LLS backs 5 new can­cer drug projects with up to $50M; Trodelvy con­tin­ues to im­press with more TNBC da­ta

The Leukemia and Lym­phoma So­ci­ety has tapped 5 new ear­ly-stage projects to back with up to $10 mil­lion each in fresh in­vest­ments. The 5 biotechs are:

Rachel Hau­r­witz

— Cari­bou, head­ed by Rachel Hau­r­witz and co-found­ed by Jen­nifer Doud­na, is work­ing on next-gen, off-the-shelf CAR-Ts to re­place the pa­tient-de­rived cells now in use.

— The LLS sup­port­ed Nex­Im­mune’s IPO, help­ing fund its work on nanopar­ti­cles that can gin up an im­mune re­sponse di­rect­ed at can­cer cells. The biotech has 2 projects now in Phase I tri­als.

— Im­mune-Onc Ther­a­peu­tics is get­ting help with their ear­ly work on IO-202, “a first-in-class an­ti­body tar­get­ing the im­mune in­hibito­ry re­cep­tor Leuko­cyte Im­munoglob­u­lin-Like Re­cep­tor sub­fam­i­ly B (LIL­RB) mem­ber 4 (LIL­RB4, al­so knowns ILT3).”

— Saar Gill’s Caris­ma, spun out of Penn, is get­ting help with its CAR-en­gi­neered macrophages.

— Abin­tus Bio is get­ting fi­nan­cial sup­port for its in vi­vo-gen­er­at­ed CARs that avoid ex­tract­ing cells, en­gi­neer­ing them and then trans­fus­ing them, back in­to pa­tients.

Hau­r­witz had this to say:

“We’re par­tic­u­lar­ly ex­cit­ed to be work­ing with LLS TAP be­cause it gives us ac­cess to their deep knowl­edge of blood can­cer and their net­work of pa­tients and drug de­vel­op­ment ex­perts.” — John Car­roll

Trodelvy con­tin­ues to im­press with more TNBC da­ta

The drug be­hind Gilead’s $21 bil­lion ac­qui­si­tion of Im­munomedics last Sep­tem­ber con­tin­ues to post good da­ta re­sults from its con­fir­ma­to­ry Phase III study.

In a pa­per pub­lished in the New Eng­land Jour­nal of Med­i­cine on Thurs­day, the drug Trodelvy se­cured a sta­tis­ti­cal­ly sig­nif­i­cant pro­gres­sion-free sur­vival and over­all sur­vival in pa­tients with re­lapsed or re­frac­to­ry metasta­t­ic triple-neg­a­tive breast can­cer. Me­di­an PFS was 5.6 months com­pared to 1.7 months on chemo, and me­di­an OS was 12.1 months ver­sus chemo’s 6.7 months.

Mer­dad Parsey

Trodelvy al­so record­ed an ob­jec­tive re­sponse rate of 35%, com­pared to just 5% on chemother­a­py.

“For a pa­tient pop­u­la­tion whose can­cer is par­tic­u­lar­ly dif­fi­cult to treat, these re­sults are ex­treme­ly en­cour­ag­ing and re­in­force the sig­nif­i­cant treat­ment ad­vance that Trodelvy rep­re­sents for pa­tients with pre­vi­ous­ly treat­ed un­re­sectable lo­cal­ly ad­vanced or metasta­t­ic TNBC,” Gilead CMO Mer­dad Parsey said in a state­ment.

Just a few weeks ago, Trodelvy se­cured a full FDA ap­proval with a broad­er la­bel than its ac­cel­er­at­ed OK in­di­ca­tion, ex­pand­ing the pa­tient pop­u­la­tion to in­clude those with un­re­sectable lo­cal­ly ad­vanced TNBC rather than just metasta­t­ic cas­es. Pa­tients must have re­ceived two or more pri­or sys­temic ther­a­pies, at least one of them for metasta­t­ic dis­ease.

The heart of Gilead’s Im­munomedics buy­out, Trodelvy came to fruition af­ter Im­munomedics worked on de­vel­op­ing the drug for about 37 years. The drug’s da­ta were so im­pres­sive at last sum­mer’s ES­MO that Gilead CEO Dan O’Day an­gled to get a deal done be­fore the con­fer­ence. — Max Gel­man

Chi­na-based mR­NA spin­off rais­es $10M as Covid-19 vac­cine IND looms

Sir­naomics’ mR­NA spin­off RNAim­mune has gath­ered a $10 mil­lion seed round to con­tin­ue build­ing out its plat­form, with a fo­cus on in­fec­tious dis­eases, can­cer and rare dis­eases.

Smooth Riv­er and Hong Kong Hon­grun led the in­vest­ment, with par­tic­i­pa­tion from Shang­hai Wal­ga Biotech­nol­o­gy, High For­est In­vest­ment and Ter­ra Mag­num Sig­ma.

Found­ed in ear­ly 2020, the lead pro­gram is for an­oth­er mR­NA vac­cine point­ed against Covid-19. As J&J and As­traZeneca run in­to in­creased flak over safe­ty fears for their vac­cines, an­a­lysts have be­gun won­der­ing how the rest of the mar­ket will play out as Pfiz­er/BioN­Tech and Mod­er­na dom­i­nate the field. That still may leave op­por­tu­ni­ties ahead, and the fledg­ling start­up sees a shot at get­ting in­to the sec­ond wave.

Patrick Lu

The biotech has an R&D cen­ter in Chi­na and raised $2.35 mil­lion in its orig­i­nal fund­ing round. An IND for Covid is planned lat­er in the year with an­oth­er pro­gram in the works for a RAS tu­mor vac­cine.

Patrick Lu, chair­man of the board of RNAim­mune, said:

“The re­cent suc­cess of Mod­er­na’s and BioN­Tech’s mR­NA vac­cines has demon­strat­ed that mR­NA tech­nolo­gies for de­vel­op­ment of nov­el ther­a­peu­tics and vac­cines are dra­mat­i­cal­ly chang­ing the land­scape of the glob­al phar­ma­ceu­ti­cal in­dus­try. RNAim­mune has tremen­dous op­por­tu­ni­ties as a glob­al mR­NA in­no­vat­ing com­pa­ny.” — John Car­roll

Ac­ne treat­ment from Bausch’s spin­out Or­tho hits all 3 end­points

In a sec­ond Phase III tri­al, Or­tho Der­ma­to­log­ics’ lat­est ac­ne treat­ment showed a ben­e­fit of pa­tients as ear­ly as 2 weeks in­to us­age, the Bausch Health spin­out an­nounced Thurs­day.

The tri­al test­ed IDP-126, a com­bi­na­tion retinoid, an­ti-bac­te­r­i­al and an­tibi­ot­ic top­i­cal to treat ac­ne vul­garis in pa­tients. If the treat­ment is ap­proved, it would be the first to use this triple com­bi­na­tion, the re­lease said.

The study achieved all 3 of its end­points at week 12, in­clud­ing a change in in­flam­ma­to­ry and non­in­flam­ma­to­ry le­sion counts with a p-val­ue of less than 0.001. Treat­ment suc­cess was 50.5% and 20.5% for IDP-126 and its place­bo, re­spec­tive­ly.

For 12 weeks, the 193 pa­tients ages 9 and up ap­plied ei­ther IDP-126 or a place­bo to their face once a day in 13 lo­ca­tions in the US.

Or­tho hopes to sub­mit a new drug ap­pli­ca­tion with the FDA in the lat­ter half of 2022, pres­i­dent Scott Hirsch said in the state­ment. — Josh Sul­li­van

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

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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).