Elvire Gouze, InnoSkel CEO

Af­ter scor­ing big Pfiz­er buy­out, a Ther­a­chon co-founder turns once again to dwarfism with $24M Se­ries A

Less than two years af­ter sell­ing her first biotech to Pfiz­er for a hefty $810 mil­lion, a French re­searcher has launched a new gene ther­a­py out­fit that’s fo­cus­ing on the same tar­get — dwarfism — but tak­ing a dif­fer­ent path to treat­ment.

The sci­en­tist is Elvire Gouze and the com­pa­ny is In­noSkel, which launched with €20 mil­lion in Se­ries A fi­nanc­ing (about $24.2 mil­lion) ear­ly Mon­day. Though In­noSkel is aim­ing to treat a whole host of rare skele­tal dis­eases that af­fect the body’s col­la­gen, it’s first go­ing af­ter what’s known as spondy­loepi­phy­seal dys­pla­sia con­geni­ta (SEDc), the sec­ond-lead­ing cause of dwarfism that stunts the growth of a pa­tient’s car­ti­lage.

Mon­day’s fundraise was co-led by Jeito Cap­i­tal and Arie Bellde­grun’s team at Vi­da Ven­tures. Ré­gion Sud In­vestisse­ment and The Turenne Group al­so par­tic­i­pat­ed in the round.

Gouze had been work­ing on skele­tal dys­pla­sia at In­serm, the French equiv­a­lent of the NIH, and helped launch the new­ly Pfiz­er-owned Ther­a­chon. She kept her aca­d­e­m­ic lab while work­ing there and came up with the idea for a gene ther­a­py in type 2 col­lagenopathies, which in­cludes SEDc.

“That’s how we start­ed In­noSkel, it’s a spin-out of my In­serm lab,” Gouze told End­points News. 

There are two main caus­es of dwarfism, Gouze said, the first of which was a lead­ing tar­get for Ther­a­chon. While the biotech’s re­search cen­ters al­so re­volve around rare mus­cu­loskele­tal dis­or­ders, Ther­a­chon’s mis­sion was to treat the com­pli­ca­tions of achon­dropla­sia like those in the body’s car­dio­vas­cu­lar, neu­ro­log­i­cal and meta­bol­ic sys­tems.

Achon­dropla­sia af­fects bone growth and aris­es from a sep­a­rate ge­net­ic mu­ta­tion, re­sult­ing in a com­plete­ly dif­fer­ent mech­a­nism but sim­i­lar char­ac­ter­is­tics of dwarfism as SEDc. Ther­a­chon’s ex­per­i­men­tal drug is an FGFR3 de­coy, which they be­lieve will cor­rect a sig­nal­ing path­way that trig­gers the ab­nor­mal growth.

Though both con­di­tions re­sult in in­di­vid­u­als with dwarfism hav­ing the same phys­i­cal ap­pear­ance, Gouze says the com­pli­ca­tions in this car­ti­lage-based dis­or­der are quite dif­fer­ent. Rather than their bones grow­ing ab­nor­mal­ly, pa­tients with SEDc see ef­fects in their growth plates — the car­ti­lage “tem­plate” of bones in chil­dren that haven’t formed yet — es­sen­tial­ly ne­ces­si­tat­ing treat­ment be­fore pu­ber­ty.

This can lead to reti­nal de­tach­ments and pa­tients hav­ing trou­ble breath­ing, Gouze said. Fur­ther dif­fi­cul­ties hold­ing up their heads and necks can re­sult in paral­y­sis.

“In this case, the pa­tients are very short and have se­vere com­pli­ca­tions,” Gouze said. “What we’re try­ing to achieve is to re­pair the ex­o­cyclic ma­trix of the car­ti­lage.”

In­noSkel isn’t ready to di­vulge how their pro­gram treats the con­di­tion, nor can Gouze pre­dict when they’ll first hit the clin­ic as the com­pa­ny is still await­ing the pub­li­ca­tion of a peer-re­viewed pa­per. Mon­day’s fund­ing will go to­ward ad­vanc­ing to hu­man tri­als, how­ev­er, and any prod­uct will look like a typ­i­cal gene-ther­a­py in­jec­tion to pro­vide pro­teins that help the car­ti­lage be­have nor­mal­ly, she said.

Try­ing to treat dwarfism is a cause that’s drawn sig­nif­i­cant op­po­si­tion from some cor­ners of the com­mu­ni­ty. It’s one Gouze is all too fa­mil­iar with giv­en her pre­vi­ous role as sci­en­tif­ic founder of Ther­a­chon, and now she’s back for more with In­noSkel. There have al­so been ef­forts by a promi­nent phar­ma, Bio­Marin, to get an achon­dropla­sia drug across the fin­ish line. The FDA ac­cept­ed Bio­Marin’s pitch for the pro­gram just last month, with an ac­tion date com­ing next Au­gust.

But Gouze says she “ful­ly re­spects” the ar­gu­ment from ad­vo­cates that dwarfism is not some­thing meant to be “cured.” Mak­ing every­one taller is not Gouze’s main goal, she says, rather it’s try­ing to pre­vent the com­pli­ca­tions that re­sult from achon­dropla­sia and now SEDc.

“When you’re an adult with dwarfism you have to live with your con­di­tion any­way, and it’s not to make them like every­body; the treat­ments are not try­ing to ‘stan­dard­ize’ them,” Gouze said. “It’s not so much to make every­body look the same, which would be use­less and ab­solute­ly not in­ter­est­ing any­way. It’s try­ing to pre­vent the com­pli­ca­tions and de­crease the pain.”

