James Li (JW)

Teamed up with Lyell, Juno/WuXi joint ven­ture files IPO bet­ting on Chi­na's CAR-T fu­ture

JW Ther­a­peu­tics got its start on the back of CAR-T tech from Juno Ther­a­peu­tics, a joint ven­ture bank­ing on the promise of mar­ry­ing cut­ting-edge Amer­i­can tech­nol­o­gy with WuXi AppTec’s state-of-the-art process de­vel­op­ment and late-stage clin­i­cal in­fra­struc­ture in Chi­na. Four years lat­er, a col­lab­o­ra­tion with a Juno co-founder’s new start­up is ce­ment­ing a bridge to the pub­lic mar­ket.

Lyell has grant­ed JW ex­clu­sive Chi­na rights to two CAR-T pro­grams tar­get­ing AFP and GPC3, anti­gens ap­plic­a­ble broad­ly across sol­id tu­mors, the com­pa­nies an­nounced last week. JW not­ed in its IPO fil­ing on the Hong Kong stock ex­change that it’s al­so nabbed rights to the T-cell an­ti-ex­haus­tion func­tion­al­i­ty.

“Sub­stan­tial” mile­stones and roy­al­ty pay­ments aren’t due un­til the first Lyell ther­a­py is ap­proved in JW ter­ri­to­ry.

Cheng Liu

The col­lab­o­ra­tion fol­lows a deal with Eu­re­ka Ther­a­peu­tics, a Cal­i­for­nia-based part­ner of both Juno and Lyell, through which JW ac­quired Syra­cuse — Eu­re­ka’s Chi­nese sub­sidiary — and its un­der­ly­ing tech plat­form. Eu­re­ka CEO Cheng Liu joined the JW board in hopes of join­ing forces to build the lead­ing cell ther­a­py com­pa­ny in Chi­na.

“Eu­re­ka still keep their in­de­pen­dence out­side Chi­na, they will do their own things,” JW chief James Li said at the time. “But in Chi­na we com­bine their ear­ly dis­cov­ery pow­er with our late-stage de­vel­op­ment and, even­tu­al­ly we are launch­ing our prod­uct next year, com­mer­cial ca­pa­bil­i­ties.”

While the terms went undis­closed, JW now re­veals that the ac­qui­si­tion was val­ued at $105 mil­lion.

Both the Lyell and Eu­re­ka pacts are ex­plic­it­ly fo­cused on sol­id tu­mors, ini­tial­ly start­ing out in he­pa­to­cel­lu­lar car­ci­no­ma.

“One thing we’ve been think­ing hard in the past cou­ple years is how we can get in­to the sol­id tu­mor space, which is ab­solute­ly — to me, it’s the fu­ture of cell ther­a­py,” Li told End­points News.

Be­ing one of the pi­o­neers along­side Fo­s­un Kite and Leg­end, he said, has al­lowed JW to think ahead of new­er ri­vals both in terms of es­tab­lish­ing the best prac­tices and think­ing ahead of the com­pe­ti­tion.

It’s not done with the deal spree. JW not­ed that it’s plan­ning to use parts of the IPO pro­ceds to ex­e­cute its op­tions on a cou­ple of  off-the-shelf nat­ur­al killer cell ther­a­py can­di­dates from Tai­wan’s Ace­po­dia, tar­get­ing HER2 and an­oth­er undis­closed tar­get.

Reg­u­la­tors at the Na­tion­al Med­ical Prod­ucts Ad­min­is­tra­tion have ac­cept­ed its NDA for rel­ma-cel, the lead CD19-tar­get­ing CAR-T ther­a­py de­vel­oped from Juno’s pro­gram. The ini­tial in­di­ca­tion is third-line dif­fuse large B cell lym­phoma — with mul­ti­ple oth­er blood can­cers lined up be­hind it.

Be­hind that there’s a BC­MA pro­gram for which JW plans to file an IND in the first half of 2021, JW wrote in its IPO ap­pli­ca­tion.

Over the years it’s raised $190 mil­lion in ven­ture fund­ing, and pre­vi­ous me­dia re­ports has sug­gest­ed it’s seek­ing to raise $200 mil­lion to $300 mil­lion in the pub­lic de­but. As is stan­dard on HKEX, JW hasn’t pen­cilled in any es­ti­mates.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Mathai Mammen (Rob Tannenbaum, Endpoints News at BIO 2018)

Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck five years ago, where the soft-spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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Samantha Du, Zai Lab CEO

Any­one still look­ing for a CD47? Zai Lab shelves PhI pro­gram af­ter re­view­ing 'com­pet­i­tive land­scape'

Over the past few years, the promise of blocking CD47 — a “don’t eat me” signal co-opted by cancer cells — has sent drugmakers big and small into a frenzy. But one biotech is now bowing out.

