News brief­ing: 10 years lat­er, teplizum­ab gets a sec­ond shot at an FDA OK; J&J files for ami­van­tam­ab ap­proval in Eu­rope

Teplizum­ab’s 10-year trek back in­to the R&D spot­light is get­ting an­oth­er as­sist from the FDA.

The agency has pro­vid­ed a brief pri­or­i­ty re­view sched­ule for the type 1 di­a­betes drug, ac­cord­ing to Proven­tion Bio. And that sets up a Ju­ly 2 PDU­FA date for a drug that al­ready won the agency’s break­through ther­a­py sta­tus.

Ash­leigh Palmer Proven­tion

Teplizum­ab had lin­gered for years in lim­bo af­ter Eli Lil­ly de­cid­ed to punt on the drug fol­low­ing a piv­otal fail­ure in di­a­betes. But its sci­en­tif­ic dis­cov­er­er, long­time UCSF vet and now biotech en­tre­pre­neur Jef­frey Blue­stone, nev­er lost hope. He found new be­liev­ers at Proven­tion, who picked up rights to the drug and quick­ly made their way back to the FDA.

An ad­vi­so­ry pan­el has been slat­ed to re­view the drug on May 27.

“The FDA’s ac­cep­tance of our BLA rep­re­sents a sig­nif­i­cant achieve­ment for Proven­tion Bio in our mis­sion to de­liv­er the first po­ten­tial dis­ease-mod­i­fy­ing T1D ther­a­py and dri­ve a par­a­digm shift in how in­di­vid­u­als at risk of de­vel­op­ing the dis­ease are treat­ed,” said Proven­tion CEO Ash­leigh Palmer. “We in­tend to work close­ly with the FDA to sup­port their re­view while al­so prepar­ing for a po­ten­tial prod­uct launch in the third quar­ter of 2021.” — John Car­roll

Aim­ing to catch Tagris­so, J&J files for ami­van­tam­ab ap­proval in Eu­rope

Rough­ly one month af­ter J&J’s Janssen filed for an FDA OK for ami­van­tam­ab, the phar­ma is now gun­ning for an­oth­er ap­proval in Eu­rope.

Janssen sub­mit­ted its MAA for the pro­gram to the EMA on Mon­day, seek­ing the green­light for pa­tients with metasta­t­ic non-small cell lung can­cer with EGFR ex­on 20 in­ser­tion mu­ta­tions af­ter chemo. It’s an­oth­er step in Janssen’s quest to take on the ex­per­i­men­tal drug’s biggest ri­val — As­traZeneca’s Tagris­so.

Like the FDA sub­mis­sion, the EMA ap­pli­ca­tion is based on the re­sults from a Phase I study as Janssen is shoot­ing for a quick OK. It’s an at­tempt to out­flank the best­selling Tagris­so, as ami­van­tam­ab would be­come the first med­ica­tion ap­proved for these spe­cif­ic mu­ta­tions.

Tagris­so is thus far ap­proved to tar­get ex­on 19 dele­tions or ex­on 21 L858R mu­ta­tions, and picked up an OK a few weeks ago for pa­tients with EGFR mu­ta­tions who had un­der­gone tu­mor re­sec­tion and op­tion­al, stan­dard post­op­er­a­tive ad­ju­vant chemother­a­py.

J&J is play­ing from be­hind de­spite be­ing grant­ed break­through ther­a­py des­ig­na­tion in March. The phar­ma has stud­ied ami­van­tam­ab both as a monother­a­py and in com­bi­na­tion with an in-house TKI dubbed laz­er­tinib. Ul­ti­mate­ly, J&J is ex­pect­ed to pit the ami­van­tam­ab/laz­er­tinib com­bo head-to-head with Tagris­so in the front­line set­ting for a Phase III tri­al. — Max Gel­man

Chi­na’s Oc­u­men­sion buys a chunk of its US part­ner’s stock

A good Chi­nese part­ner these days doesn’t just help com­mer­cial­ize your drug in Asia — it may al­so be­come an in­vestor in time of need.

Case in point: Wa­ter­town, MA-based Eye­Point Phar­ma­ceu­ti­cals has just scored $15.7 mil­lion from Oc­u­men­sion Ther­a­peu­tics, which is de­vel­op­ing its two ap­proved oc­u­lar drugs, Yu­tiq and Dexy­cu, and ex­pect­ing its first launch­es in Chi­na lat­er this year. Oc­u­men­sion laid low un­til its own pub­lic de­but on the Hong Kong stock ex­change last Ju­ly, break­ing off from the con­trol of the mar­quee in­vestors at 6 Di­men­sions Cap­i­tal.

The in­jec­tion, which buys 3.01 mil­lion shares of Eye­Point stock at $5.22 each, brings its cash and cash equiv­a­lents up to $44 mil­lion, just enough to get through the end of the year.

