 mm at end 4Q19. Solid expects cash “will be sufficient to fund its operating expenses and capital requirements into 2021”.Related News: Pfizer Sets Wheels In Motion For Covid-19 Vaccine Production Ramp AstraZeneca-Merck Ovarian Cancer Treatment Gets FDA Approval Seres Therapeutics Reports Weak Earnings, But Significant Upside Lies Ahead More recent articles from Smarter Analyst: * GM Director Displays Confidence in Company Despite Recent Troubles * iQIYI Sinks 4% As Online Ad-Revenue Falls Sharply * Pfizer Seeks To Raise $4 Billion From Debt Sale * Bluebird Prices New Shares At $55, Seeks To Raise $500 Million

Top Ranked Momentum Stocks to Buy for May 13th

More than 500 biotech companies operate in the US, doing their part to make it the world’s hub for innovation. Only about 20 of them turn a profit, though, highlighting the dangers of the biotech sector. High overhead, long research terms, and the uncertainties of clinical testing and regulatory approval all weigh on them, making biotech at once the riskiest and most potentially profitable of the stock market’s segments.Enter Joseph Edelman. The hedge fund veteran started Perceptive Advisors back in 1999 with just $6 million, and his hedge has grown to hold over $5 billion in assets under management today. Edelman’s fund’s flagship, the Perceptive Life Sciences Fund, specializes in small- and mid-cap biotech companies. That Edelman knows how to spot the winners is clear from the Life Sciences Fund’s annualized gains of 30% since its inception.In the words of Brad Loncar, of Loncar Investments, biotech is the “most volatile sector in the stock market… More so than any industry, it’s important to be diversified... Even excellent companies with great science fail all the time and have big setbacks, because it’s that difficult of a business.” Edelman has thrived in this difficult business for the last twenty years, and in his most recent 13F filing, for the quarter ending this past September 30, he doubled down on three companies.We’ve looked up each in the TipRanks database, to find a line on why.Amarin Corporation (AMRN)A mid-cap company, valued at $7 billion, Amarin is the largest of the companies on this list. Perceptive bought over 1.4 million shares of AMRN in the third-quarter, bringing the fund’s total holding to 7.6 million shares. Edelman’s fund first bought into Amarin in Q3 2018, and now holds more than $173 million of AMRN shares.It’s not hard to understand why a biotech-focused hedge fund would go large on Amarin. The company has only one product on the market, but that one is a true humdinger. The medication, Vascepa, is an omega-3 based treatment for hypertriglyceridemia, with a proven record of reducing triglycerides in adult patients. Vascepa has been commercially available since 2013, and is predicted to reach $2.2 billion in annual sales by 2024.Vascepa’s success in the open market is clear from Amarin’s quarterly results. The company reported Q3 revenues of $112 million, up 103% from the year-ago quarter, and a cash-on-hand position of $677 million. Underneath these strong numbers was equally strong prescription performance for Vascepa – the company reported over 865,000 new and repeat orders for the drug in the quarter, up 9.9% sequentially. Importantly, the FDA is set to make a final decision on a label expansion for Vascepa to include patients at risk of a heart attack and other major adverse cardiovascular events (MACE). The company should get either green or red light by December 28, 2019. AMRN shares have been getting plenty of love lately from Wall Street’s analysts, but also some skepticism.Louise Chen, writing from Cantor, says, “The peak sales potential of Vascepa is underappreciated and that upward earnings revisions should drive Amarin shares higher.” Her price target of $35 suggests an upside of over 70% to the stock. (To watch Chen's track record, click here)Jefferies analyst Michael Yee is also bullish on AMRN, citing the upcoming FDA decision. He states, “While some investors do not like the risk/reward debate on the December 28 potential FDA approval and label wording, [we are] not too concerned about the exact wording. Based on commentary by the FDA at the end of the panel, [we think] the agency might be broader on the label than some investors expect. Expect Amarin to rise toward $30 per share...” Yee’s price target implies a 51% upside. (To watch Yee's track record, click here)Meanwhile, Amarin stock has hit a new 52-week high last Friday, with a valuation that almost reached the $9 billion level. Indeed, the stock's valuation is way too high for Oppenheimer analyst Leland Gershell who initiated coverage on AMRN with an "underperform" rating (i.e. Sell) and a $7.00 price target. Gershell opined, "We forecast sales growth to underwhelm and heavy selling costs to impede profitability. Furthermore, we believe that a ~12-month stream of late-stage competitor data starting next month will increasingly weigh on shares as these products, which we believe offer superior profiles, are factored into models. While some may regard AMRN as a probable M&A target, we see the likelihood of this outcome as only shrinking with time." Gershell's price target implies about 60% downside from current levels.Overall, the word on the Street rings largely bullish on this fish oil drug maker, with TipRanks analytics demonstrating AMRN as a Buy. Out of 10 analysts tracked in the last 3 months, 7 are bullish on Amarin stock, 2 remain sidelined, and 1 is bearish on the stock. With a return potential of nearly 35%, the stock's consensus target price stands at $27.30. (See Amarin stock analysis on TipRanks)Solid Biosciences (SLDB)This small-cap biotech focuses on developing treatments for Duchenne muscular dystrophy (DMD), the most common form of the devastating genetic disorder. DMD is a sex-linked recessive trait, and affects about 1 in 5,000 male children at birth. SLDB’s main product, drug candidate SGT-001, is at the Phase I/II stage of development.Clinical failures and safety concerns have dogged Solid through much of this year. Back in May, SGT-001 saw disappointing numbers in an early testing readout, leading to a 64% drop in share value for SLDB – but also to an increased dosage in ongoing test. That led to a ‘patient event’ in May, in which a test subject reported an adverse reaction. The FDA stepped in and put a clinical hold on the drug at that point.The company was able to address FDA concerns and resume testing, but last week it saw another FDA clinical hold. This hold was again the result of a patient experiencing an ‘adverse event.’ According to reports, the patient suffered kidney damage. SLDB shares, which had not truly recovered from February’s hit, dropped an additional 64% on the news.With this as background, we can look at Edelman’s purchase. His fund bought more than 2.8 million shares of SLDB in Q3, bringing the total holding to 6.7 million shares. At the end of the quarter, the shares were priced near $10 each; they are now trading at just $3.68. This is down 64% from the end of Q3. Edelman has a history of holding on to his biotech purchases for a long time, regardless of performance or clinical testing results. He has said that "an investor who doesn’t like risk should not be in this business."Solid hasn’t been getting much love on Wall Street analyst lately. Credit Suisse analyst Martin Auster writes of the SGT-001 program, “This is the second time that the program has had a clinical hold, which supports our view that the drug has a more challenging safety profile relative to competitors. While the company tried to refocus investors on the upcoming biopsy data from the first 2 high dose patients later this year, we see investors as likely discounting this efficacy update until the company receives clarity on the path forward for the program and shores up its balance sheet.” Auster’s $2 price target implies a downside risk to the stock of 45%. (To watch Auster's track record, click here)All in all, the analyst consensus on SLDB is a Moderate Sell, based on 2 recent sell ratings issued in the past 3 months. Analysts are worried about the company’s path forward when its prime drug candidate has lost the trust of regulators. (See SLDB stock analysis on TipRanks)VBI Vaccines (VBIV)VBI aims at developing a new generation of vaccines, targeting, in the company’s words, ‘unmet needs in infectious disease and immune-oncology.’ The company’s most advanced programs are prophylactic treatments for Hepatitis B, Sci-B-Vac, which has been approved for use in 10 countries, and VLP, which is in Phase 3 testing.In the immune-oncology field, the company has VBI-1901 in testing for patients with recurrent glioblastoma. The company’s Chief Medical Officer said, “We are encouraged by the data we’ve seen to-date in Part A of the study.” VBI-1901 is now in Phase 1 testing.Also in Q3, Edelman and his Life Sciences Fund bought 20 million shares of VBIV, increasing their holding of the company by 78%. The fund now holds 45.9 million shares of the company, with a value of $27 million. This is a significant drop in value from the $45 million recorded at the end of last quarter.Canaccord analyst John Newman sees upside potential in this small company. He writes, “VBIV expects to present top-line data in January from the CONSTANT Phase 3 trial, which we expect to be positive… We expect the data will include key immune repression measures such as geometric mean concentration (GMC) of antibodies across the three independent manufactured lots of Sci-B-Vac.” Newman does, however, lower his price target to $4. Even so, his implied upside is an impressive 577%. (To watch Newman's track record, click here)VBI Vaccines has received 2 recent reviews, both Buys. Shares are selling for minimal 59 cents, but the stock has a $3.50 price target which suggests an-opening 466% upside potential. (See VBI Vaccines stock analysis on TipRanks)

