FDA puts the brakes on a rare dis­ease biotech’s PhI­II as reg­u­la­tors wait for an up­date — shares crum­ble

Lit­tle Abeona Ther­a­peu­tics $ABEO is back in trou­ble this morn­ing.

The biotech re­port­ed that the FDA is slam­ming the brakes on their Phase III study of EB-101 for rare cas­es of re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa, which is char­ac­ter­ized by frag­ile skin that blis­ters quick­ly.

The drug us­es gene trans­fer to de­liv­er COL7A1 genes in­to a pa­tient’s own skin cells, which are then put back in­to the pa­tient’s skin for im­proved wound heal­ing. It has RMAT and break­through sta­tus at the FDA but doesn’t have ap­proval for the Phase III.

It won’t get that green light un­til the com­pa­ny pro­vides “ad­di­tion­al da­ta points” on trans­port sta­bil­i­ty.

The biotech’s shares were quick­ly ham­mered on the news, drop­ping more than 20%.

João Sif­fert, the CEO of Abeona, got the job last fall af­ter Alex­ion vet and then new CEO Carsten Thiel was boot­ed af­ter the board ac­cused him of per­son­al mis­con­duct. The biotech start­ed the day with a mar­ket cap of $160 mil­lion.

“Ini­ti­at­ing the VI­ITAL piv­otal Phase III tri­al for EB-101 is the Com­pa­ny’s top pri­or­i­ty,” said Sif­fert in a state­ment. “Ef­forts to gath­er sup­ple­men­tal da­ta points on trans­port sta­bil­i­ty of EB-101 are on­go­ing and we are con­fi­dent that the re­quest­ed ad­di­tion­al da­ta will be sub­mit­ted to the FDA prompt­ly. Look­ing ahead, we be­lieve that com­ple­tion of our CMC work cou­pled with the durable safe­ty and ef­fi­ca­cy da­ta, now out to five years in some pa­tients, will ul­ti­mate­ly be crit­i­cal to sup­port a fu­ture Bi­o­log­ics Li­cense Ap­pli­ca­tion.”

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

His­toric drug pric­ing re­forms pass; Pfiz­er ac­quires GBT; The long search for non-opi­oid pain 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.

The Endpoints Weekly has officially crossed the 60,000 mark on subscribers — thanks to all of your support. As the editorial team grows, we’ve been able to do a lot more, with many of those on display this week. Be sure to check out Lei Lei Wu’s deep dive on pain R&D. If you missed it, you may also rewatch her companion panel here.

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Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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No­var­tis re­ports two pa­tient deaths af­ter treat­ment with Zol­gens­ma

Two children with spinal muscular atrophy have died after receiving Novartis’ Zolgensma, a gene therapy designed as a one-time treatment for the rare fatal disease.

The deaths, which resulted from acute liver failure, occurred in Russia and Kazakhstan, Novartis confirmed in a statement to Endpoints News. Having notified health authorities across all the markets where Zolgensma is available, it will update the drug label “to specify that fatal acute liver failure has been reported,” a spokesperson wrote.

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FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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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.

Elcin Barker Ergun, Menarini Group CEO

Amid Roche and Sanofi's oral SERD set­backs, Menar­i­ni gets speedy re­view at FDA

Menarini and Radius Health are getting a speedy review at the FDA for their oral SERD breast cancer drug months after the field opened up with competitors failing and fleeing.

It was a one-two-three punch in March, April and May as Sanofi flunked its first big test for its oral selective estrogen receptor degrader (SERD), Roche also flamed out in a Phase II and G1 Therapeutics ended its program after scoping out the data and potential partners.

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House pass­es his­toric drug pric­ing re­forms, lin­ing up decades-in-the-mak­ing win for Biden and De­moc­rats

The US House of Representatives today voted along party lines (all Dems voted for it), 220-207 to pass new, wide-ranging legislation that will allow Medicare drug price negotiations for the first time ever, and cap seniors’ drug expenses to $2,000 per year and seniors’ insulin costs at $35 per month.

Setting up a major victory for President Joe Biden, representatives returned from their summer recess to pass the Inflation Reduction Act, even as many noted the bill would only modestly reduce inflation.

Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.