Aside from the two lead drugs for chron­ic non-in­fec­tious uveitis and post­op­er­a­tive in­flam­ma­tion, Eye­Point is plan­ning a Phase I tri­al for an ex­per­i­men­tal com­pound in wet age-re­lat­ed mac­u­lar de­gen­er­a­tion in the com­ing months. — Am­ber Tong

Up­start from Chris Garabe­di­an’s in­cu­ba­tor hits the clin­ic with gene ther­a­py for a rare ge­net­ic dis­or­der

A few months af­ter its launch out of long­time in­dus­try in­vestor Chris Garabe­di­an’s VC fund, Forge Bi­o­log­ics has se­cured an IND for its Krabbe dis­ease gene ther­a­py can­di­date, FBX-101.

The Colum­bus, OH-based biotech is mov­ing for­ward with a Phase I/II study of the can­di­date, which com­bines an AAV with um­bil­i­cal cord mar­row. GMP man­u­fac­tur­ing is com­plet­ed, ac­cord­ing to Forge, and screen­ing for en­roll­ment will be­gin this month. The com­pa­ny ini­tial­ly said it’s ex­pect­ed to be in the clin­ic by the end of 2020, af­ter snag­ging a $40 mil­lion Se­ries A round in Ju­ly.

Krabbe dis­ease is a rare ge­net­ic dis­or­der that in many cas­es caus­es death by age 2. Pa­tients are born with mu­ta­tions in the galac­to­syl­ce­rami­dase (GALC) gene, which en­codes an en­zyme that helps break down lipid mol­e­cules in­side cells. This leads to the tox­ic buildup of psy­cho­sine, a lipid mol­e­cule that can’t be de­grad­ed in cells, par­tic­u­lar­ly in brain cells and pe­riph­er­al nerves.

“This com­bi­na­tion ap­proach is ex­treme­ly ex­cit­ing be­cause the pre­clin­i­cal da­ta demon­strate sig­nif­i­cant cor­rec­tion of sur­vival, be­hav­ior and neu­ro­mus­cu­lar func­tion in an­i­mal mod­els com­pared to ei­ther trans­plant or AAV treat­ment alone,” said Forge CMO Maria Es­co­lar, who led near­ly 20 years of Krabbe dis­ease re­search. — Nicole De­Feud­is

Cul­li­nan sets terms for $150 mil­lion IPO

Cul­li­nan On­col­o­gy’s New Year’s res­o­lu­tion is a pub­lic de­but — and on Mon­day, the Cam­bridge, MA-based biotech set the terms for a $150 mil­lion raise.

The com­pa­ny is of­fer­ing 8.3 mil­lion shares at a $17 to $19 range. But if 2021 is any­thing like last year, it could stand to raise more. As of Dec. 4, over 80 biotechs had raised a record $14.9 bil­lion in 2020.

Cul­li­nan plans on us­ing $45 mil­lion to $55 mil­lion of IPO pro­ceeds for its lead can­di­date: a small mol­e­cule de­signed as an ir­re­versible EGFR in­hibitor, which is cur­rent­ly in Phase I/IIa for non small cell lung can­cer pa­tients with EGFRex20ins mu­ta­tions. The pipeline al­so in­cludes CLN-049, a bis­pe­cif­ic an­ti­body tar­get­ing FLT3 and CD3 for the treat­ment of AML, and CLN-619, a mon­o­clon­al an­ti­body de­signed to stim­u­late NK and T cell re­spons­es for the treat­ment of sol­id tu­mors. — Nicole De­Feud­is

In­smed aims for full ap­proval of MAC lung dis­ease treat­ment

In­smed has dosed the first pa­tient in a post-mar­ket­ing pro­gram to get full ap­proval for its My­cobac­teri­um avi­um com­plex (MAC) lung dis­ease treat­ment Arikayce.

The drug, amikacin li­po­some in­hala­tion sus­pen­sion, re­ceived ac­cel­er­at­ed ap­proval back in Sep­tem­ber 2018 as part of a com­bi­na­tion an­tibac­te­r­i­al reg­i­men for adults with lim­it­ed or no al­ter­na­tive treat­ment op­tions. The front­line clin­i­cal tri­al pro­gram con­sists of two parts dubbed ARISE and EN­CORE, and is de­signed to ful­fill the FDA’s post-mar­ket­ing re­quire­ment for full ap­proval, and sup­port a sup­ple­men­tal new drug ap­pli­ca­tion as a front­line treat­ment for MAC lung dis­ease.

“We are very pleased to ini­ti­ate the front­line clin­i­cal de­vel­op­ment pro­gram for ARIKAYCE, po­ten­tial­ly lay­ing the ground­work for a new stan­dard of care for pa­tients with MAC lung dis­ease,” In­smed CMO Mar­ti­na Flam­mer said in a state­ment. — Nicole De­Feud­is

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

$DNA is once again on NYSE; FDA clears Soliris chal­lenger for the mar­ket; Flag­ship’s think­ing big again with eR­NA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

I still remember the uncertainty in the air last year when nobody was sure whether ASCO would cancel their in-person meeting. But it’s now back again for the second virtual conference, and Endpoints News is here for it. Check out our 2-day event reviewing the landscape of cancer R&D and send news our way.

