Verve Not Ready To Pull Plug On Anti-PCSK9 Gene Editing Candidate

But Firm Shifts Focus To Back-Up Candidate

The company told Scrip that the sixth patient with elevated liver enzymes and low platelets recovered after hospital observation and oral steroids, and it may still continue VERVE-101’s development.

• Source: Shutterstock

Verve Therapeutics, Inc. has paused enrollment in its Phase Ib Heart-1 trial of gene-edited cholesterol medicine VERVE-101 after a patient experienced liver enzyme elevations and low platelet counts. But while the company has decided to prioritize its development of a different asset with the same target, VERVE-102, it said VERVE-101 still might have a shot.

The biotech company said on 2 April that it had voluntarily paused enrollment in the study in heterozygous familial hypercholesterolemia (HeFH) after a sixth patient on the 0.45mg/kg dose experienced...

Key Takeaways
  • Verve paused enrollment in its Phase Ib study of gene-editing candidate VERVE-101 after a sixth patient experienced grade 3 ALT elevations and thrombocytopenia.

  • ...

Read the full article – start your free trial today!

Join thousands of industry professionals who rely on Scrip for daily insights

  • Start your 7-day free trial
  • Explore trusted news, analysis, and insights
  • Access comprehensive global coverage
  • Enjoy instant access – no credit card required

More from Gene Therapies

Bluebird, Private Equity Firms Look To Sweeten Buyout Deal

 

Shareholders are being offered a flat fee of $5 per share as an alternative to $3 plus a contingent value right, which could be a more attractive option if they want cash up front.

Which Firms Are Most Exposed To Prasad’s Possible Accelerated Approvals Clampdown?

 

Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.

Abeona Sets $3.1m Price For Rare Skin Disorder Gene Therapy

 
• By 

Abeona plans to offer an outcomes-based payment model for Zevaskyn, which likely will see complementary use with Krystal’s Vyjuvek in recessive dystrophic epidermolysis bullosa patients.

Verve Moves Toward Lilly Opt-In For PCSK9 Program With Phase Ib Results

 

The company posted positive initial results from the Heart-2 trial in HeFH and CAD, with strong LDL cholesterol lowering and a clean safety profile.

More from Advanced Therapies

Which Firms Are Most Exposed To Prasad’s Possible Accelerated Approvals Clampdown?

 

Vaccine and accelerated approvals are expected to face greater scrutiny under new CBER leader Vinay Prasad, but he has promised “no light switch change” to policy, giving some reassurance to companies whose share prices have been battered.

Atara Does An About Face As FDA Lifts Ebvallo Hold

 

The biotech said it will have a type A meeting with the agency to resubmit for the cell therapy’s approval in EBV+ PTLD.

Zelluna Believes Its TCR Tech Can Unlock Natural Killer Cell Potential

 

While CAR-NKs have pivoted towards autoimmune disease, the Norwegian biotech believes its unique platform can deliver in oncology