John Quisel

Roche schiz­o­phre­nia drug that failed PhI­II gets a new life in rare dis­or­der as Or­biMed leads $90M for start­up

Un­til a few years ago, GlyT1, a glycine trans­porter, seemed like a promis­ing tar­get for an add-on ther­a­py to treat neg­a­tive symp­toms of schiz­o­phre­nia.

But sci­en­tists were thrust­ed back to the draw­ing board in 2014, when Roche re­port­ed that its GlyT1 in­hibitor, bitop­er­tin, had failed two Phase III tri­als. A few months lat­er, they scrapped the pro­gram al­to­geth­er.

The drug sat on the shelves for sev­en years. Then Disc Med­i­cine came along.

The biotech, which had li­censed bitop­er­tin from Roche in a $200 mil­lion deal with an undis­closed up­front, has now nabbed $90 mil­lion in Se­ries B cash to take the drug in a com­plete­ly dif­fer­ent di­rec­tion: rare ge­net­ic dis­or­ders known as ery­thro­poi­et­ic por­phyr­ias.

Will Sav­age

Disc Med­i­cine had a lead pro­gram on its own: DISC-0974, an ane­mia drug that works by con­trol­ling iron me­tab­o­lism and is al­ready in the clin­ic. The hema­tol­ogy-fo­cused com­pa­ny, though, had been re­search­ing oth­er ways to treat dis­eases by go­ing af­ter “fun­da­men­tal com­po­nents” of path­ways re­lat­ed to red blood cells, said CEO John Quisel, so as to ful­ly take ad­van­tage of the ex­pert net­work and ca­pa­bil­i­ties it’s built.

One of those path­ways is heme syn­the­sis. In ery­thro­poi­et­ic por­phyr­ias, a buildup of por­phyrins lead to tox­ic re­ac­tions to sun­light — 30 min­utes un­der the sun could cause se­vere pain and a burn­ing sen­sa­tion.

Across a se­ries of cel­lu­lar and mouse ex­per­i­ments, re­searchers found that tar­get­ing a heme metabo­lite called pro­to­por­phyrin IX could fix the de­fect that re­sults in por­phyrin ac­cu­mu­la­tion.

Be­cause Roche had been look­ing at glycine con­trol as a way to treat schiz­o­phre­nia pa­tients, in­ves­ti­ga­tors had mea­sured those met­rics and showed that’s ex­act­ly what the drug did in pa­tients — even though they were “hema­to­log­i­cal­ly nor­mal.” Clin­i­cal da­ta from thou­sands of pa­tients al­so sug­gest a clean safe­ty pro­file.

While a host of GlyT1 in­hibitors had been cre­at­ed, many had failed in ear­li­er stages for safe­ty or oth­er rea­sons. Roche of­fered the best mol­e­cule with a sol­id clin­i­cal pack­age.

“It’s very rare to be able to move in­to a Phase II pro­gram with this kind of in­for­ma­tion al­ready es­tab­lished by Roche,” Quisel said.

“The task left for us at Disc is to demon­strate how much we are ad­dress­ing the un­met need in EPP pa­tients,” added Will Sav­age, who was pro­mot­ed to CMO last week as part of a quar­tet of lead­er­ship changes.

With the new cash in­fu­sion, the team — now stand­ing at 20 — ex­pects to have mul­ti­ple Phase II tri­als for bitop­er­tin and DISC-0974 up and run­ning, then se­cure proof-of-con­cept da­ta in 2023. Then there are the ear­ly-stage dis­cov­ery pro­grams lin­ing be­hind.

Or­biMed led the new round, joined by new in­vestors Ar­ix Bio­science, Janus Hen­der­son In­vestors, 5AM Ven­tures, Rock Springs Cap­i­tal, Nan­ta­ha­la Cap­i­tal Man­age­ment, Wil­lett Ad­vi­sors, and Alexan­dria Ven­ture In­vest­ments. Ex­ist­ing back­ers, in­clud­ing At­las Ven­ture, No­vo Hold­ings and Ac­cess Biotech­nol­o­gy, al­so par­tic­i­pat­ed.

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.

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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Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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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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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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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.

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.

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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Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

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