Geron sur­vives, for now, but the ju­ry at J&J is still out on the fu­ture of ime­tel­stat

Geron has sur­vived an­oth­er scrape with dis­as­ter, but it’s still op­er­at­ing un­der a dark cloud.

This morn­ing the biotech an­nounced that J&J’s re­view of the da­ta from two stud­ies of its drug ime­tel­stat war­rant­ed con­tin­ued work in myelodys­plas­tic syn­dromes and myelofi­bro­sis. But the phar­ma gi­ant $JNJ is still re­serv­ing the right to quit if the da­ta don’t hold up lat­er in the year.

That’s not a big vote of con­fi­dence.

In­vestors re­spond­ed to the pos­i­tive, though, send­ing shares of Geron up 17% in pre-mar­ket trad­ing.

The stud­ies for this drug in­clude a Phase II/III study in low-risk myelodys­plas­tic syn­dromes. Now Geron says that they’ll be shar­ing da­ta with the FDA be­fore de­cid­ing whether to launch the sec­ond part of that study, which is be­ing tweaked.

Geron al­so isn’t like­ly to spur much ex­cite­ment with the ob­ser­va­tion that the spleen vol­ume re­sponse in myelofi­bro­sis was less than that seen for oth­er drugs. But Geron hur­ried on to note that there were sev­er­al signs of clin­i­cal ben­e­fit to en­cour­age re­searchers, adding that “the da­ta sug­gest a po­ten­tial over­all sur­vival ben­e­fit as­so­ci­at­ed with ime­tel­stat treat­ment in these pa­tients.”

En­roll­ment in that sec­ond study, though, is still on hold — al­so not an en­cour­ag­ing sign. And the ju­ry is still out:

Geron ex­pects the longer-term da­ta from the tri­al, po­ten­tial health au­thor­i­ty feed­back, and the to­tal­i­ty of ime­tel­stat pro­gram in­for­ma­tion, in­clud­ing an as­sess­ment of the evolv­ing treat­ment land­scape in MF and the po­ten­tial ap­pli­ca­tion of ime­tel­stat in mul­ti­ple hema­to­log­ic ma­lig­nan­cies, in­clud­ing MDS, will in­form Janssen’s de­ci­sion whether to con­tin­ue de­vel­op­ment of ime­tel­stat in re­lapsed or re­frac­to­ry MF.

Geron shares $GERN took a beat­ing last fall af­ter its drug, a telom­erase in­hibitor, ran in­to trou­ble dur­ing its key Phase II tri­al run by J&J. The low dose group — re­lapsed or re­sis­tant to JAK1 ther­a­py — was not see­ing an ad­e­quate re­sponse and in­ves­ti­ga­tors opt­ed to shut that wing of the tri­al down, trans­fer­ring pa­tients over to the high dose. And new en­roll­ment in the high dose arm was sus­pend­ed so in­ves­ti­ga­tors could take a care­ful look at more ma­ture da­ta.

Geron re­or­ga­nized back in 2011, drop­ping its work on stem cell ther­a­pies and re­mak­ing it­self as a can­cer drug de­vel­op­er. The biotech has gone more than two decades, though, with­out find­ing a drug that war­rants ap­proval.

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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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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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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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.

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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Drug short­age so­lu­tions brought be­fore Sen­ate Home­land Se­cu­ri­ty com­mit­tee

With more than 300 active drug shortages, the Senate Committee on Homeland Security and Governmental Affairs had its hands full on Wednesday with multiple experts testifying on drug shortages and possible solutions.

A picture of the shortage situation. presented by Erin Fox, an adjunct professor at the College of Pharmacy at the University of Utah, explained how some patients have died due to drug shortages, including with medication errors when substitutes were dosed incorrectly or when an emergency product was not available.

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FDA re­jects Ab­b­Vie's in­fu­sion ther­a­py for Parkin­son's, re­quests more in­fo on pump de­vice

The FDA rejected AbbVie’s 24-hour infusion therapy for Parkinson’s, saying it needs more information on a device used to administer the treatment before it can clear it.

The Chicago-area drugmaker said in a press release that the complete response letter from the agency didn’t include any requests for more efficacy or safety trials related to the drug, known as ABBV-951. The company said it aims to “resubmit the NDA as soon as possible.”

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