FDA keeps clin­i­cal hold in place on Lari­mar's lead drug with job cuts like­ly on the hori­zon

The FDA will ex­tend its clin­i­cal hold on Lari­mar Ther­a­peu­tics’ lead rare dis­ease drug CTI-1601, ask­ing for more da­ta be­fore it will re­con­sid­er the com­pa­ny’s case, Lari­mar said in a re­lease Tues­day.

The agency ini­tial­ly placed a hold on the Phase I pro­gram in Friedre­ich’s atax­ia back in May af­ter the com­pa­ny re­port­ed mul­ti­ple deaths of non-hu­man pri­mates in pre­clin­i­cal test­ing.

Shares in $LRMR were trad­ing down rough­ly 62% Tues­day morn­ing, from $8.56 to $3.40.

Ca­r­ole Ben-Mai­mon

With the hold now in place for even longer, Lari­mar said it is eval­u­at­ing da­ta from com­plet­ed stud­ies to fig­ure out if it wants to take on the headache of a new study. The com­pa­ny is al­so re­con­sid­er­ing tim­ing on a planned open-la­bel ex­pan­sion study, as well as an as­cend­ing-dose pe­di­atric tri­al.

“Our next step is to en­gage with the agency to de­ter­mine how we can meet their re­quest in the most ef­fi­cient and ex­pe­di­tious man­ner,” CEO Ca­r­ole Ben-Mai­mon said in a state­ment. “Based on all avail­able clin­i­cal and non-clin­i­cal da­ta, we con­tin­ue to be­lieve there is a path for­ward through the res­o­lu­tion of the CTI-1601 clin­i­cal hold.”

Once known as Chon­dr­i­al Ther­a­peu­tics, Lari­mar went pub­lic back in 2019 as part of a SPAC re­verse merg­er with a plat­form based on pro­tein re­place­ment ther­a­pies. CTI-1601 was con­ceived as the flag­ship pro­gram in that plat­form, look­ing to treat the rare ge­net­ic dis­ease Friedre­ich’s atax­ia.

The non-hu­man pri­mates that died in a pre­clin­i­cal tri­al had been giv­en the high­est dos­es of CTI-1601, a re­com­bi­nant fu­sion pro­tein de­signed to de­liv­er hu­man fratax­in in­to the mi­to­chon­dria, as part of a study around ex­tend­ed dos­ing of the drug. Pa­tients with FA are un­able to pro­duce enough of the pro­tein, which is es­sen­tial for meta­bol­ic func­tions.

Now, Lari­mar is look­ing to slash and burn as it paves its cash run­way out past 2023, Ben-Mai­mon said in a re­lease:

We re­main com­mit­ted to CTI-1601’s fur­ther de­vel­op­ment and are work­ing to­wards this goal with a strong cash po­si­tion which pro­vides run­way at least in­to 2023. We in­tend to op­er­ate un­der a cost re­duc­tion plan while re­solv­ing the clin­i­cal hold to man­age burn and ex­tend our cash run­way if need­ed.

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.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Eu­ro­pean Com­mis­sion de­lays pro­pos­al for ma­jor changes to phar­ma leg­is­la­tion

The European Commission has once again delayed the release of its proposal for an overhaul of the continent’s pharmaceutical legislation.

The release, previously anticipated on March 29, will occur “slightly later” than expected due to the “very busy College agendas of the last few weeks,” a Commission spokesperson told Endpoints News via email.

While the agency hasn’t provided an updated timeline, the spokesperson said the agenda is “always indicative and adoption dates of Commission proposals may change any time, especially when these proposals concern reforms of complex legislations of major importance.”

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.