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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a rather narrow market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,700+ biopharma pros reading Endpoints daily — and it's free.

FDA hands ac­cel­er­at­ed nod to Seagen, Gen­mab's so­lo ADC in cer­vi­cal can­cer, but com­bo stud­ies look even more promis­ing

Biopharma’s resident antibody-drug conjugate expert Seagen has scored a clutch of oncology approvals in recent years, finding gold in what are known as “third-gen” ADCs. Now, another of their partnered conjugates is ready for prime time.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,700+ biopharma pros reading Endpoints daily — and it's free.

Time for round 2: Il­lu­mi­na-backed VC snags $325M for its next fund

Illumina Ventures closed off its second investment fund with a total commitment of $325 million, offering fresh fuel to back a slate of startups that have already included a smorgasbord of companies, covering everything from diagnostics to biotech drug development and genomics.

Fund II brings the total investment under Illumina Ventures’ oversight to $560 million, which has been focused on early-stage companies. And it has a transatlantic portfolio that includes SQZ, Twist and Encoded Therapeutics.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,700+ biopharma pros reading Endpoints daily — and it's free.