FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a dif­fi­cult few years for gene and cell ther­a­py start­up Abeona Ther­a­peu­tics. Its new­ly crowned chief Carsten Thiel was forced out last year fol­low­ing ac­cu­sa­tions of un­spec­i­fied “per­son­al mis­con­duct,” and this Sep­tem­ber, the FDA im­posed a clin­i­cal hold on its ther­a­py for a form of “but­ter­fly” dis­ease. But things are be­gin­ning to perk up. On Mon­day, the com­pa­ny said the reg­u­la­tor had lift­ed its hold and the ex­per­i­men­tal ther­a­py is now set to be eval­u­at­ed in a late-stage study.

Carsten Thiel

The ther­a­py, EB-101, is be­ing de­vel­oped to treat re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa, which is caused by the de­fi­cien­cy of the pro­tein COL7 and for which there is no ap­proved ther­a­py. Abeona’s au­tol­o­gous ther­a­py in­volves trans­fer­ring COL7A1 genes in­to a pa­tient’s own skin cells, fol­low­ing which the cells are trans­plant­ed back in­to the pa­tient to en­able nor­mal Type VII col­la­gen ex­pres­sion and wound heal­ing.

Ear­ly Phase I/II da­ta show that EB-101 was safe and in­duced durable wound heal­ing with up to five years of fol­lowup, and Type VII col­la­gen ex­pres­sion was ob­served more than two years af­ter treat­ment. How­ev­er, in Sep­tem­ber, the FDA im­posed a hold on the pro­gram, ask­ing for ad­di­tion­al da­ta points on the “trans­port sta­bil­i­ty of EB-101 to clin­i­cal sites.”

The com­pa­ny, which al­so said it was re­view­ing ‘strate­gic op­tions’ in re­sponse to in­ter­est ear­li­er this year, now ex­pects to kick off the late-stage VI­ITAL study in the first quar­ter of 2020. The tri­al is ex­pect­ed to en­roll 10 to 15 pa­tients, with rough­ly 30 chron­ic wound sites. The main end­point of the study is the pro­por­tion of wounds with greater than 50% heal­ing at three months, com­par­ing treat­ed with un­treat­ed wound sites on the same pa­tient.

Abeona’s shares $ABEO jumped more than 9% to $3.28 in ear­ly Mon­day trad­ing.

Epi­der­mol­y­sis bul­losa (EB) is a group of ge­net­ic skin con­di­tions that cause the skin to blis­ter and tear due to min­i­mal con­tact — in­fants born with the dis­ease are called ‘but­ter­fly chil­dren’ as their skin is con­sid­ered as frag­ile as a wing of a but­ter­fly.

In April, Cas­tle Creek swooped to part­ner with the em­bat­tled gene and cell ther­a­py Fi­bro­cell to shep­herd its lead gene ther­a­py for re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa in­to late-stage de­vel­op­ment. Months lat­er, the New Jer­sey-based der­ma­tol­ogy com­pa­ny ac­quired its part­ner in a deal worth $63.3 mil­lion.

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.

FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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Andy Plump, Takeda R&D chief (Jeff Rumans for Endpoints News)

What kind of PhI­Ib da­ta is worth $4B cash? Take­da’s Andy Plump has some thoughts on that

A few months back, when Takeda caused jaws to drop with its eye-watering $4 billion cash upfront for a mid-stage TYK2 drug from Nimbus, it had already taken a deep dive on the solid Phase IIb data Nimbus had assembled from its dose-ranging study in psoriasis.

Now, it’s rolling that data out, eager to demonstrate what inspired the global biopharma to go long in a neighboring, but new, disease arena for the pipeline. And the most avid students of the numbers will likely be at Bristol Myers Squibb, who will have a multi-year head start on pioneering the TYK2 space with Sotyktu (deucravacitinib) as Takeda makes its lunge for best-in-class status.

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FDA warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

No­vo Nordisk re­mains un­der UK scruti­ny as MHRA con­ducts its own re­view in 'in­cred­i­bly rare' case

The UK’s Medicines and Healthcare products Regulatory Agency is now reviewing Novo Nordisk’s marketing violation that resulted in its loss of UK trade group membership last week. Novo Nordisk was suspended on Thursday from the Association of the British Pharmaceutical Industry (ABPI) for two years after an investigation by its regulatory arm found the pharma broke its conduct rules.

MHRA said on Tuesday that its review of the Prescription Medicines Code of Practice Authority (PMCPA) investigation is standard practice. An MHRA spokesperson emphasized in an email to Endpoints News that the situation with Novo Nordisk is “incredibly rare” while also noting ABPI took “swift and proportionate action.”

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Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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FTC says patent bat­tle over Parkin­son's drug could have 'sig­nif­i­cant im­pli­ca­tion­s' for pa­tients

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

Harpreet Singh, Immatics CEO

Im­mat­ics an­nounces mul­ti­ple pipeline changes with lat­est fi­nan­cial re­sults

The T-cell biotech Immatics is looking to make some changes to its pipeline.

Immatics released its 2022 financial results on Tuesday and announced that it’s planning to discontinue its program for IMA201, an experimental cell therapy for solid tumors that express the antigens known as MAGE4/8. It plans to shift focus to IMA401, a TCR bispecific which goes after the same target.

The German-based biotech said it will treat the remaining patients enrolled in the program before the discontinuation. No other reasons were given for the discontinuation. Endpoints News reached out to Immatics for more details but did not receive a response by press time.

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Mar­ket­ingRx roundup: What could a US Tik­Tok ban mean for phar­ma? Pfiz­er, Lil­ly lead phar­ma March Mad­ness ad­ver­tis­ers

Just as pharma marketers finally make moves into TikTok, the threat of a US ban on the social media channel is now looming. Already banned on federal employee phones by an initial Congressional act, more bills and maybe bans are on the way. With rare bipartisan agreement, lawmakers have introduced legislation that would give the US president the power to ban TikTok (although not mentioned by name) and other foreign-owned technology platforms that represent a security threat to the US.

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