Carole Ben-Maimon, CEO of Larimar Therapeutics

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.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

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Mene Pangalos (AstraZeneca via YouTube)

As­traZeneca shuts the PhI­II door for Ion­is' PC­SK9 drug de­spite pos­i­tive PhI­Ib

When Ionis and AstraZeneca unveiled the first round of mid-stage data for their antisense PCSK9 drug, Mene Pangalos, AstraZeneca’s EVP of biopharmaceuticals R&D, underscored the drug’s “potential best-in-class efficacy profile.”

But now that the second batch is in, it appears AZD8233 isn’t hitting the mark after all.

Ionis announced Friday morning that although the candidate, also dubbed ION449, met the primary endpoint in the Phase IIb SOLANO trial, its partners at AstraZeneca have decided not to move it into Phase III studies because the “results did not achieve pre-specified efficacy criteria.”

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Up­dat­ed: Bio­gen throws it­self back in­to mud­dled da­ta ar­gu­ments with more de­tails on its an­ti­sense ALS drug

With a highly watched FDA decision deadline coming in late January, Biogen and Ionis dropped the full data on the Phase III study of their ALS drug tofersen in the New England Journal of Medicine on Wednesday.

Biogen is looking for approval for tofersen in a very small subset of ALS patients — some 2%, according to the paper — who have a SOD1 gene mutation, which has previously been linked to ALS. Tofersen is meant to reduce levels of mutant SOD1 proteins.

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Richard Pazdur, FDA's OCE director (Flatiron Health via YouTube)

FDA's OCE makes the case for ac­cel­er­at­ed ap­proval rid­er in user fee reau­tho­riza­tion

Four experts from the FDA’s Oncology Center of Excellence took to the New England Journal of Medicine yesterday to make the case for not only improving the agency’s ability to expeditiously pull dangling accelerated approvals when, on the rare occasion, confirmatory trials fail, but also better building “quality and efficiency into the AA on-ramp.”

The timely perspective arrives as Congress has exactly one week left to draft, release and sign off on the reauthorized user fee deals before layoff notices will be sent to drug reviewers. That package, which is likely to hitch a ride with the continuing resolution, may or may not include several policy riders (opposed by Republicans), including one that would allow the FDA to require confirmatory trials to be underway before an AA is granted, and would improve the process by which FDA can withdraw AAs.

As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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