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.

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.

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.

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 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.