The Daily Biotech Pulse: Axovant Surges On Gene Therapy Study Results, FDA Approves Merck's Ebola Vaccine, Abeona Announces Offering

The following is a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech stocks that hit 52-week highs Dec. 19.)

• Acceleron Pharma Inc XLRN
• Ardelyx Inc ARDX
• AstraZeneca plc AZN
• Axsome Therapeutics Inc AXSM (priced its 2-million-share common stock offering at $87 per share)
• Clearside Biomedical Inc CLSD
• Cortexyme Inc CRTX
• Cue Biopharma Inc CUE
• Exagen Inc XGN
• Globus Medical Inc GMED
• Horizon Therapeutics PLC HZNP
• HEALTH SCIENCES/SH IMVT (The acquisition vehicle announced merger with Immuvant, a clinical-stage biopharma company focusing on autoimmune disorders)
• IVERIC bio Inc ISEE
• Johnson & Johnson JNJ (Barclays upgraded shares to Overweight)
• The Medicines Company MDCO
• Merck & Co., Inc. MRK
• Mirum Pharmaceuticals Inc MIRM
• Momenta Pharmaceuticals, Inc. MNTA
• Monopar Therapeutics Inc. MNPR (Went public Thursday)
• Myokardia Inc MYOK
• NeoGenomics, Inc. NEO
• Novan Inc NOVN
• Oramed Pharmaceuticals, Inc. ORMP
• Principia Biopharma Inc PRNB
• Quest Diagnostics Inc DGX
• Recro Pharma Inc REPH
• Sanofi SA SNY
• SpringWorks Therapeutics Inc SWTX

Down In The Dumps

(Biotech stocks that hit 52-week lows Dec. 19.)

• Aethlon Medical, Inc. AEMD
• Anixa Biosciences Inc ANIX
• Aridis Pharmaceuticals Inc ARDS
• China SXT Pharmaceuticals Inc SXTC
• Correvio Pharma Corp CORV
• KemPharm Inc KMPH
• Merrimack Pharmaceuticals Inc MACK
• Miragen Therapeutics Inc MGEN
• Nemaura Medical Inc NMRD
• Theratechnologies Inc THTX (pre-announced fourth-quarter revenue and issued 2020 guidance.)
• Zosano Pharma Corp ZSAN

Stocks In Focus

Axovant Says Gene Therapy Found Effective In Genetic Peadiatric Disorder

Axovant Gene Therapies Ltd AXGT released preliminary findings from an expanded access treatment in which one child with GM1 gangliosidosis was administered its investigational AXO-AAV-GM1 gene therapy, which showed safety, tolerability and clinical improvement.

"The subject was observed to have clinically significant improvements based on neurological exam, the Vineland-3 scale, Clinical Global Impression (CGI) assessments, and nutritional status," Cynthia Tifft, an expert in ganglioside storage disorders, said in a statement on data collected at a six-month follow-up.

The company said it hopes to complete enrollment in Part A of the ongoing registrational study in early 2020.

The stock rallied 13.63% to $5.92 in after-hours trading.

FDA Approves Merck's Ebola Vaccine

The FDA announced that it has approved Merck's Ervebo — a vaccine for the prevention of Ebola virus disease in individuals 18 years of age and older. This is the first FDA-approved vaccine for the Ebola virus.

Ervebo is administered as a single-dose injection and is a live, attenuated vaccine that has been genetically engineered to contain a protein from the Zaire ebolavirus.

See also: 8 Biotech Stocks Morgan Stanley Recommends For 2020

Amgen, Allergan File Regulatory Application For Biosimilar to Roche's Blood Cancer Drug

Amgen, Inc. AMGN and Allergan plc AGN said they have submitted a BLA to the FDA for ABP 798, a biosimilar to Roche Holdings AG Basel ADR's RHHBY's blood cancer drug Rituxan.

"The U.S. filing for ABP 798 marks an important milestone for Amgen, as it affirms our commitment to providing high quality biosimilars that offer more life-altering biological treatment options and contribute to the sustainability of healthcare systems," said David Reese, EVP of R&D at Amgen.

Gilead Submits NDA For Filgotinib In Rheumatoid Arthritis

Gilead Sciences, Inc. GILD said it has submitted an NDA to the FDA for filgotinib, an investigational oral selective JAK1 inhibitor for the treatment of moderate-to-severe rheumatoid arthritis.

The company said it has submitted a priority voucher that would help shorten the review period.

Gilead is co-developing the drug with GALAPAGOS NV/S ADR GLPG.

Chiasma Added to Nasdaq Biotechnology Index

Chiasma Inc CHMA said it has been selected for addition to the Nasdaq Biotechnology Index, effective prior to the market open Monday, Dec. 23.

Offerings

Abeona Therapeutics Inc ABEO said it intends to offer shares of its common stock and pre-funded warrants instead of common stock in an underwritten public offering. All the shares earmarked for the offering are being sold by the company.

Great Point Partners, an existing shareholder, has expressed willingness to buy up to $33 million in the offering, Abeona said. 

The stock plunged 26% to $3.67 in after-hours trading.

Related Link: The Week Ahead In Biotech: Avadel, Epizyme In Focus In A Quiet Week