Pro­to­cols: Snubbed at the FDA, again, Chi­as­ma chops staff, again; J&J lands sec­ond BTD for de­pres­sion drug es­ke­t­a­mine

A few days af­ter ac­knowl­edg­ing that it was slapped down, again, by reg­u­la­tors, Chi­as­ma is chop­ping near­ly half its work­force in a sec­ond re­struc­tur­ing. Chi­as­ma CEO Mark Leucht­en­berg­er says the move to ax 44% of its re­main­ing staff was need­ed so the com­pa­ny can con­serve its cash while talk­ing to the FDA about its re­quire­ments on prov­ing the ef­fi­ca­cy of My­capssa (oc­treotide) for acromegaly. The FDA re­ject­ed My­capssa and re­cent­ly re­peat­ed a de­mand that the biotech de­sign and ex­e­cute a much bet­ter late-stage study. Reg­u­la­tors were un­hap­py with Chi­as­ma’s sin­gle arm study for a drug that is seek­ing to re­place the in­jecta­bles now in use.

J&J has picked up its sec­ond break­through drug des­ig­na­tion for es­ke­t­a­mine. The in­tranasal ver­sion of ke­t­a­mine, a pow­er­ful anes­thet­ic used in par­ty cir­cles as ‘Spe­cial K,’ has won an­oth­er ti­tle for de­pres­sion with an im­mi­nent risk of sui­cide. The drug was hand­ed the orig­i­nal BTD based on its po­ten­tial for treat­ing ma­jor de­pres­sion. Ke­t­a­mine has been stud­ied in a host of aca­d­e­m­ic tri­als, demon­strat­ing a pow­er­ful, though typ­i­cal­ly short-lived, im­pact on de­pres­sion as well as sui­ci­dal think­ing. Oth­er de­vel­op­ers like Al­ler­gan have their own CD­MA re­cep­tor ri­vals in the clin­ic. The BTD is in­tend­ed to help speed the reg­u­la­to­ry re­view of a drug with po­ten­tial to help change the stan­dard of care in its field.

Vedan­ta Bio­sciences has  land­ed a U.S. patent for its phar­ma­ceu­ti­cal com­po­si­tions and mix­tures of Clostrid­i­um live bac­te­r­i­al strains.

As ex­pect­ed, Eleven Bio has wrapped up its $270 mil­lion deal with Roche cov­er­ing the world­wide de­vel­op­ment and com­mer­cial­iza­tion rights to EBI-031. The deal in­cludes $30 mil­lion in up­front cash. This is the biotech’s last re­main­ing pipeline project fol­low­ing the back-to-back tri­al fail­ures of its lead drug.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Alaa Halawa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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Sanofi, Re­gen­eron boast PhI­II win with Dupix­ent in COPD, clear­ing first bar for ex­pan­sion

Dupixent, the blockbuster anti-inflammatory drug from Sanofi and Regeneron, has cleared a high-stakes Phase III study in chronic obstructive pulmonary disease, the companies announced Thursday morning.

If they hold up in a second, identical trial, the data pave the way for Dupixent to become the first biologic to treat patients whose COPD remains uncontrolled despite being on maximal standard-of-care inhaled therapy — the patient population studied in the pivotal program. The companies had spotlighted this as a key readout as they look to expand the Dupixent franchise and explore its full potential.

Genen­tech to stop com­mer­cial man­u­fac­tur­ing at Cal­i­for­nia head­quar­ters

Genentech is halting commercial manufacturing at its California headquarters — and laying off several hundred employees.

The move is the result of a decision Genentech made in 2007 to relocate manufacturing operations from its South San Francisco headquarters location to other facilities or move the work to CDMOs, said Andi Goddard, Genentech’s SVP of quality and compliance for pharmaceutical technical operations, in an interview with Endpoints News. Genentech has made changes in capabilities and invested more in technology, so it doesn’t need as many large-scale manufacturing facilities as it did in the past, she said.

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In­cyte wins ac­cel­er­at­ed ap­proval for PD-1 in rare skin can­cer

Incyte touted an accelerated approval for its PD-1 retifanlimab in a rare skin cancer on Wednesday, roughly a year and a half after the drug suffered a rejection in squamous cell carcinoma of the anal canal (SCAC).

Retifanlimab, marketed as Zynyz, was approved for metastatic or recurrent locally advanced Merkel cell carcinoma (MCC), a fast-growing skin cancer typically characterized by a single, painless nodule. It’s roughly 40 times rarer than melanoma, according to the nonprofit Skin Cancer Foundation — but incidence is growing, particularly among older adults, Incyte said in its announcement.

A new study finds that many patient influencers are sharing prescription drug experiences along with health information.

So­cial me­dia pa­tient in­flu­encers ‘danc­ing in the gray’ of phar­ma mar­ket­ing, more clar­i­ty need­ed, re­searcher says

It’s no surprise that patient influencers are talking about their health conditions on social media. However, what’s less clear is what role pharma companies are playing, how big the patient influencer industry is, and just how is information about prescription drugs from influencers relayed — and received — on social media.

While University of Colorado associate professor Erin Willis can’t answer all those questions, she’s been researching the issue for several years and recently published new research digging into the communication styles, strategies and thinking of patient influencers, many of whom partner with pharma companies.

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Vas Narasimhan, Novartis CEO (Gian Ehrenzeller/Keystone via AP)

No­var­tis pulls the plug on UK-based car­dio­vas­cu­lar study

Novartis is calling off a UK-based trial for Leqvio in the primary prevention of cardiovascular events in patients with high cholesterol, the company confirmed on Wednesday.

The Swiss pharma giant made the decision after “careful evaluation,” a spokesperson told Endpoints News via email. The trial, dubbed ORION-17, was planned in partnership with England’s National Health Service (NHS) and was part of the company’s strategy to establish Leqvio as a standard of care in cardiovascular disease management.