Im­muno­core's bis­pe­cif­ic lands speedy re­view with FDA, EMA; Low-pro­file gene ther­a­py play­er grabs $30M

UK TCR-fo­cused biotech Im­muno­core an­nounced that it scored a pri­or­i­ty re­view with the FDA for lead drug tebenta­fusp.

The bis­pe­cif­ic al­ready boasts a break­through ther­a­py des­ig­na­tion thanks to pos­i­tive Phase III da­ta. The pri­or­i­ty re­view shaves four months off the reg­u­lar 10-month process, lead­ing to an ex­pect­ed PDU­FA date of Feb. 23.

Across the At­lantic, the EMA agreed to ac­cel­er­ate its as­sess­ment of Im­muno­core’s mar­ket­ing au­tho­riza­tion ap­pli­ca­tion, which it has al­ready ac­cept­ed. That ac­cel­er­at­ed time­line re­duces the as­sess­ment process from up to 210 days to 150 days, ex­clud­ing clock stops.

Bahi­ja Jal­lal, CEO of Im­muno­core, said in a re­lease: “There is an ur­gent need for an ap­proved treat­ment for metasta­t­ic uveal melanoma, an ag­gres­sive form of can­cer for which there are very lim­it­ed treat­ment op­tions.” — Paul Schloess­er

Xalud Ther­a­peu­tics nabs $30M to push non-vi­ral gene ther­a­py in in­flam­ma­tion

Xalud Ther­a­peu­tics an­nounced that it fin­ished a round of Se­ries C fi­nanc­ing with $30 mil­lion raised. Ac­cord­ing to CEO Diem Nguyen, “We are well po­si­tioned to con­tin­ue ad­vanc­ing XT-150, our lead ther­a­peu­tic can­di­date, through clin­i­cal de­vel­op­ment and fur­ther ex­pand our plat­form.”

Nguyen, who took on the role of CEO in Jan­u­ary, has led a com­pa­ny that has op­er­at­ed un­der a very low pro­file. Very lit­tle in­for­ma­tion about the com­pa­ny be­fore Nguyen took the helm is pub­licly avail­able.

The NYC-based biotech has spent a sig­nif­i­cant amount of its re­sources on XT-150, a lo­cal­ly in­jectable, non-vi­ral gene ther­a­py that tar­gets chron­ic in­flam­ma­tion. That ther­a­py is cur­rent­ly in a Phase IIb tri­al for os­teoarthri­tis of the knee and a Phase I/IIa clin­i­cal study for pe­riph­er­al neu­ro­path­ic pain. — Paul Schloess­er

FDA ex­pands la­bel for J&J’s Xarel­to 

J&J sub­sidiary Janssen Phar­ma­ceu­ti­cals has the FDA’s bless­ing for an ex­pand­ed pe­riph­er­al artery dis­ease (PAD) in­di­ca­tion for Xarel­to plus as­pirin to in­clude pa­tients who have re­ceived re­cent low­er-ex­trem­i­ty revas­cu­lar­iza­tion (LER) due to symp­to­matic PAD. FDA’s ap­proval is based on da­ta from a Phase III study known as VOY­AGER PAD.

That study showed that when Xarel­to and as­pirin are com­bined, pa­tients with symp­to­matic PAD and LER had a 15% de­creased risk in ma­jor ad­verse limb and car­dio­vas­cu­lar events.

With this ap­proval, Xarel­to, a blood thin­ner, brings its num­ber of in­di­ca­tions to nine.

“We’re thrilled to bring Xarel­to to even more pa­tients with PAD who have been liv­ing for two decades with­out any new in­no­va­tion in the an­tithrom­bot­ic space,” said James List, glob­al ther­a­peu­tic area head, car­dio­vas­cu­lar and me­tab­o­lism of Janssen R&D. — Paul Schloess­er

Lux Cap­i­tal leads $50M in­fu­sion in­to rare dis­ease re­search plat­form

All­Stripes has some new mon­ey to play around with.

The San Fran­cis­co-based com­pa­ny an­nounced a new $50 mil­lion Se­ries B round Tues­day morn­ing, led by Lux Cap­i­tal. All­Stripes said the cash will sup­port launch­ing 100 new rare dis­ease re­search pro­grams while ex­pand­ing its glob­al op­er­a­tional foot­print.

