The Daily Biotech Pulse: Pfizer-BioNTech Vaccine Gets Temporary UK Authorization, Ovid's Trial Disappointment, Vanda Snags FDA Nod

Here's a roundup of top developments in the biotech space over the last 24 hours.

Scaling The Peaks

(Biotech Stocks Hitting 52-week Highs Dec. 1)

  • AbbVie Inc ABBV
  • Arcturus Therapeutics Holdings Inc ARCT
  • argenx SE – ADR ARGX
  • Atara Biotherapeutics Inc ATRA
  • Auris Medical Holding Ltd EARS (announced positive efficacy data from lab testing of AM-301 against airborne pathogens and allergens, including SARS-CoV-2)
  • Beam Therapeutics Inc BEAM
  • Bicycle Therapeutics PLC BCYC
  • Bioanalytical Systems, Inc. BASI
  • BioNTech SE – ADR BNTX
  • BioTelemetry Inc BEAT
  • Blueprint Medicines Corp BPMC
  • BridgeBio Pharma Inc BBIO (announced FDA acceptance of NDA for infigratinib for individuals with cancer of the bile ducts)
  • Cardiff Oncology Inc CRDF
  • Cellectis SA CLLS
  • Cerevel Therapeutics Holdings Inc CERE
  • Codiak BioSciences Inc CDAK
  • strong>Crispr Therapeutics AG CRSP
  • Eidos Therapeutics Inc EIDX
  • Fate Therapeutics Inc FATE
  • Foghorn Therapeutics Inc. FHTX
  • Generation Bio Co GBIO
  • Guardant Health Inc GH
  • GW Pharmaceuticals PLC- ADR GWPH
  • Inhibrx Inc INBX
  • Marinus Pharmaceuticals Inc MRNS
  • Mirati Therapeutics Inc MRTX
  • Moderna Inc MRNA (filed conditional marketing authorization application with the European Medicines Agency)
  • Natera Inc NTRA
  • Neuronetics Inc STIM
  • Nurix Therapeutics Inc NRIX
  • Oncorus Inc ONCR
  • Otonomy Inc OTIC
  • Pacific Biosciences of California Inc PACB
  • Pfizer Inc. PFE (European regulators set the date for clearing its conditional marketing application for its investigational coronavirus vaccine)
  • Praxis Precision Medicines Inc PRAX
  • Precigen Inc PGEN
  • Rhythm Pharmaceuticals Inc RYTM
  • Spero Therapeutics Inc SPRO
  • Stoke Therapeutics Inc STOK
  • Veracyte Inc VCYT
  • Vericel Corp VCEL
  • Zai Lab Ltd – ADR ZLAB

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows Dec. 1)

  • Histogen Inc HSTO (reacted to an adverse readout from the Phase 1b/2a clinical trial of HST-001 in male patients with androgenic alopecia)

Stocks In Focus

Mesoblast's Stem Cell Therapy Gets Fast Track Designation For Treating ARDS In COVID-19 Patients

Mesoblast MESO said the FDA has granted fast track designation for remestemcel-L in the treatment of acute respiratory distress syndrome due to COVID-19 infection.

The designation is accorded to facilitate development and expedite review of therapies to treat serious and life-threatening conditions with no or limited treatment options. The FTD would mean a BLA remestemcel-L is eligible for both rolling submission and priority review.

The stock rose 10.12% to $16.65 in after-hours trading.

Vanda's Hetlioz Approved for Sleep Disturbances Associated With Smith-Magenis Syndrome

Vanda Pharmaceuticals Inc. VNDA said the FDA approved Hetlioz capsule and liquid formulations for treating adults and children, respectively, with sleep disturbances associated with Smith-Magenis Syndrome, a rare neurodevelopmental disorder.

The company said Hetlioz capsules for adults will be available immediately, while the liquid formulation for children is expected to be available in the first quarter of 2021.

The stock added 6.89% to $13.19 in after-hours trading.

Ovid Therapeutics' OV101 Flunks a Late-stage Study In Angelman Syndrome

Ovid Therapeutics Inc OVID announced topline results from the Phase 3 NEPTUNE study of OV101 for the treatment of Angelman syndrome, showing the study did not meet the primary endpoint of change in overall score on the Clinical Global Impression-Improvement-Angelman syndrome.

