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The Daily Biotech Pulse: Translate Bio's Cystic Fibrosis Study Disappoints, Adverum Nominates Directors, Gain Therapeutics To Debut On Nasdaq

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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 March 17)

  • AngioDynamics, Inc. (NASDAQ: ANGO)

  • Anixa Biosciences, Inc. (NASDAQ: ANIX)

  • BioAtla, Inc. (NASDAQ: BCAB)

  • Harvard Bioscience, Inc. (NASDAQ: HBIO)

  • IDEAYA Biosciences, Inc. (NASDAQ: IDYA)

  • Immatics N.V. (NASDAQ: IMTX) (announced positive Phase 1 readout for a trio of candidates in solid tumors)

  • NGM Biopharmaceuticals, Inc. (NASDAQ: NGM)

  • NuVasive, Inc. (NASDAQ: NUVA)

  • Omnicell, Inc. (NASDAQ: OMCL)

  • Oncternal Therapeutics, Inc. (NASDAQ: ONCT)

  • Organogenesis Holdings Inc. (NASDAQ: ORGO)

  • PLx Pharma Inc. (NASDAQ: PLXP)

  • Sierra Oncology, Inc. (NASDAQ: SRRA)

  • Silverback Therapeutics, Inc. (NASDAQ: SBTX)

  • STRATA Skin Sciences, Inc. (NASDAQ: SSKN)

  • Urovant Sciences Ltd. (NASDAQ: UROV)

Down In The Dumps

(Biotech Stocks Hitting 52-week Lows March 17)

  • Foghorn Therapeutics Inc. (NASDAQ: FHTX)

  • Galapagos NV (NASDAQ: GLPG)

  • Galecto, Inc. (NASDAQ: GLTO)

Stocks In Focus Translate Bio mRNA Product Candidate For Cystic Fibrosis Fails To Improve Lung Function In Phase 1/2 Study

Translate Bio (NASDAQ: TBIO) announced a second interim analysis from the Phase 1/2 study of MRT5005 in patients with cystic fibrosis, showing that repeat dosing was generally safe and well tolerated, with no serious adverse events.

The company, however, said there was no pattern of increases in the percent of predicted forced expiratory volume in 1 second, a measure of lung function.

MRT5005 is the first clinical-stage mRNA product candidate designed to address the underlying cause of cystic fibrosis by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator protein to the lung epithelial cells through nebulization.

The stock was down 25.99% premarket at $19.02.

Adverum Responds To Activist Investor, Names Slate Of Directors

Adverum Biotechnologies, Inc. (NASDAQ: ADVM) announced the appointments of Leslie Trigg and Katey Owen to its board effective immediately, and the retirement of Eric Dobmeier and David Goel from the board after long-standing tenures.

In a separate release, the company said it does not believe giving Sonic Fund II — which has a 5% stake in the company — control of the board is in the best interest of stockholders.

The statement is in response to Sonic's suggestion to nominate five director candidates to stand for election at Adverum's 2021 annual meeting.

The company announced a slate of directors that are expected to be nominated by its board.

The stock was up 8.74% premarket at $11.70.

Vericel To Join S&P SmallCap 600 Index

Vericel Corp. (NASD: VCEL) will replace QEP Resources Inc. (NYSE: QEP) in the S&P SmallCap 600 effective prior to the opening of trading on Monday, March 22.

This follows S&P 500 constituent Diamondback Energy Inc. (NASD: FANG) acquiring QEP Resources in a transaction that closed Wednesday.

Vericel was up 11.67% premarket at $52.24.

GlaxoSmithKline Out-Licenses Two Pre-Phase 2 Programs To Boston Pharma

GlaxoSmithKline PLC (NYSE: GSK) and Boston Pharmaceuticals announced a three-year out-license and option agreement, under which Boston Pharma will become a preferred GSK partner for select pre-Phase 2 programs. This new agreement builds on the 2018 agreement between both companies that provided for Boston Pharma acquiring five GSK programs.

Initially, GlaxoSmithKline will out-license and option two programs: a monoclonal antibody against tumors and a neurological disorder drug.

Boston Pharma will assume responsibility for further development of these programs through proof-of-concept. GlaxoSmithKline will have the option to reacquire each program under pre-agreed terms for subsequent development and worldwide commercialization.

Bristol-Myers Squibb shares were trading down slightly at $62.13 premarket Thursday.

Related Link: The Week Ahead In Biotech (March 14-20): Update On Fibrogen-AstraZeneca's Anemia Drug, Earnings and Presentations

Boston Scientific Gets FDA Nod For Radioembolization Tech to Treat Liver Cancer

Boston Scientific Corporation (NYSE: BSX) announced FDA approval of the TheraSphere Y-90 Glass Microspheres, developed for the treatment of patients with hepatocellular carcinoma.

The approval expands access to this life-prolonging therapy for a greater number of patients, the company said.

The therapy has been utilized under a humanitarian device exemption — an FDA classification that required institutional review board approval and limited the number of patients treated with the therapy per year.