The Price of Re­lief: Ex­plor­ing So­lu­tions to the Ris­ing Costs of On­col­o­gy Drugs

In 2020, The National Cancer Institute estimated about 1.8 million new cases of cancer diagnosed in the United States, while the costs associated with treatment therapies continued to escalate. Given the current legislative climate on drug pricing, it’s never been more important to look at the evolution of drug pricing globally and control concerns of sustainable and affordable treatments in oncology.

Lat­est news on Pfiz­er's $3B+ JAK1 win; Pacts over M&A at #JPM22; 2021 by the num­bers; Bio­gen's Aduhelm reck­on­ing; The sto­ry of sotro­vimab; 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.

For those of you who attended #JPM22 in any shape or form, we hope you had a fruitful time. Regardless of how you spent the past hectic week, may your weekend be just what you need it to be.

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Susan Galbraith, AstraZeneca EVP, Oncology R&D

Can­cer pow­er­house As­traZeneca rolls the dice on a $75M cash bet on a buzzy up­start in the on­col­o­gy field

After establishing itself in the front ranks of cancer drug developers and marketers, AstraZeneca is putting its scientific shoulder — and a significant amount of cash — behind the wheel of a brash new upstart in the biotech world.

The pharma giant trumpeted news this morning that it is handing over $75 million upfront to ally itself with Scorpion Therapeutics, one of those biotechs that was newly birthed by some top scientific, venture and executive talent and bequeathed with a fortune by way of a bankroll to advance an only hazily explained drug platform. And they are still very much in the discovery and preclinical phase.

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A $3B+ peak sales win? Pfiz­er thinks so, as FDA of­fers a tardy green light to its JAK1 drug abroc­i­tinib

Back in the fall of 2020, newly crowned Pfizer chief Albert Bourla confidently put their JAK1 inhibitor abrocitinib at the top of the list of blockbuster drugs in the late-stage pipeline with a $3 billion-plus peak sales estimate.

Since then it’s been subjected to serious criticism for the safety warnings associated with the class, held back by a cautious FDA and questioned when researchers rolled out a top-line boast that their heavyweight contender had beaten the champ in the field of atopic dermatitis — Dupixent — in a head-to-head study.

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Robert Califf, FDA commissioner nominee (Graeme Sloan/Sipa USA/Sipa via AP Images)

Rob Califf ad­vances as Biden's FDA nom­i­nee, with a close com­mit­tee vote

Rob Califf’s second confirmation process as FDA commissioner is already much more difficult than his near unanimous confirmation under the Obama administration.

The Senate Health Committee on Thursday voted 13-8 in favor of advancing Califf’s nomination to a full Senate vote. Several Democrats voted against Califf, including Sen. Bernie Sanders and Sen. Maggie Hassan. Several other Democrats who aren’t on the committee, like West Virginia’s Joe Manchin and Ed Markey of Massachusetts, also said Thursday that they would not vote for Califf. Markey, Hassan and Manchin all previously expressed reservations about the prospect of Janet Woodcock as an FDA commissioner nominee too.

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Michel Vounatsos, Biogen CEO (World Economic Forum/Ciaran McCrickard)

Bio­gen vows to fight CM­S' draft cov­er­age de­ci­sion for Aduhelm be­fore April fi­nal­iza­tion

Biogen executives made clear in an investor call Thursday they are not preparing to run a new CMS-approved clinical trial for their controversial Alzheimer’s drug anytime soon.

As requested in a draft national coverage decision from CMS earlier this week, Biogen and other anti-amyloid drugs will need to show “a meaningful improvement in health outcomes” for Alzheimer’s patients in a randomized, placebo-controlled trial to get paid for their drugs, rather than just the reduction in amyloid plaques that won Aduhelm its accelerated approval in June.

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CRO own­er pleads guilty to ob­struct­ing FDA in­ves­ti­ga­tion in­to fal­si­fied clin­i­cal tri­al da­ta

The co-owner of a Florida-based clinical research site pleaded guilty to lying to an FDA investigator during a 2017 inspection, revealing that she falsely portrayed part of a GlaxoSmithKline pediatric asthma study as legitimate, when in fact she knew that certain data had been falsified, the Department of Justice said Wednesday.

Three other employees — Yvelice Villaman Bencosme, Lisett Raventos and Maytee Lledo — previously pleaded guilty and were sentenced in connection with falsifying data associated with the trial at the CRO Unlimited Medical Research.

‘Skin­ny la­bels’ on gener­ics can save pa­tients mon­ey, re­search shows, but re­cent court de­ci­sions cloud fu­ture

New research shows how generic drug companies can successfully market a limited number of approved indications for a brand name drug, prior to coming to market for all of the indications. But several recent court decisions have created a layer of uncertainty around these so-called “skinny” labels.

While courts have generally allowed generic manufacturers to use their statutorily permitted skinny-label approvals, last summer, a federal circuit court found that Teva Pharmaceuticals was liable for inducing prescribers and patients to infringe GlaxoSmithKline’s patents through advertising and marketing practices that suggested Teva’s generic, with its skinny label, could be employed for the patented uses.

A patient in Alaska receiving an antibody infusion to prevent Covid hospitalizations in September. All but one of these treatments has been rendered useless by Omicron (Rick Bowmer/AP Images)

How a tiny Swiss lab and two old blood sam­ples cre­at­ed one of the on­ly ef­fec­tive drugs against Omi­cron (and why we have so lit­tle of it)

Exactly a decade before a novel coronavirus broke out in Wuhan, Davide Corti — a newly-minted immunologist with frameless glasses and a quick laugh — walked into a cramped lab on the top floor of an office building two hours outside Zurich. He had only enough money for two technicians and the ceiling was so low in parts that short stature was a job requirement, but Corti believed it’d be enough to test an idea he thought could change medicine.

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