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Solid Biosciences Inc. (SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy, announced that it received written communication from the U.S. Food and Drug Administration (FDA) regarding the clinical hold placed on the Company’s IGNITE DMD Phase I/II clinical trial. The program remains on clinical hold and the Company will continue to work with the FDA to address their requests. In April 2020, the Company submitted a response to the FDA that included changes to the clinical protocol designed to enhance patient safety, as well as information related to improvements to its manufacturing process.

Applied Genetic's (AGTC) gene therapy candidate shows promise in an early-stage study to treat X-linked retinitis pigmentosa, a rare eye disorder.

Solid Biosciences Inc. (SLDB), a life sciences company focused on advancing meaningful therapies for Duchenne muscular dystrophy, announced today the appointment of Ian F. Smith to its board of directors effective April 15, 2020. Mr. Smith is a highly accomplished life sciences executive with more than 20 years of finance and operating leadership experience with public and private biopharmaceutical companies. “We are excited to add Ian to our board,” said Ilan Ganot, Chief Executive Officer, President and Co-Founder of Solid Biosciences.

Solid Biosciences Inc. (SLDB) today announced biomarker data from two patients dosed in the second cohort of IGNITE DMD, its Phase I/II study of SGT-001. SGT-001 is the company’s gene transfer candidate under investigation for Duchenne muscular dystrophy (Duchenne). The data from these patients show SGT-001 microdystrophin expression and associated neuronal nitric oxide synthase (nNOS) function, providing evidence that SGT-001 has the potential to result in therapeutic benefit for patients with Duchenne.