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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Cell and gene ther­a­pies from acad­e­mia: EMA to help 5 projects go­ing af­ter un­met clin­i­cal needs

The European Medicines Agency said Thursday that it’s launching a new pilot program to help academic and other nonprofit researchers developing advanced therapy medicinal products, which includes cell and gene therapies.

Academics have proven to be enormously useful in feeding new products, like chimeric antigen receptor (CAR)-T cell therapies first developed by Memorial Sloan Kettering, and ushered to the market by biopharma companies. Jean Bennett, formerly with the University of Pennsylvania, also saw her research lead to the approval of gene therapy Luxturna, which Roche now owns.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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Aligos co-founders Leonid Beigelman (L) and Lawrence Blatt

Ali­gos co-founders shoot back at J&J IP com­plaint with one of their own al­leg­ing fraud

This story goes all the way back to 2014.

In November of 2014, Johnson & Johnson acquired Alios BioPharma, an infectious disease biotech that was co-founded by Lawrence Blatt and Leonid Beigelman.

Following J&J’s 2014 acquisition, Blatt and Beigelman would become employees at J&J’s Janssen arm, with Blatt being the global head of infectious diseases and vaccines and Beigelman being Janssen’s VP of medicinal chemistry. But Blatt and Beigelman left Janssen in 2017, starting Aligos one year later.

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Bill Hinshaw, Axcella CEO

Ax­cel­la touts in­ter­im NASH da­ta but needs cash to stay afloat for 2024 topline read­out

Axcella Therapeutics, with an empty bank account looming in the first quarter of next year and a recent setback in a mid-stage long Covid study, is out with some interim data on its NASH drug.

The biotech isn’t yet saying how the drug performed on the primary endpoint of improvement in the nonalcoholic fatty liver disease (NAFLD) Activity Score (NAS), but the first batch of results has sent shares $AXLA soaring nearly 15% before Thursday’s opening bell.