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.

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.

Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

BREAK­ING: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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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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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.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

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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Marcelo Bigal, Ventus Therapeutics CEO

No­vo Nordisk joins No­var­tis, Roche in NL­RP3 are­na, bet­ting $70M cash on NASH, car­diometa­bol­ic us­es

As a drug target, the NLRP3 inflammasome has drawn serious interest from Big Pharma, inspiring a series of M&A deals from Novartis and Roche on top of venture investments by others. Now Novo Nordisk is jumping on the bandwagon — and the Danish pharma giant is taking the target where it knows best.

Novo Nordisk is getting its NLRP3 inhibitors from Ventus Therapeutics, a Versant-backed startup that set out to make some of the best NLRP3 drugs out there by incorporating new insights into the structure of the target complex.

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