View more earnings on IBB

TheraSphere is now the only radioembolization technology indicated for the treatment of unresectable HCC in the U.S.

The stock was up 0.77% to $39.18 in premarket trading Thursday.

Gilead, Novo Nordisk Expand NASH Drug Collaboration

Gilead Sciences, Inc. (NASDAQ: GILD) and Novo Nordisk A/S (NYSE: NVO) announced an expanded clinical collaboration in non-alcoholic steatohepatitis, or NASH.

The companies will conduct a Phase 2b study to investigate the safety and efficacy of Novo Nordisk's diabetes drug semaglutide, and a fixed-dose combination of Gilead's cilofexor and firsocostat, alone and in combination in people with compensated cirrhosis due to NASH.

The four-arm study in approximately 440 patients will evaluate the treatments' impact on liver fibrosis improvement and NASH resolution and will begin recruitment in the second half of 2021.

In premarket trading Thursday, Gilead shares were down 1.15% to $63.68 and Novo Nordisk was slipping 0.81% to $71.20.

Merck Files With SEC To Spin Off Women's Health, Biosimilar and Older Brands Into Separate Company

Merck (NYSE: MRK) said it has filed a Form 10 registration statement with the U.S. Securities and Exchange Commission in connection with the intended spinoff of its women's health, biosimilars and established brands businesses into a standalone, publicly traded company, Organon.

The stock edged up 0.26% to $77.50 in after-hours trading.


ThermoGenesis Holdings, Inc. (NASDAQ: THMO) reported a decline in revenue from $13 million in 2019 to $9.7 million in 2020. The net loss, however, narrowed from $3.36 per share to $2.60 per share.

The stock was down 4.95% premarket at $2.88.


PhaseBio Pharmaceuticals, Inc. (NASDAQ: PHAS) priced its underwritten public offering of 16 million shares of its common stock at a public offering price of $3.50 per share. The gross proceeds to PhaseBio from the offering are expected to be approximately $56 million. The offering is expected to close on or about March 22.

The stock was down 14.15% premarket at $3.70.

ContraFect Corporation (NASDAQ: CFRX) priced its underwritten public offering of 10 million shares of its common stock at a public offering price of $5 per share. All shares in the offering are to be sold by ContraFect.

The stock was down 12.38% premarket at $5.45.

Solid Biosciences Inc. (NASDAQ: SLDB) said it has commenced an underwritten public offering of $85 million of shares of its common stock. All of the shares are being offered by the company.

The stock was down 4.66% premarket at $6.34.

On The Radar Clinical Readouts Muscular Dystrophy Association Conference Presentations

Sarepta Therapeutics, Inc. (NASDAQ: SRPT): Phase 1 data for SRP-9001 in Duchenne muscular dystrophy, and new long-term functional data from Phase 1/2 study of gene therapy candidate SRP-9003 that is being developed for limb-girdle muscular dystrophy type 2E.

Solid Biosciences Inc.: one-year efficacy and safety data from Phase 1/2 ascending dose study of single intravenous infusion of SGT-001 microdystrophin gene therapy for DMD (4 p.m.); The shares came under pressure earlier this week despite the company releasing positive data for the same program.

Amicus Therapeutics, Inc. (NASDAQ: FOLD): Phase 3 data for AT-GAA in late-onset Pompe disease.


  • Legend Biotech Corporation (NASDAQ: LEGN) (before the market open)

  • RedHill Biopharma Ltd. (NASDAQ: RDHL) (before the market open)

  • Ocugen, Inc. (NASDAQ: OCGN) (before the market open)

  • ADC Therapeutics SA (NYSE: ADCT) (before the market open)

  • SQZ Biotechnologies Company (NASDAQ: SQZ) (before the market open)

  • HOOKIPA Pharma Inc. (NASAQ: HOOK) (before the market open)

  • Applied Therapeutics, Inc. (NASDAQ: APLT) (before the market open)

  • Precision BioSciences, Inc. (NASDAQ: DTIL) (before the market open)

  • PDS Biotechnology Corporation (NASDAQ: PDSB) (before the market open)

  • Metacrine, Inc. (NASDAQ: MTCR) (before the market open)

  • Entera Bio Ltd. (NASDAQ: ENTX) (before the market open)

  • UroGen Pharma Ltd. (NASDAQ: URGN) (before the market open)

  • Galmed Pharmaceuticals Ltd. (NASDAQ: GLMD) (before the market open)

  • Acutus Medical, Inc. (AFIB) (NASDAQ: after the close)

  • Alpine Immune Sciences, Inc. (NASDAQ: ALPN) (after the close)

  • Viveve Medical, Inc. (NASDQ: VIVE) (after the close)

  • Salarius Pharmaceuticals, Inc. (NASDAQ: SLRX) (after the close)


Bethesda, Maryland-based Gain Therapeutics, Inc. priced its 3.64-million share initial public offering at a price of $11 per share, at the midpoint of the estimated price range of $10-$12.

The shares of clinical-stage biopharma, which is focused on developing therapeutics for lysosomal storage disorders, will begin trading on the Nasdaq under the ticker symbol "GANX."

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

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