The Daily Biotech Pulse: Vertex Pulls Plug On Protein Deficiency Drug, FDA Nod For Regeneron's Ebola Treatment, Can Fite Issues Psoriasis Readout

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 Oct. 14)

  • 10X Genomics Inc TXG
  • Aileron Therapeutics Inc ALRN
  • Amicus Therapeutics, Inc. FOLD
  • Bio-Rad Laboratories, Inc. Class B Common Stock BIO
  • Blueprint Medicines Corp BPMC
  • Calliditas Therapeutics Adr Rep 2 Ord Shs CALT
  • Cellectis SA CLLS
  • Crispr Therapeutics AG CRSP
  • Denali Therapeutics Inc DNLI
  • Immunomedics, Inc IMMU - The Hart-Scott-Rodino waiting period for its impending acquisition by Gilead Sciences, Inc. GILD expired, meeting one of the conditions necessary for the consummation of the transaction
  • Insulet Corporation PODD
  • Kronos Bio Inc KRON (went public Friday)
  • Kura Oncology Inc KURA
  • Kymera Therapeutics Inc KYMR
  • Madrigal Pharmaceuticals Inc MDGL
  • Mirati Therapeutics Inc MRTX
  • NeoGenomics, Inc NEO
  • Nevro Corp NVRO
  • Novocure Ltd NVCR
  • Ocular Therapeutix Inc OCUL
  • Oncorus Inc ONCR
  • Pacific Biosciences of California Inc PACB
  • PRA Health Sciences Inc PRAH
  • Replimune Group Inc REPL (abstract comprising data for its lead candidate RP1 in advanced solid tumors to be presented at the November Society for Immunotherapy of Cancer annual meeting made available online)
  • Seres Therapeutics Inc MCRB
  • SpringWorks Therapeutics Inc SWTX
  • Spruce Biosciences Inc SPRB
  • Surface Oncology Inc SURF
  • Tcr2 Therapeutics Inc TCRR
  • Twist Bioscience Corp TWST
  • Ultragenyx Pharmaceutical Inc RARE
  • Veracyte Inc VCYT

Down In The Dumps

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

  • Aziyo Biologics Inc AZYO (went public Thursday)
  • Cumberland Pharmaceuticals, Inc. CPIX
  • Dyne Therapeutics Inc DYN
  • Psychemedics Corp. PMD
  • Polypid Ltd PYPD

Stocks In Focus

Vertex Stalls VX-814 Study In Inherited Protein Deficiency

Vertex Pharmaceuticals Incorporated VRTX said it has decided to stop dosing in the Phase 2 proof-of-concept trial that is evaluating VX-814 in alpha-1 antitrypsin deficiency, or AATD. The decision is based on the safety and pharmacokinetic observed to date in the trial, the company said.

The company also opted to discontinue development of VX-814.

"Analysis of the PK data from the study indicated that exposures achieved were low," Vertex said.

Based on these data, the company said it concluded that it would not be feasible to safely reach targeted exposure levels, and thus meaningful increases in AAT levels, with VX-814, and the study was stopped.

The stock was down 9.75% at $245 premarket Thursday.

FDA OKs Regeneron's Ebola Antibody Cocktail

Regeneron Pharmaceuticals Inc REGN said the FDA approved Inmazeb — an antibody cocktail comprising atoltivimab, maftivimab and odesivimab-ebgn — for the treatment of infection caused by Zaire ebolavirus in adult and pediatric patients, including newborns of mothers who have tested positive for the infection.

The stock was down 0.47% premarket at $598.

Related Link: The Week Ahead In Biotech: J&J Earnings Kickstarts Pharma Earnings, More IPOs In The Pipeline

AMAG Requests Hearing On FDA's Proposal To Withdraw Approval Of Pre-Term Birth Drug

AMAG Pharmaceuticals, Inc. AMAG said it has formally requested a public hearing in response to the FDA's proposal to withdraw its approval of Makena, along with its equivalent generics, to reduce preterm birth.

The hearing request will be followed by a submission of supporting documentation to the FDA, Amag said. That submission will provide further detail on the company's reasoning for a hearing, recognizing clinicians' decade-long use of this treatment and the public health implications of withdrawing it, Amag said.

The stock was trading 1.71% higher premarket at $13.68.


SI-Bone Inc SIBN priced its underwritten public offering of 3.19 million shares of its common stock at $22 each. Of the shares being offered, 3 million are being offered by SI-BONE and 190,053 shares by a selling stockholder.

The company expects to raise gross proceeds of about $66 million from the 3 million shares it intends to sell.

The offering is scheduled to close Oct. 19, subject to the satisfaction of customary closing conditions.

The stock was down 5.05% premarket at $22.20.

On The Radar

Clinical Readouts

Can Fite Biopharma ADR Representing 30 Ord Shs CANF is scheduled to host a conference call with investors at 4:15 p.m. ET to provide an update on its Phase 3 study evaluating its lead candidate piclidenoson in psoriasis, and the positive interim data analysis and recommendations from the study's Independent Data Monitoring Committee.


  • Brainstorm Cell Therapeutics Inc BCLI (before the market open)
  • Intuitive Surgical, Inc. ISRG (after the close)

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

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