Gaurav Shah, Rocket Pharmaceuticals CEO

FDA lifts clin­i­cal hold on Rock­et's gene ther­a­py for heart fail­ure af­ter 3-month de­lay

New Jer­sey’s Rock­et Phar­ma­ceu­ti­cals hit a ma­jor road­block for its AAV-de­liv­ered gene ther­a­py for heart fail­ure in May af­ter the FDA came ask­ing for more de­tails on tar­get pa­tients. Three months down the road, Rock­et is clear for liftoff once again.

The FDA on Mon­day lift­ed its clin­i­cal hold on Rock­et’s Phase I tri­al for gene ther­a­py can­di­date RP-A501 for Danon dis­ease, an ul­tra-rare ge­net­ic dis­or­der that can lead to heart fail­ure in chil­dren and young adults, the com­pa­ny said in a re­lease.

It’s been a lit­tle more than three-month wait for Rock­et af­ter the agency ground­ed the tri­al, ask­ing for a re­vised pro­to­col on “pa­tient se­lec­tion and man­age­ment.”

On Mon­day, Rock­et said it had ad­dressed those is­sues and was ready to move for­ward, cit­ing no new safe­ty red flags churned up dur­ing the agency’s re­view. The com­pa­ny ex­pects to re­sume dos­ing in the adult co­hort “as soon as pos­si­ble” and is gear­ing up to be­gin en­roll­ment for a pe­di­atric co­hort as well, Rock­et said.

Un­like the rest of its pipeline, which us­es lentivi­ral vec­tors to trans­port its gene ther­a­pies, RP-A501 is Rock­et’s on­ly can­di­date that us­es AAV9 for de­liv­ery, a vec­tor that has re­ceived in­tense scruti­ny af­ter a wave of safe­ty flags in re­cent years. The ther­a­py us­es that vec­tor to trans­port a func­tion­al ver­sion of lyso­some-as­so­ci­at­ed mem­brane pro­tein 2 (LAMP-2) in­to pa­tients’ cells — the mu­tat­ed form of the pro­tein caus­es a buildup of au­tophago­somes and glyco­gen in the heart and oth­er tis­sues that can cause heart fail­ure and death, par­tic­u­lar­ly in boys and young men.

Rock­et be­lieves there are some­where be­tween 15,000 and 30,000 pa­tients with Danon in the US and Eu­rope. The drug would be­come the first gene ther­a­py ap­proved for mono­genic heart fail­ure, Rock­et said.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Fireside chat between Hal Barron and John Carroll, UKBIO19

It’s time we talked about bio­phar­ma — live in Lon­don next week

Zoom can only go so far. And I think at this stage, we’ve all tested the limits of staying in touch — virtually. So I’m particularly happy now that we’ve revved up the travel machine to point myself to London for the first time in several years.

Whatever events we have lined up, we’ve always built in plenty of opportunities for all of us to get together and talk. For London, live, I plan to be right out front, meeting with and chatting with the small crowd of biopharma people we are hosting on October 12 at Silicon Valley Bank’s London headquarters. And there’s a lengthy mixer at the end I’m most looking forward to, with several networking openings between sessions.

FDA+ roundup: Ad­comm date set for Cy­to­ki­net­ics heart drug; New gener­ic drug guid­ance to re­duce fa­cil­i­ty de­lays

The FDA on Wednesday set Dec. 13 as the day that its Cardiovascular and Renal Drugs Advisory Committee will review Cytokinetics’ potential heart drug, meaning regulators aren’t likely to meet the Nov. 30 PDUFA date that was previously set.

The drug, known as omecamtiv mecarbil, read out its first Phase III in November 2020, hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as the key to breaking into the market, failing to significantly differ in reducing cardiovascular death from placebo.

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Take­da to pull key hy­poparathy­roidism drug from the mar­ket af­ter years of man­u­fac­tur­ing woes

Takeda on Tuesday morning made an announcement that almost 3,000 people with the rare disease known as hypoparathyroidism were fearing.

Due to unresolved supply issues and manufacturing woes, Takeda said it will cut its losses and discontinue its hypoparathyroidism drug, known as Natpara (parathyroid hormone), halting all manufacturing of the drug by the end of 2024, but the entire inventory will be available until depleted or expired, a company spokesperson said via email.

Tim Miller, Forge Biologics CEO

CNS-fo­cused gene ther­a­py biotech forges man­u­fac­tur­ing deal for hear­ing loss drug

A growing gene therapy company is teaming up with one of the largest gene therapy manufacturers to get its drug into patients in clinical trials.

Myrtelle announced on Monday that it inked a deal with Forge Biologics to make Myr-201, a gene therapy indicated for monogenic hearing loss. The target patient population, Myrtelle said, is people with a type of hearing loss called DFNB8, or autosomal recessive deafness 8.

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Pfizer and BioNTech's original Marvel comic book links evolving Covid vaccine science to Avengers' evolving villain-fighting tools.(Source: Pfizer LinkedIn post)

Pfiz­er, BioN­Tech part­ner with Mar­vel for Avengers and Covid-fight­ing com­ic book

Pfizer and BioNTech are collaborating with Marvel to celebrate “everyday” people getting Covid-19 vaccines in a custom comic book.

In the “Everyday Heroes” digital comic book, an evolving Ultron, one of the Avengers’ leading villains, is defeated by Captain America, Ironman and others. The plotline and history of Ultron is explained by a grandfather who is waiting with his family at a clinic for Covid-19 vaccinations.

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Two CRLs lat­er, Illi­nois-based CD­MO hit with FDA warn­ing let­ter over 's­pore-form­ing' bac­te­ria and fun­gi

Dupo, IL-based contract manufacturer Sterling Pharmaceuticals has had a long, tough year.

Last September, partner Verrica Pharmaceuticals’ NDA for a potential skin disease treatment was hit with a second CRL, with the company pinning the problems on Sterling. Eight months later, Verrica was hit with a third CRL due to further issues at Sterling, following a February reinspection by FDA of the Sterling site.

David Cory, Eiger BioPharmaceuticals CEO (via MultiVu)

FDA re­jects Eiger's emer­gency use re­quest for re­pur­posed Covid-19 treat­ment

Eiger BioPharmaceuticals is no longer planning to submit an emergency use application to the FDA.

The Palo Alto, CA-based biotech had hoped to repurpose its hepatitis D treatment, peginterferon lambda, for mild-to-moderate Covid-19 — submitting a request to the FDA for a pre-EUA meeting back in September with data from a Phase III platform trial in hand.

However, that meeting was rejected by federal regulators because of “concerns about the conduct of the TOGETHER study,” per an Eiger statement.

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