Zai Lab is deprioritizing ZL-1201, its CD47 inhibitor, scrapping plans for a Phase II trial. It will now “pursue out-licensing opportunities,” the company said in its Q2 update. The decision was based on a review of the competitive landscape, it added, without going into further details.

Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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Bernhardt Zeiher, outgoing Astellas CMO (Astellas)

Q&A: Astel­las' re­tir­ing head of de­vel­op­ment re­flects on gene ther­a­py deaths

For anyone who’s been following discussions about the safety alarms surrounding the adeno-associated viruses (AAV) commonly used to deliver gene therapy, Astellas should be a familiar name.

The Japanese pharma — which bought out Audentes Therapeutics near the end of 2019 and later built a gene therapy unit around the acquisition — rocked the field when it reported three patient deaths in a trial testing AT132, the lead program from Audentes designed to treat a rare muscle disease called X-linked myotubular myopathy (XLMTM).

When the company restarted the trial, it adjusted the dose and instituted a battery of other measures to try to prevent the same thing from happening again. But tragically, the first patient to receive the new regimen died just weeks after administration. The therapy remains under clinical hold, and just weeks ago, Astellas flagged another safety-related hold for a separate gene therapy candidate. In the process of investigating the deaths, the company has also taken flak about the way it disclosed information.

Big questions remain — questions that can have big implications about the future of AAV gene therapies.

Bernhardt Zeiher did not imagine any of it when he first joined Astellas as the therapeutic area leader in inflammation, immunology and infectious diseases. But his ascent to chief medical officer and head of development coincided almost exactly with Astellas’ big move into gene therapy, putting him often in the driver’s seat to grapple with the setbacks.

As Zeiher prepares to retire next month after a 12-year tenure — leaving the unfinished tasks to his successor, a seasoned cancer drug developer — he chatted with Endpoints News, in part, to discuss the effort to understand what happened, lessons learned and the criticism along the way.

The transcript has been lightly edited for length and clarity.

Endpoints: I want to also ask you a bit about the gene therapy efforts you’ve been working on. Astellas has really been at the forefront of discovering the safety concerns associated with AAV gene therapy. What’s that been like for you?

Zeiher: Well, I have to admit, it’s been a bit of a roller coaster. We acquired Audentes. Huge amount of enthusiasm. What we saw with AT132 — that was the lead program in XLMTM — was just remarkable efficacy. I mean, kids who went from being on ventilators, not able to eat for themselves, sit up, do things like that, to off ventilators, walking, you know, really — one investigator called it this Lazarus-like effect. It was just really dramatic efficacy. And then to have the safety events that occurred. So they actually occurred within that first year of the acquisition. So we had the three patient deaths. Me and my organization became very, very much involved. In fact, Ed Conner, who had been the chief medical officer, he left after some of the deaths, but I stepped in as the kind of acting chief medical officer, we had another chief medical officer who was involved, and then we had a fourth death, and I became acting again for a period of time.

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HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

US weighs new route of ad­min­is­tra­tion for mon­key­pox vac­cine as cas­es climb — re­port

Less than a week after HHS Secretary Xavier Becerra declared monkeypox a national health emergency, reports have emerged that the US plans to extend its vaccine supply by opting for a different route of administration.

Officials are expected to call for intradermal injection of Bavarian Nordic’s Jynneos vaccine — the only shot approved specifically for monkeypox in the US — as opposed to subcutaneous injection, unnamed sources told both the New York Times and Washington Post on Tuesday.

'Messy at best': Is the US re­peat­ing the same Covid mis­steps with mon­key­pox mes­sag­ing?

When Kyle Planck first suspected he might have monkeypox in late June, he went to the CDC website and found six photos of different types of lesions. And that was about it for general public information.

Planck, who is a sixth-year PhD pharmacology researcher at Weill Cornell, kept looking though and found a separate part of the CDC website meant for healthcare professionals. There he found a medical slide deck with more pictures, professional journal articles and more details about symptoms and diagnosis.

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Andy Jassy, Amazon CEO (Isaac Brekken/AP Images for NFL, File)

Up­dat­ed: FDA slaps Ama­zon with a warn­ing let­ter for sell­ing OTC mole re­moval prod­ucts

The FDA’s Center for Drug Evaluation and Research on Tuesday released a warning letter sent last week to Amazon CEO Andy Jassy in Seattle for selling mole removal products over-the-counter, or, as the FDA explains, “introducing, delivering, or causing the introduction or delivery into interstate commerce of products that are unapproved new drugs.”

“There are no over-the-counter (OTC) drugs that can be legally sold for mole or skin tag removal, and FDA has safety concerns about drugs marketed OTC directly to consumers for these uses,” the agency said in its Aug. 4 warning.