Blue­bird goes 2-for-2 on gene ther­a­pies at FDA ad­comm with thumbs up for beti-cel

For the sec­ond straight day, the FDA’s Cel­lu­lar, Tis­sue and Gene Ther­a­pies ad­comm vot­ed unan­i­mous­ly in fa­vor of FDA ap­prov­ing a blue­bird bio gene ther­a­py, this time by a 13-0 vote in fa­vor of beti-cel as a po­ten­tial treat­ment for a blood dis­or­der known as β-tha­lassemia for those who re­quire reg­u­lar blood trans­fu­sions.

The sec­ond straight unan­i­mous thumbs up opens the po­ten­tial for two FDA ap­provals lat­er this sum­mer for blue­bird — al­though the agency on Fri­day raised some man­u­fac­tur­ing con­cerns for both ther­a­pies.

Ad­comm mem­bers praised the ef­fi­ca­cy of the po­ten­tial gene ther­a­py as “out­stand­ing” and “tremen­dous,” and that the ben­e­fits “def­i­nite­ly out­weigh the risks.” The FDA pre­sen­ter’s fi­nal ben­e­fit-risk as­sess­ment slide made clear that blue­bird’s clin­i­cal stud­ies demon­strate that 89% of sub­jects with TDT who re­ceived beti-cel have achieved trans­fu­sion in­de­pen­dence.

An­drew Oben­shain, CEO of blue­bird, said in a state­ment:

“To­day’s ad­vi­so­ry com­mit­tee rec­om­men­da­tion is recog­ni­tion of the sub­stan­tial body of clin­i­cal da­ta that sup­port beti-cel as a po­ten­tial­ly cu­ra­tive treat­ment op­tion for these pa­tients. We are grate­ful to the mem­bers of the be­ta-tha­lassemia com­mu­ni­ty who con­tributed to to­day’s dis­cus­sion and re­main com­mit­ted to work­ing with the FDA as it com­pletes its re­view of the beti-cel Bi­o­log­ics Li­cense Ap­pli­ca­tion.”

The com­mit­tee on Thurs­day al­so gave a big thumbs up to blue­bird bio’s po­ten­tial gene ther­a­py for the rare but fa­tal con­di­tion known as cere­bral adrenoleukody­s­tro­phy (CALD) by a vote of 15-0, de­spite FDA safe­ty con­cerns.

If both ther­a­pies win ap­proval, the biotech al­so would win two pri­or­i­ty re­view vouch­ers (worth up to about $200 mil­lion to­tal), which could pro­vide des­per­ate re­lief for blue­bird, which laid off about 30% of its work­force in April.

The agency al­so raised con­cerns about the lentivi­ral vec­tor used with beti-cel be­cause of the can­cer cas­es seen de­vel­op­ing in those re­ceiv­ing treat­ment from two oth­er po­ten­tial blue­bird gene ther­a­pies that use a sim­i­lar lentivi­ral vec­tor with a dif­fer­ent safe­ty pro­file. But most of the ad­comm mem­bers found the safe­ty pro­file of beti-cel to be ad­e­quate.

The FDA does not have to fol­low the ad­vice of its ad­comm but it of­ten does.

Ques­tions about a po­ten­tial $2.1 mil­lion price tag for beti-cel al­so might raise some eye­brows, but non­prof­it ICER has called the po­ten­tial gene ther­a­py cost-ef­fec­tive.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pearl Huang, Dunad Therapeutics CEO (Ken Richardson, PR Newswire)

Long­time biotech leader Pearl Huang takes the reins as CEO of No­var­tis-backed up­start

It has only been a few months since Pearl Huang exited the top seat at Cygnal Therapeutics, but now she’s back at the helm of another biotech.

After taking a few months off — passing an exam in that time to get her captain’s license from the US Coast Guard — she’s been named CEO of Dunad Therapeutics, a biotech focused on developing a small molecule covalent therapies that was founded in 2020. Huang told Endpoints News that two factors attracted her to going back to the c-suite: the company’s technology and its co-founders.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Laurence Reid, Decibel CEO

Still in pre­clin­i­cal test­ing for ear gene ther­a­pies, Deci­bel touts small snap­shot of chemo-in­duced hear­ing loss drug

Though Decibel Therapeutics has largely pivoted toward gene therapies for the inner ear, its lead clinical candidate simply aims to protect cancer patients from chemotherapy-induced hearing loss. On Tuesday, the biotech presented its first efficacy data for the program, and execs like what they see.

Decibel reported interim results from a Phase Ib study showing the experimental drug, dubbed DB-020, largely protected a small group of patients from losing their hearing. Researchers used a particularly unique study design, administering the compound in one of each patients’ ears before they received cisplatin chemotherapy and placebo in the other.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Ben Zimmer, Priovant CEO

Roivant un­veils lat­est spin­out as Pfiz­er en­trusts JAK1/TYK2 to Pri­o­vant

In November, Pfizer disclosed it’s spun out the Phase II dual JAK1/TYK2 inhibitor to a startup formed in collaboration with an unnamed, experienced partner.

We now know who the partner is. And as Pfizer and Roivant officially take the wraps off Priovant Therapeutics, the companies reveal that they have started two registrational trials of the drug, brepocitinib, as part of a broader plan to develop a big, first-in-class franchise spanning multiple orphan and specialty autoimmune diseases.

No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

Up­dat­ing the Covid-19 vac­cine: FDA of­fers a qual­i­fied thumbs-up ahead of ad­comm

The FDA’s adcomm of outside vaccine experts will meet tomorrow to discuss how to protect the US from a likely coming wave of Omicron cases in the fall and winter, and whether to deploy vaccines that specifically target the Omicron variant.

While the data so far are limited, the FDA sounded an upbeat tone in the briefing documents on Pfizer/BioNTech’s candidates, released this weekend ahead of the VRBPAC meeting.

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.