The Daily Biotech Pulse: Marinus Epilepsy Study Meets Goal, Novavax Reaches Vaccine Manufacturing Deal, Outset Medical's IPO

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs Sept. 14)

10X Genomics Inc TXG
Applied Molecular Transport Inc. AMTI
Avenue Therapeutics Inc ATXI
Avidity Biosciences Inc RNA
BioLife Solutions Inc BLFS
Denali Therapeutics Inc DNLI
Guardant Health Inc GH
Immunomedics, Inc. IMMU - announced a deal to be bought by Gilead Sciences, Inc. GILD for $21 billion
Inspire Medical Systems Inc INSP
Kintara Therapeutics Inc KTRA
Kura Oncology Inc KURA
Nurix Therapeutics Inc NRIX
Pacific Biosciences of California Inc PACB (announced the appointment of Susan Kim as CFO, effective Sept. 28)
Shockwave Medical Inc SWAV
SI-Bone Inc SIBN
Silk Road Medical Inc SILK
Stoke Therapeutics Inc STOK
Trillium Therapeutics Inc TRIL
Turning Point Therapeutics Inc TPTX

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Sept. 14)

Acorda Therapeutics Inc ACOR
AEterna Zentaris Inc. AEZS
BELLUS Health Inc BLU
PainReform Ltd PRFX
Predictive Oncology Inc POAI
Sunesis Pharmaceuticals, Inc. SNSS

Stocks In Focus

Marinus Reports Positive Late-Stage Readout In Epilepsy

Marinus Pharmaceuticals Inc MRNS announced positive top-line results from its registrational Phase 3 clinical trial, dubbed Marigold, evaluating the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder, a rare, genetic epilepsy with refractory seizures.

Based on these results, Marinus said it plans to submit an NDA for ganaxolone to the FDA in mid-2021 and a marketing authorization application to the European Medicines Agency by the end of the third quarter of 2021.

Separately, the company said it has entered into a five-year development contract with the BARDA for the development of intravenous ganaxolone for the treatment of refractory status epilepticus, a life-threatening condition in which a significant number of patients do not respond to first- and second-line anticonvulsant drugs.

The BARDA will provide base period funding of $21 million, funding for preclinical studies in nerve agent exposure animal models and up to $30 million in additional funding, contingent on favorable clinical and preclinical outcomes.

In premarket trading, Marinus shares were rallying 54.98% to $3.27.

PPD Lifts Q3 Guidance, Announces Secondary Offering

Contract research organization PPD Inc PPD upwardly revised its third-quarter revenue and EBITDA guidance, citing the exceptional circumstances surrounding COVID-19 and better-than-anticipated key operating metrics and financial results in July and August.

The company raised its third-quarter revenue guidance from a range of $1.065 billion to $1.085 billion to a range of $1.157 billion to $1.188 billion. Analysts expect revenue of $1.08 billion. The company lifted its adjusted EBITDA guidance from $211 million-$215 million to $223 million-$227 million.

Separately, the company said certain stockholders of the company, including stockholders affiliated with Hellman & Friedman and The Carlyle Group Inc., have commenced an underwritten public offering of 38 million shares of its common stock. The company said it is not selling any shares and will not receive any proceeds from the offering.

The stock declined 4.58% to $32.26 in after-hours trading.

Plus Therapeutics Lead Drug Fast Tracked In Glioblastoma

Plus Therapeutics Inc PSTV said the FDA has granted fast track designation for its lead investigational drug, Rhenium NanoLiposomes, for the treatment of patients with recurrent glioblastoma.

In premarket trading Tuesday, the stock was up 61.48% to $3.94.

Novavax, Serum Institute Boost Vaccine Manufacturing Capacity

Novavax, Inc. NVAX announced an amendment to its existing agreement with Serum Institute of India Private Limited under which the latter will also manufacture the antigen component of NVX CoV2373, Novavax's COVID-19 vaccine candidate. This will increase Novavax's manufacturing capacity of NVX-CoV2373 to over 2 billion doses annually when all planned capacity has been brought online by mid-2021, the company said.

In premarket trading Tuesday, the stock was rising 6.33% to $112.50.

