Per­lara col­lab­o­rates with Har­vard/Un­di­ag­nosed Dis­eases Net­work; Verona shares soar on pos­i­tive CF da­ta

→ Per­lara, the rare dis­ease com­pa­ny found­ed by Twit­ter’s fa­vorite biotech rebel Ethan Perl­stein, an­nounced two sci­en­tif­ic dis­cov­ery col­lab­o­ra­tions Fri­day with Har­vard Med­ical School and the Un­di­ag­nosed Dis­eases Net­work. The re­search part­ner­ships are for two rare mono­genic neu­rode­vel­op­men­tal dis­or­ders. One is caused by a mu­ta­tion in GNA01 and the oth­er caused by a mu­ta­tion in RPS6KA3 (Cof­fin-Lowry Syn­drome). Per­lara plans to de­vel­op and val­i­date ne­ma­tode and fly “pa­tient avatars” of path­o­gen­ic GNA01 and RPS6KA3 mu­ta­tions for use in high-through­put phe­no­typ­ic drug screens. Perl­stein, CEO of the Per­lara, is best known in the bio­phar­ma world through his out­spo­ken Twit­ter pres­ence, where he’s laid bare the dif­fi­cul­ty of rais­ing cap­i­tal as a young com­pa­ny work­ing on “dis­eases thought too rare to at­tract the in­ter­est of phar­ma­ceu­ti­cal com­pa­nies.” He’s been called rad­i­cal for his trans­paren­cy, and a rebel for chal­leng­ing fundrais­ing norms on a pub­lic stage. In lieu of sig­nif­i­cant ven­ture cap­i­tal funds, Perl­stein has in­stead re­lied heav­i­ly on part­ner­ships with pa­tient groups since the com­pa­ny’s 2014 in­cep­tion. Last year, how­ev­er, the com­pa­ny did raise a $7.4M Se­ries A.

→  Shares of Verona Phar­ma shot up 25% $VR­NA on Fri­day af­ter the Lon­don-based biotech tout­ed Phase IIa da­ta for its cys­tic fi­bro­sis ther­a­py RPL554. Pa­tients in the small study achieved a 6% sus­tained im­prove­ment in FEV1, ac­cord­ing to re­searchers, af­ter a sin­gle dose. The com­pa­ny de­scribes the drug as a “dual in­hibitor of the en­zymes phos­pho­di­esterase 3 and 4 that acts as both a bron­chodila­tor and an an­ti-in­flam­ma­to­ry agent in a sin­gle com­pound.”

→ The FDA has ac­cept­ed for re­view Re­gen­eron $REGN and its part­ner Sanofi’s $SNY mar­ket­ing ap­pli­ca­tion for Dupix­ent (dupilum­ab). The duo is ask­ing for the OK to sell the drug as an add-on main­te­nance treat­ment in cer­tain adults and ado­les­cents at least 12 years old with mod­er­ate-to-se­vere asth­ma. The ap­pli­ca­tion is backed by clin­i­cal da­ta from 2,888 adults and ado­les­cents from three piv­otal tri­als, the com­pa­nies said in a state­ment. The ac­tion date is set for Oc­to­ber 20. Dupix­ent, a ful­ly hu­man mon­o­clon­al an­ti­body, is al­ready ap­proved in the US for atopic der­mati­tis.

Sev­en plucky di­ag­nos­tics com­pa­nies win a $249M round of con­tracts af­ter sur­viv­ing NI­H's Covid-19 'Shark Tank' com­pe­ti­tion

As US Covid-19 deaths creep past 150,000 and officials stress the importance of contact tracing, the NIH’s Rapid Acceleration of Diagnostics (RADx) program has inked contracts totaling $248.7 million to expand testing capabilities.

The seven contracts, which were chosen “Shark Tank”-style from a pool of 100 proposals, are part of an effort to bump daily testing capacity to 2% of the country’s population by late summer or fall. That would be about 6 million people per day, compared to the current 520,000 to 823,000 tests being administered daily.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

Endpoints Premium

Premium subscription required

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

Covid-19 roundup: Eli Lil­ly retro­fits RVs for first-of-its-kind an­ti­body tri­al with NIH

Eli Lilly and the NIH are about to start a first-of-its-kind trial that researchers and developers have talked about for months as a way of providing temporary immunity to the most at-risk populations.

