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.”

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

German pharma giant Bayer will be looking to make a significant investment into one of its US plants that produces over-the-counter drugs.

Bayer announced that it will spend $43.6 million to expand its facility in Myerstown, PA, a small town east of Harrisburg. Bayer plans to increase the site by 70,000 square feet and will have room for the installation of eight packaging lines and an area to install rooftop solar panels. The project is expected to be completed by 2025 and will add around 50 to 75 jobs.

As the global biologics market is expected to hit nearly the half-trillion-dollar mark this year, new JAMA research points to the importance of timely biosimilar entry, particularly as fewer biosimilars are entering the US than in Europe, and as monthly treatment costs for biosimilars were “substantially higher” in the US compared with Germany and Switzerland.

Among the three countries, biosimilar market share at launch was highest in Germany, but increased at the fastest rate in the US, the authors from the University of Zurich’s Institute of Law wrote in JAMA Network Open today.

The European Medicines Agency said Friday that it’s pulling from all European markets pholcodine-containing medicines, which are an opioid used in adults and children for the treatment of dry cough and in combo with other drugs as a treatment for cold and flu.

The decision to pull the medicines comes as the EMA points to the results from the recent ALPHO study, which show that use of pholcodine during the 12 months preceding anesthesia is linked to a risk of an anaphylactic reaction related to the neuromuscular blocking agents (NMBAs) used (with an adjusted OR of 4.2, and a 95% confidence interval of 2.5 to 6.9).