Carsten Brunn, Selecta Biosciences CEO

FDA lifts a clin­i­cal hold on Se­lec­ta gene ther­a­py af­ter an­oth­er de­lay ham­pers R&D plans

Three months af­ter Se­lec­ta Bio­Sciences got word that the FDA had slapped a clin­i­cal hold on its Phase I/II study of methyl­malonic acidemia drug SEL-302 to clear up some CMC is­sues, the biotech says they are back in busi­ness.

“I am pleased to an­nounce that on March 9th the FDA lift­ed the clin­i­cal hold on our SEL-302 gene ther­a­py pro­gram to treat methyl­malonic acidemia. We look for­ward to start­ing our phase 1 clin­i­cal tri­al ex­pe­di­tious­ly and to bring hope to those pa­tients and fam­i­lies seek­ing a po­ten­tial­ly durable and life­long treat­ment for this ter­ri­ble dis­ease,” said Se­lec­ta CEO Carsten Brunn in a pre­pared state­ment.

Back in No­vem­ber Brunn em­pha­sized that reg­u­la­tors had not raised any clin­i­cal is­sues around the gene ther­a­py, which leads that seg­ment of the pipeline. The dis­ease pre­vents chil­dren from me­tab­o­liz­ing amino acids, trig­ger­ing dan­ger­ous tox­i­c­i­ty.

Askle­pios had been part­nered on this pro­gram un­til the spring of last year when it hand­ed back full rights to Se­lec­ta. At the time, the biotech flagged a de­lay on its IND due to “a man­u­fac­tur­ing is­sue we be­lieve is re­lat­ed to a com­po­nent sourced from a third-par­ty.”

Se­lec­ta has been roughed up bad­ly by the shel­lack­ing be­ing hand­ed out to biotech com­pa­nies. Its stock has shed 55% of its val­ue in the past year. And it’s not the on­ly gene ther­a­py play­er to feel the reg­u­la­to­ry heat in the past year, as a fresh crop of safe­ty fears has af­flict­ed a large group of biotechs.

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.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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An­oth­er warn­ing let­ter for Lupin as FDA iden­ti­fies de­fi­cien­cies at In­dia-based site

With few new details of what needs fixing, Lupin disclosed last week that the FDA recently sent a warning letter to its Tarapur, India-based site.

After an inspection from March 22 to April 4, Lupin disclosed in an April stock filing that it received a Form 483 with four observations, but it didn’t offer any details on the observations.

Similar to comments made in April, the company said last week it does not believe the FDA slap will disrupt its drug supplies or the existing revenues from operations of this facility.

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Silviu Itescu, Mesoblast CEO

Mesoblast sends in im­proved po­ten­cy as­say, look­ing to re­sub­mit to FDA on acute graft-ver­sus-host dis­ease drug

In 2020, the FDA rejected Mesoblast’s remestemcel-L, or Ryoncil, its lead candidate for pediatric acute graft-versus-host disease (aGVHD) that didn’t respond to steroids. The FDA raised a number of concerns, first objecting to Mesoblast’s single arm, open-label trial, though regulators struggled to describe how a randomized trial would work, since pediatricians and parents were reluctant to put children with aGVHD in a placebo arm.

Tim Whitten, Taiho Oncology CEO

FDA green­lights Ot­su­ka sub­sidiary's bile duct can­cer drug

Otsuka subsidiary Taiho Oncology nabbed an accelerated approval on Friday for Lytgobi (futibatinib), its new kinase inhibitor to treat adults with a type of previously treated bile duct cancer, known as metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

The accelerated approval (meaning a confirmatory trial is necessary to show clinical benefit) follows a shortened six-month review, thanks to the FDA’s Real-Time Oncology Review program.