GenMark Molecular Test Gets CE Mark

GenMark Diagnostics, Inc GNMK said it has achieved a CE mark under the European In‐Vitro Diagnostic Devices Directive for its ePlex Respiratory Pathogen Panel 2.

The molecular test provides results in less than two hours for more than 20 viruses and bacteria that cause common and often serious respiratory infections, including COVID-19, influenza A and B, respiratory syncytial virus and rhinovirus, the company said.

GenMark has launched the test in the U.S. and is awaiting emergency use authorization from the FDA.

The stock was trading 2.31% higher to $12.83 premarket Tuesday.

Miragen CEO Departs, Company Announces Strategic Review

Miragen Therapeutics Inc MGEN announced the appointment of Lee Rauch, currently serving as chief operating officer, as CEO following the resignation of William Marshall, who has also resigned from the board.

The company also said following a comprehensive review of its R&D pipeline that it has decided to prioritize its resources toward advancing the development of its lead compound MRG-229 in idiopathic pulmonary fibrosis.

The company's board has elected to conduct a comprehensive review of available strategic alternatives with a focus on maximizing stockholder value, and has appointed Ladenburg Thalmann & Co to assist with the review.

The stock was down 5.32% premarket at 89 cents.

Matinas Announces Positive End-Of-Phase 2 Meeting With FDA

Matinas BioPharma Holdings Inc MTNB said the official minutes of its end-of-Phase 2 meeting with the FDA concerning the development and registration pathway for MAT9001, its prescription omega-3 therapy, confirmed that the FDA and Matinas are aligned on key next steps for MAT9001's Phase 3 program and the registration pathway for an initial indication to treat severe hypertriglyceridemia.

The company said it remains on track to initiate its Phase 3 program in the first half of 2021.

The stock was adding 2.81% to 74 cents in premarket trading Tuesday.

Novus Acquires Anelixis, Privately Places $108M In Debt

Novus Therapeutics Inc NVUS announced the acquisition of Anelixis Therapeutics, Inc., a privately held clinical-stage biotechnology company developing a next generation anti-CD40 Ligand antibody as a potential treatment for organ and cellular transplantation, autoimmune diseases and neurodegenerative diseases.

Concurrent with the acquisition, Novus also announced a private placement of non-voting convertible preferred stock to institutional investors in order to raise about $108 million. Additionally, the company said its board previously appointed David-Alexandre Gros as CEO and director.

The stock was up 111.08% at 80 cents premarket Tuesday.

Earnings

Champions Oncology Inc's CSBR fiscal-year first-quarter revenue rose 42% year-over-year to $9.5 million. The company reversed to a profit of 2 cents per share on non-GAAP basis from a loss of 5 cents per share.

The stock climbed 13.72% to $8.62 premarket Tuesday.

Offerings

89bio Inc ETNB said it has commenced an underwritten public offering of 3 million shares of its common stock, including 2.7 million shares to be offered by the company and remaining 300,000 shares by certain company stockholders.

The company said it intends to use the net proceeds, along with its existing cash and cash equivalents, to support its BIO89-100 programs in nonalcoholic steatohepatitis and severe hypertriglyceridemia; for the manufacture and scale up of BIO89-100; and for other general corporate purposes.

On The Radar

PDUFA Dates

The FDA is set to rule on Bausch Health Companies Inc's BHC NDA for EM-100, a preservative-free ophthalmic ketotifen solution that is being evaluated for allergic conjunctivitis. Bausch in-licensed EM-100 from Eton Pharmaceuticals Inc ETON in February 2019.

Clinical Readouts

Prothena Corporation PLC PRTA is scheduled to present at the International Parkinson and Movement Disorder Society Virtual Congress 2020 results from Part 1 of the Phase 2 PASADENA study of prasinezumab in early Parkinson's disease.

IPOs

San Jose, California-based Outset Medical, Inc. priced its upsized initial public offering of 8.95 million shares at $27, above the estimated price range of $22-$24, for raising gross proceeds of $241.7 million.

The medical technology company, which provides dialysis systems, said its shares will be listed on the Nasdaq under the ticker symbol "OM."

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