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.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

Abortion-rights protesters regroup and protest following Supreme Court's decision to overturn Roe v. Wade. (AP Photo/Gemunu Amarasinghe)

Fol­low­ing SCO­TUS de­ci­sion to over­turn abor­tion pro­tec­tions, AG Gar­land says states can't ban the abor­tion pill

Following the Supreme Court’s historic decision on Friday to overturn Americans’ constitutional right to an abortion after almost 50 years, Attorney General Merrick Garland sought to somewhat reassure women that states will not be able to ban the prescription drug sometimes used for abortions.

Following the decision, the New England Journal of Medicine also published an editorial strongly condemning the reversal, saying it “serves American families poorly, putting their health, safety, finances, and futures at risk.”

Yong Dai, Frontera Therapeutics CEO

Scoop: Lit­tle-known Or­biMed-backed biotech clos­es $160M round to start gene ther­a­py tri­al

Frontera Therapeutics, a China and US biotech, has closed a $160 million Series B and received regulatory clearance to test its first gene therapy stateside, Endpoints News has learned.

Led by the largest shareholder, OrbiMed, the biotech has secured $195 million total since its September 2019 founding, according to an email reviewed by Endpoints. The lead AAV gene therapy program is for an undisclosed rare eye disease, according to the source.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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DEM BioPharma CEO David Donabedian (L) and executive chair Jan Skvarka

Long­wood sets an­oth­er 'don't eat me' biotech in­to gear with help of for­mer Tril­li­um CEO Jan Skvar­ka

Jonathan Weissman and team are out with a cancer-fighting biotech riding the appetite for those so-called “don’t eat me” and “eat me” signals.

The scientific co-founder — alongside fellow Whitehead Institute colleague Kipp Weiskopf and Stanford biologist Michael Bassik — has launched DEM BioPharma with incubator Longwood Fund and a crop of other investors.

In all, the nascent, 10-employee biotech has $70 million to bankroll hematology- and solid tumor-based programs, including a lead asset that could enter human trials in two to three years, CEO David Donabedian told Endpoints News.

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Spanish Prime Minister Pédro Sanchez and European Commission President Ursula von der Leyen (AP Photo/Geert Vanden Wijngaert)

EU to launch vac­cine de­vel­op­ment and man­u­fac­tur­ing part­ner­ship with Latin Amer­i­can and Caribbean coun­tries

While European companies, including BioNTech, are focused on increasing vaccine access to African countries by setting up vaccine manufacturing facilities, the European Union is looking westward to Latin America and the Caribbean.

Speaking at a press conference with Spanish Prime Minister Pédro Sanchez, EU Commission president Ursula von der Leyen said that the EU is launching a new initiative for vaccines and medicines manufacturing in Latin America, to get drugs to Latin America and the Caribbean faster.

De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Kelly Martin, Radius Health CEO

VC firms take os­teo­poro­sis drug­mak­er Ra­dius Health pri­vate for al­most $900M

After attacks from activist investors and disappointing returns on share prices, Radius Health has now agreed to new ownership, a direction resulting in leaving the Nasdaq.

Radius Health, a biotech out of Massachusetts with one approved product in its arsenal, announced Thursday morning that it agreed to be acquired by two VC firms: Gurnet Point Capital and Patient Square Capital. The deal, worth around $890 million, will include debt assumption and the payout of $1 CVR per share for investors. And on top of that, OrbiMed is providing debt financing.