The Daily Biotech Pulse: CDC AdComm Backs Novavax COVID-19 Vaccine, European Approval For PTC Therapeutics' Gene Therapy, Merck's Keytruda Flunks In Head, Neck Cancer Study

Zinger Key Points
  • FDA said that first vaccine lot from Novavax has met all release specifications and is acceptable for use.
  • PTC Therapeutics' Upstaza is the first marketed gene therapy directly infused into the brain.

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

Stocks In Focus

CDC Advisory Committee Recommends Novavax's COVID-19 Vaccine For Adults

The U.S. Centers for Disease Control and Prevention's advisory committee recommended using Novavax Inc's NVAX COVID-19 vaccine as a two-dose primary series for individuals aged 18 and above.

CDC director Rochelle Walensky said the decision "expanded the options available" to U.S. adults for vaccinations.

Soleno Therapeutics Provides Regulatory Update On Prader-Willi Syndrome Candidate

Soleno Therapeutics Inc SLNO provided an update following recent communications with the FDA regarding the development of once-daily DCCR (diazoxide choline) extended-release tablets for Prader-Willi syndrome.

The FDA acknowledged that data from a proposed randomized withdrawal phase of Study C602 would have the potential to address its concerns regarding the adequacy of the overall efficacy data supportive of a marketing application submission for DCCR. 

Shares are up 31.5% at 25 cents during the premarket session.

VBL Therapeutics Plummet On Disappointing Ovarian Cancer Trial

Vascular Biogenics Ltd or VBL Therapeutics' VBLT Phase 3 OVAL trial of VB-111) in platinum-resistant ovarian cancer did not meet the primary endpoints of achieving a statistically significant improvement in progression-free survival or overall survival.

The company will discontinue the OVAL trial and review the data from ongoing Phase 2 trials in metastatic colorectal cancer and recurrent glioblastoma multiforme.

As VBL evaluates the next steps with the ofra-vec program, it continues to move the VB-601 program towards a first-in-human clinical trial, expected to begin in the fourth quarter of 2022. 

Shares are down 78.5% at 44 cents during the premarket session.

FDA Clears Sorrento's STI-1558 Phase 1 Study To Treat COVID-19 Infection

The FDA has signed off Sorrento Therapeutics Inc's SRNE Phase 1 study of its oral main viral protease (Mpro) inhibitor, STI-1558 in patients with impaired renal and hepatic function.

The Phase 1 study will examine the PK in patients with moderate renal and hepatic impairment. 

Using a cross-over design, two doses of STI-1558 (300 mg and 600 mg) will be studied in 12 subjects across three cohorts. 

Belite Bio Seeks FDA Nod For Late-Stage Genetic Eye Disease Study

Belite Bio Inc BLTE submitted an Investigational New Drug Application to the FDA to support Phase 3 development of LBS-008 for Stargardt Disease (STGD1), a rare genetic eye disease.

Belite Bio is currently conducting a two-year Phase 2 trial and a two-year Phase 3 trial of LBS-008 in adolescent STGD1 subjects.

Inozyme Pharma Post Encouraging Early Data From Genetic Disorder Trial

Inozyme Pharma Inc INZY has announced preliminary biomarker, safety, and pharmacokinetic (PK) data from the first three subjects in the Phase 1 portion of its ongoing Phase 1/2 trial of INZ-701 in ABCC6 Deficiency,

At the 0.2 mg/kg dose level of INZ-701, all three subjects showed rapid and significant increases in PPi levels. In preclinical models, PPi was a key predictive biomarker of therapeutic benefit in ABCC6 Deficiency.

Also Read: EXCLUSIVE: Adial Pharma's AD04 Effective In Heavy Drinker Alcohol Use Disorder Patients.

Merck's Keytruda Therapy Fails In Late-Stage Head, Neck Cancer Study

Merck & Co Inc MRK announced that the Phase 3 KEYNOTE-412 trial of the Keytruda (pembrolizumab) regimen did not meet its primary endpoint of event-free survival (EFS) for unresected locally advanced head and neck squamous cell carcinoma.

The regimen included Keytruda, with concurrent chemoradiation therapy (CRT) followed by Keytruda as maintenance therapy.

At the study's final analysis, there was an improvement in EFS for patients who received the regimen compared to placebo plus CRT.

PTC Therapeutics' Gene Therapy Scores European Approval For Genetic Disorder

The European Commission has approved PTC Therapeutics Inc's PTCT Upstaza (eladocagene exuparvovec) for aromatic L-amino acid decarboxylase (AADC) deficiency.

It is approved for patients 18 months and older.

AADC deficiency is an inherited disorder that affects the way signals are passed between certain cells in the nervous system.


Revolution Medicines Inc RVMD priced its underwritten public offering of 11.5 million shares at $20 per share, with gross proceeds of $230 million.

On The Radar


Biogen Inc BIIB: Before the market open.

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