Larimar to respond to FDA this quarter on 15-month clinical hold of PhI Friedreich's ataxia drug
More than a year after the FDA clamped down on Larimar Therapeutics with a clinical hold on its lead drug, the biotech thinks it has a way to get that lifted — and an answer could come next quarter if all goes to plan.
Amid a 15-month setback, the biotech plans on filing a complete response to the FDA’s concerns over its drug, CTI-1601, sometime this quarter, which is supposed to elicit an answer from the agency within 30 days, per the regulator.
At the same time Larimar reported its Q2 financial status, the biotech said Thursday it had received meeting minutes from FDA following a sit-down with the regulator. The purpose of that conversation, Larimar said, was to get feedback on what was still needed to resolve a clinical hold on the biotech’s lead drug candidate.
CTI-1601 is a recombinant fusion protein that has been investigated to treat the rare genetic disease Friedreich’s ataxia, or FA. The inherited disease is a progressive one, gradually causing damage to the nervous system and movement problems. How the drug would work was it would deliver human frataxin into the mitochondria, as patients with FA cannot produce enough of the protein vital for metabolic functions.
As for what the company is planning to do to get the drug back in the clinic, Larimar CEO Carole Ben-Maimon said in a statement that the meeting “informed the preparation and planned submission of a complete response that we believe will enable CTI-1601’s return to the clinic.”
The CEO then went on to mention the biotech has also proposed a Phase II dose exploration study to provide additional information on the drug’s safety profile, on top of pharmacokinetic and pharmacodynamic profiles, to find a working dose level for long-term dosing.
Larimar declined to comment to Endpoints News beyond the company statement issued Thursday.
Investors supported the move, sending the near-penny stock outfit’s share price $LRMR up 20% in pre-market trading. The biotech closed Thursday at $1.98 per share.
The company has approximately $54.9 million in cash and marketable debt securities as of June 30. At the current pace, Larimar, without an approved product, has enough runway to last into Q3 next year.
The biotech’s CTI-1601 program had been originally placed on clinical hold by the FDA back in May 2021 following mortalities in non-human primates in a 26-week toxicology study, sending the company’s share price down by close to half practically overnight and scuttling a planned $95 million cash infusion. Nine months later, the FDA decided to extend the clinical hold while asking for more data — which sent the biotech’s stock price down close to 50% again in the aftermath. The biotech’s share price is down 86% since the hold was put in place.
And Larimar is not the only company going after Friedreich’s ataxia, which currently has no cure — nor the only one who’s hit roadblocks. Just earlier this week, Reata Pharmaceuticals put out word that the FDA was still concerned with its FA drug candidate omaveloxolone — and that with more data in hand, the regulatory agency just delayed a PDUFA decision by three months and punted it to February 2023.