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Gene ther­a­py from Bio­gen's $800M buy­out flops in mid-stage study, deal­ing blow to new am­bi­tions

The #2 candidate from Biogen’s $800 million ocular gene therapy buyout has failed in a mid-stage trial, dealing an early blow to the big biotech’s plans to revitalize its pipeline with new technologies.

Biogen announced that the candidate, an experimental treatment for a rare and progressive form of blindness called X-linked retinitis pigmentosa (XLRP), failed to sufficiently improve vision in patients’ treated eye — patients only received an injection in one eye — after a year, on a standard scale, compared to their untreated eye. The company said they saw “positive trends” on several secondary endpoints, including visual acuity, but declined to say whether the trial actually hit any of those endpoints.

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Vas Narasimhan (Photographer: Simon Dawson/Bloomberg via Getty Images)

No­var­tis whiffs on En­tresto study af­ter heart at­tacks — but that does­n't mean it's go­ing down qui­et­ly

If Novartis learned one thing from its interaction with the FDA over its latest heart failure approval for Entresto, it was that missing a primary endpoint may not be the nail in the coffin. Now, Entresto has missed again on a late-stage study in high-risk heart patients, and it’s already sowing the seeds for a path forward regardless.

Novartis’ Entresto couldn’t best standard-of-care ramipril in staving off a composite of deaths and heart failure events in patients with left ventricular systolic dysfunction and/or pulmonary congestion who have had a prior heart attack, according to topline data from the Phase III PARADISE-MI study revealed Saturday at the virtual American College of Cardiology meeting.

Michael Dell (Richard Drew, AP Images)

'Dude, you're get­ting a Del­l' — as a new deep-pock­et biotech in­vestor

What happens when you marry longtime insiders in the global biotech VC game with the family fund of tech billionaire Michael Dell, a synthetic biology legend out of MIT and Harvard and the former director of the NCI?

Today, the answer is a newly financed, $200 million biotech SPAC now cruising the industry for a top player interested in finding a short cut to Nasdaq.

Orion Biotech Opportunities priced their blank check company today, raising $200 million with Dell’s multibillion-dollar MSD group’s commitment on investing another $20 million in a forward-purchase agreement.

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BAR­DA slows its $9B en­gine for new Covid-19 ther­a­peu­tics

The Biomedical Advanced Research and Development Authority is cooling its jets in looking for new, potential Covid-19 treatments, at least in the near term.

An HHS spokesperson told Endpoints News via email, “to date, BARDA has obligated more than $9 billion for the development and/or purchase of 13 therapeutics, beginning in February 2020 with support to develop Regeneron’s monoclonal antibody therapeutic. Therapeutics are an important element of the COVID-19 response, and we are focused on the programs currently underway and/or in negotiation using the funds available to us.”

Scynex­is takes out $60M loan ahead of ex­pect­ed ap­proval; Com­pass Ther­a­peu­tics ac­quires new bis­pe­cif­ic an­ti­body in buy­out

As the PDUFA date for their vaginal yeast infections quickly approaches, Scynexis is taking out a loan to ensure it can hit the ground running for a potential commercial launch.

Scynexis closed terms on a $60 million loan with Hercules Capital and Silicon Valley Bank, the New Jersey biotech announced Friday, with its ibrexafungerp candidate set for a June 1 PDUFA. Approval of the program is anticipated, the company said, after which the oral antifungal will be branded as Brexafemme.

Bris­tol My­ers backs up its case for heart drug mava­camten as FDA weighs app in car­diomy­opa­thy

When Bristol Myers Squibb signed off on its $13 billion acquisition of MyoKardia back in October, it was making a big bet that lead drug mavacamten could prove a game changer in cardiac myopathy. Now, with the drug up for FDA review, Bristol Myers is backing up its case with new quality of life data.

Patients dosed with myosin inhibitor mavacamten posted a clinically significant increase in scores on the Kansas City Cardiomyopathy Questionnaire, a catch-all summary of symptoms and quality of life markers, over placebo at 30 weeks, according to data from the Phase III EXPLORER-HCM study presented Saturday at the virtual American College of Cardiology meeting.

UP­DAT­ED: Apel­lis bags FDA nod for Soliris chal­lenger with a dif­fer­ent path­way to PNH — but can it slay the gi­ant?

With a blockbuster rare disease giant in its sights in Alexion’s Soliris, small biotech Apellis has reason to think its competitor is worthy of the spotlight. Now, with the FDA on its side, Apellis will get its chance to be the David to Alexion’s Goliath.

The FDA on Friday approved Empaveli (pegcetacoplan), a C3 complement inhibitor the biotech thinks can prove a worthy challenger to Alexion’s C5 inhibitors Soliris and follow-up drug Ultomiris in rare disease paroxysmal nocturnal hemoglobinuria (PNH).