Lilly announced this morning that it will start a 2,400-person trial with the National Institute for Allergy and Infectious Diseases to test whether its experimental Covid-19 neutralizing antibody can prevent people in nursing homes and assisted living facilities from developing the disease. The idea, known as passive immunity, is that rather than waiting on a vaccine to induce people to develop antibodies, doctors can give them lab-grown antibodies. Ideally, those antibodies will either attack the new SARS-CoV-2 infection, if the patient has recently been exposed, or persist in the blood for several weeks and prevent infection or disease for that period.

Im­mu­nic's lead MS drug hits pri­ma­ry and key sec­ondary end­points in PhII, but ques­tions re­main

Just a week after its lead program began enrolling patients in a study to treat Covid-19, Immunic Therapeutics is making more waves.

This time, the biotech is providing a glimpse at topline data from a Phase II trial studying the efficacy of vidofludimus calcium, or IMU-838, in relapsing-remitting multiple sclerosis patients. Taken orally, the candidate met its primary endpoint in reducing the cumulative number of combined unique active MRI lesions after 24 weeks for a 45 mg dose compared to a placebo, as well as a key secondary endpoint in such reductions for the 30 mg dose.

FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

Frank Zhang (AP Images)

CAR-T fil­ing in sight, Frank Zhang grabs full con­trol of J&J-part­nered Leg­end Biotech, steps down from Gen­Script

Two months after Yuan Xu steered Legend Biotech to a $424 million public debut on the Nasdaq, founder and chairman Frank Zhang is grabbing the reins as CEO.

In conjunction with the move, Zhang is also stepping down from the helm of GenScript — a position he’s held for 18 years. GenScript, a Hong Kong-listed CRO, hatched Legend as a subsidiary in 2015 before spinning it out, and remains a majority shareholder.

Elizabeth Nabel speaks at a news conference, Oct. 7, 2019 (Elise Amendola/AP Images)

Brigham and Wom­en's pres­i­dent Eliz­a­beth Nabel fol­lows Mon­cef Slaoui off Mod­er­na's board

Amid recent scrutiny on how Moderna’s top executives have been cashing out their increasingly valuable shares, the biotech is parting ways with a board member who’s also heading a hospital where its Covid-19 vaccine is being tested.

Elizabeth Nabel — the president of Brigham and Women’s Hospital — has followed in Moncef Slaoui’s footsteps in resigning from Moderna’s board of directors. She took the role in 2015, two years before the Operation Warp Speed leader did; and as with Slaoui and MIT professor Robert Langer, her term was due to expire in 2021.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,300+ biopharma pros reading Endpoints daily — and it's free.

Tony Coles, Cerevel Therapeutics CEO

Adding $445M, Tony Coles and his big Pfiz­er neu­ro spin­out hitch a ride to Wall Street on Per­cep­tive’s SPAC

Two years ago, after Pfizer abruptly shut down its entire neuroscience division, Bain Capital bet $350 million that those assets were still worth something and packaged them into a new biotech: Cerevel Therapeutics. A year later, they got seasoned executive Tony Coles, who had recently jumped back into the C-suite of another neuroscience startup, to run the company.

Now Coles is steering Cerevel public, in what he says is the largest ever transaction of its kind. Cerevel has agreed to merge with Perceptive Advisors’ specialty acquisition company ARYA II. Between the roughly $125 million Perceptive raised through ARYA and an additional investment of $320 million Bain Capital, Perceptive and — yes, really — Pfizer, among others, Cerevel will now move forward with an added $445 million in its coffers.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,300+ biopharma pros reading Endpoints daily — and it's free.

Roche de­clares a PhI­II fail­ure for Covid-19 as the IL-6 re­pur­pos­ing the­o­ry bites the dust

Another big IL-6 drug has failed to move the needle for Covid-19 patients, leaving that particular field of repurposed drug R&D on the ropes for the pandemic.

This morning it was Roche’s turn to outline a Phase III failure for Actemra, adding compelling data that have now all but extinguished the theory that an IL-6 drug could significantly help the most severely afflicted patients. That comes just weeks after Regeneron and Sanofi hit the red light on their trial for Kevzara after getting back-to-back readouts that made Roche’s trial a long shot at best.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 86,300+ biopharma pros reading Endpoints daily — and it's free.