“Be­gin­ning re­search on a rare con­di­tion can feel like be­ing dropped in­to a new world with­out a map and we are on a mis­sion to change that with da­ta,” CEO Nan­cy Yu said in a state­ment. “This in­vest­ment will al­low us to bet­ter sup­port the rare dis­ease com­mu­ni­ty, where each per­son’s ex­pe­ri­ence is es­sen­tial to un­der­stand­ing dis­ease pro­gres­sion.”

In ad­di­tion to the re­search ex­pan­sion, All­Stripes al­so plans to build out its tech­nol­o­gy and da­ta au­toma­tion to im­prove re­search in­sights, which will fur­ther de­vel­op its plat­form ca­pa­bil­i­ties. That will help the biotech con­tin­ue to in­vest in grow­ing the com­pa­ny’s team to sup­port cre­at­ing the play­book for rare dis­ease re­search, the biotech said.

All­Stripes is a pub­lic ben­e­fit cor­po­ra­tion that ad­vo­cates for more re­al-world ev­i­dence while treat­ments are be­ing de­vel­oped. It aims to build tools that make re­search more in­clu­sive for the glob­al rare dis­ease com­mu­ni­ty. Cur­rent­ly, the com­pa­ny is part­nered with 30 pa­tient ad­vo­ca­cy groups. — Max Gel­man

Catch­ing up with gi­ants, Chi­na’s Rei­s­tone touts PhII alope­cia area­ta for JAK1 in­hibitor

A Shang­hai biotech is join­ing Eli Lil­ly, Pfiz­er and Con­cert Phar­ma and oth­ers in the rush to bring JAK in­hibitors in­to alope­cia area­ta.

In a Phase II study, Rei­s­tone Bio­phar­ma re­ports, its oral JAK1 block­er SHR0302 met the pri­ma­ry end­point of spurring sta­tis­ti­cal­ly sig­nif­i­cant and clin­i­cal­ly rel­e­vant per­cent­age change of SALT score — a mea­sure of hair loss — ver­sus place­bo.

Re­cruit­ed across Aus­tralia, Chi­na and the US, 94 pa­tients en­tered the tri­al with 25% or greater scalp hair loss and were ran­dom­ized to one of four groups: 8mg drug, 4mg, 2 mg or place­bo. The high and mid dos­es met the pri­ma­ry end­point as well as the sec­ondary end­point of ab­solute change in SALT score at week 24.

Rei­s­tone first li­censed SHR0302 from Jiang­su Hen­grui.

CEO Min Ir­win calls the re­sults “en­cour­ag­ing” and says Phase III plan­ning is un­der­way.

Clear­ly cog­nizant of the safe­ty con­cerns that have plagued the class, the biotech high­light­ed that “safe­ty of SHR0302 was con­sis­tent with es­tab­lished pro­files from pre­vi­ous stud­ies of oth­er JAK class of med­ica­tion and was well tol­er­at­ed with no ma­jor ad­verse car­dio­vas­cu­lar events (MACE), deaths, or ve­nous throm­boem­bol­ic events (VTEs) re­port­ed from the study.”

But it’s far from the on­ly one mak­ing those claims, as a slate of play­ers clam­or for a stake in this au­toim­mune dis­ease. Lil­ly, in par­tic­u­lar, is lin­ing up its NDA any day now fol­low­ing a break­through ther­a­py des­ig­na­tion. — Am­ber Tong

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.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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

Up­dat­ed: 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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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.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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Bo Cumbo, new Solid Bio CEO

Sol­id Bio gets a new CEO, $75M cash and drops lead drug as big in­vestors ju­ry-rig a merg­er deal

Three months after Endpoints News broke the story that gene therapy outfit AavantiBio had gutted its CMC group in a reorganization, the biotech’s CEO has helped engineer a merger with struggling penny stock player Solid Bio. And he’s going to remain in charge of the combined operation, as Solid founder Ilan Ganot steps aside.

The merger news this morning features some high-profile investors.

Perceptive Advisors, RA Capital Management and Bain Capital Life Sciences are leading a $75 million raise to add to the pool of cash Solid will have after the tie-up. That will leave Solid $215 million in cash as Bain’s Adam Koppel jumps on the board — enough to pay for ops and get through some key data milestones on their way into 2025.

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