David Chang (Jeff Rumans for Endpoints News)

Al­lo­gene, Over­land plot speedy Asia play for off-the-shelf CAR-T with $117M joint ven­ture

David Chang got a good look at what it takes to bring CAR-T ther­a­py to Chi­na and Asia mar­kets when he and Arie Bellde­grun struck a deal with Fo­s­un Phar­ma to form a joint ven­ture around its pi­o­neer­ing lym­phoma treat­ment — which would come to be known as Yescar­ta — in 2017, just af­ter sub­mit­ting a BLA to the FDA.

With Al­lo­gene, the duo’s high pro­file start­up aimed at de­liv­er­ing off-the-shelf CAR-T, he’s do­ing the same.

“With cell ther­a­py, there is a con­tin­u­ous ef­fort to em­ploy the next gen­er­a­tion tech­nol­o­gy and re­fine what we’re do­ing,” Chang told End­points News, ex­plain­ing his reser­va­tions on a sim­ple out-li­cens­ing mod­el.

Hua Mu

Al­lo­gene’s part­ner of choice is Over­land Phar­ma­ceu­ti­cals, the well-heeled Hill­house start­up that en­tered the stage just days ago and al­ready un­veiled a sim­i­lar deal with ADC Ther­a­peu­tics. Over­land is pour­ing $117 mil­lion in­to Al­lo­gene Over­land Bio­pharm, $40 mil­lion in the form of an up­front to Al­lo­gene and the rest in­ject­ed straight in­to JV op­er­a­tions.

Con­trast that with the $20 mil­lion that Fo­s­un com­mit­ted to set up shop with Kite, and you can de­duce just how far the cell ther­a­py space in Chi­na has come.

Hua Mu, in­ter­im CEO and CMO of Over­land, would know. Dur­ing his stint at WuXi AppTec be­fore even­tu­al­ly land­ing a ven­ture part­ner role at Hill­house, he was in­volved in the dis­cus­sions with Juno that even­tu­al­ly gave birth to JW Ther­a­peu­tics — and de­spite the promis­ing clin­i­cal da­ta and clear need, both sides had ques­tions. Juno made it clear they wouldn’t want to get in un­less Chi­na start­ed reg­u­lat­ing CAR-T as a drug rather than med­ical tech­nol­o­gy; WuXi wasn’t en­tire­ly sure cell ther­a­py is a vi­able class of med­i­cine.

Af­ter a sweep­ing reg­u­la­to­ry re­form and a cou­ple of FDA ap­provals lat­er, the ques­tions have now changed. The lead pro­grams at Fo­s­un Kite and JW are both un­der re­view in Chi­na with OKs ex­pect­ed im­mi­nent­ly. It’s a mat­ter of who can get it and how fast.

“So there’s no doubt about cell ther­a­py as a vi­able new ther­a­py,” Mu said. “The ques­tion is how to make it bet­ter, how to make it more ap­plic­a­ble, how to make it more use­ful to ad­dress the un­met med­ical need.”

Ed Zhang

The promise of en­gi­neer­ing healthy donor cells to re­place the first-gen­er­a­tion, per­son­al­ized ap­proach ap­peared to of­fer the an­swer. Lin­ger­ing un­cer­tain­ty re­gard­ing cell ther­a­py im­por­ta­tion means it will take time to sort out how pre­cise­ly to get the drug ma­te­r­i­al to pa­tients, but the part­ners are al­so start­ing at an ear­li­er stage of de­vel­op­ment.

Since Al­lo­gene’s lead pro­gram in BC­MA has just gen­er­at­ed ini­tial Phase I da­ta, Chang still sees op­por­tu­ni­ties to in­clude come Chi­nese pa­tients in its piv­otal study down the line. Aside from that, it’s hand­ed over three oth­er cell ther­a­py con­structs tar­get­ing CD70, FLT3 and DLL3 to treat a num­ber of both hema­to­log­ic and sol­id tu­mors.

Over­land has al­so se­cured rights in Tai­wan, South Ko­rea and Sin­ga­pore, giv­ing the com­pa­ny plen­ty to work on while talk­ing to reg­u­la­tors and ex­plor­ing lo­cal man­u­fac­tur­ing in Chi­na.

Hill­house’s pres­ence in the re­gion, span­ning hos­pi­tal net­works, reg­u­la­to­ry tact, clin­i­cal ex­pe­ri­ence, CMC knowhow as well as sales and dis­tri­b­u­tion mus­cle, was a big part of why Over­land won over many oth­er bid­ders af­ter all.

For Over­land, the kind of in­dus­tri­al scale pro­duc­tion that al­lo­gene­ic cell ther­a­py en­ables — with each batch pro­duc­ing enough prod­ucts for 100 pa­tients and a turn­around time shrunk from weeks to days — fits well with the large, price-con­scious mar­ket that it’s try­ing to ad­dress.

“From cost of man­u­fac­tur­ing to ef­fi­cien­cy, I think al­lo­gene­ic brings a dif­fer­ent ball game,” said Ed Zhang, who’s dou­bling as chief busi­ness of­fi­cer and chief op­er­at­ing of­fi­cer.

And if every­thing goes well, it will be ball game plays out in Asia al­most in re­al-time as it does in the US and Eu­rope.

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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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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#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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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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#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: Iver­ic Bio, un­de­terred by loom­ing PDU­FA for com­peti­tor, touts sub­group da­ta on GA drug

CHICAGO — While its competitor is on the cusp of likely securing the first FDA nod, Iveric Bio is trudging ahead with its potential treatment for geographic atrophy, an advanced form of AMD, and has new data to support its upcoming NDA filing.

The biotech said its drug was more favorable than sham across all subgroups in the second Phase III study of the investigational complement C5 protein inhibitor, performing similarly to a previous, smaller late-stage trial.

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#AAO22 conference in Chicago (Photo credit: Associate editor Kyle LaHucik)

#AAO22: In bid for first FDA nod in ge­o­graph­ic at­ro­phy, Apel­lis claims an­oth­er first in eye dis­ease field

CHICAGO — Eight weeks before patients and industry find out if the FDA approves the first treatment for geographic atrophy, an advanced form of age-related macular degeneration, the biotech behind the drug is out with some new data on a secondary endpoint.

In what study investigator Charles Wykoff called the “first direct evidence of function preservation by slowing GA growth” in an investigational treatment, Apellis Pharmaceuticals’ drug pegcetacoplan led to less loss of retinal sensitivity versus sham  within 250 microns on either side of the GA lesion over 24 months.

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