Initial results showed no difference between OV101 and placebo on secondary endpoints that included sleep, communication, motor function, socialization, daily living skills and behavior domains.

The company said it will now focus on the development of its other late-stage asset, OV935, in two rare epilepsies: Dravet and Lennox-Gastaut syndromes.

The stock slumped 49.77% to $3.34 in after-hours trading.

Pfizer/BioNTech Coronavirus Vaccine Issued Conditional Approval In U.K.

Pfizer and BioNTech announced the Medicines & Healthcare Products Regulatory Agency in the U.K. granted a temporary authorization for emergency use of their investigational COVID-19 vaccine BNT162b2.

In premarket trading, Pfizer shares were adding 3.40% to $40.75 and BioNTech was advancing 6.57% to $121.50.

Blueprint Medicines-Roche's Cancer Drug Gets Label Expansion to Treat RET-altered Thyroid Cancers

Blueprint Medicines and partner Roche Holdings AG Basel ADR Common Stock RHHBY said the FDA approved Gavreto for the treatment of patients with RET-altered thyroid cancers. The accelerated approval expands the labeled indications for Gavreto to include adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer, the companies said.

Gavreto was initially approved for RET fusion-positive non-small cell lung cancer in Sept. 2020.

Separately, Roche said its Elecsys anti-SARS-CoV-2 S antibody test has received emergency use authorization from the FDA. The Elecsys blood test can be used to measure the level of antibodies in people who have been exposed to the SARS-CoV-2 virus. This follows Elecsys' launch for markets accepting the CE Mark announced Sept. 18.

Axsome Announces Positive Mid-stage Results For Depression Drug

Axsome Therapeutics Inc AXSM announced positive results from the open-label Phase 2 COMET-TRD trial of AXS-05 in patients with treatment resistant depression. Patients treated with AXS-05 experienced rapid, substantial, and durable improvements in depressive symptoms and functional impairment that was sustained with long-term treatment, the company said.

The company said results of the Phase 2 COMET-SI trial of major depressive disorder patients with suicidal ideation are still expected before year end.


Reata Pharmaceuticals Inc RETA said it has priced a public offering of 2 million shares at a price to the public of $140.85 per share, for raising gross proceeds of $281.7 million. The offering is expected to close on or about Dec. 4, subject to customary closing conditions.

In after-hours trading, the stock was down 9.12% to $138.

Beigene Ltd BGNE priced its underwritten public offering of 1.512 million ADSs, each representing 13 of its ordinary shares, by fund partnerships affiliated with Baker Bros. Advisors at a public offering price of $225 per ADS.

BeiGene clarified that it will not receive any of the proceeds from the sale of the shares but will bear certain expenses incident to this offering. The offering is expected to close on Dec. 4, subject to customary closing conditions.

The stock pulled back 3.24% to $8.47 in after-hours trading.

Burning Rock Biotech Ltd BNR said it filed a registration statement on Form F-1 with the SEC, relating to a public secondary offering by certain selling shareholders of 1.838 million ADSs, each representing one Class A ordinary share of the Company.
The company said it will not receive any proceeds from the sale of the ADSs by the selling shareholders.

The stock was down 2.79% to $28.35.

Allena Pharmaceuticals Inc ALNA said it has entered into an underwriting agreement with H.C. Wainwright & Co., under which the latter has agreed to purchase on a firm commitment basis 8 million shares of its common stock at a price to the public of $1.25 per share, less underwriting discounts and commissions. The closing of the offering is expected to occur on or about Dec. 4, subject to satisfaction of customary closing conditions.

The stock was seen receding 5.26% to $1.26.

Syneos Health Inc SYNH said it commenced an underwritten secondary offering by affiliates of Thomas Lee Partners and Advent International Corporation of 6 million shares of its common stock. The company said it is not selling any shares and will not receive any proceeds from the sale of the shares.

The stock was slipping 5.28% to $62.25.

Related Link: Attention Biotech Investors: Mark Your Calendar For December PDUFA Dates

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