Keen to stay out front in cell ther­a­py, Gilead­'s Kite builds its own vi­ral vec­tor man­u­fac­tur­ing op­er­a­tions

Days af­ter woo­ing Christi Shaw to the helm at Kite, Gilead is once again tout­ing just how se­ri­ous it is about grow­ing its pi­o­neer­ing CAR-T and fol­low-up cell ther­a­py busi­ness. The sub­sidiary is build­ing a new fa­cil­i­ty ded­i­cat­ed to vi­ral vec­tor man­u­fac­tur­ing with­in a bi­o­log­ics site at Ocean­side, Cal­i­for­nia.

Tim Moore BMWS

Right now, Kite gets it vi­ral vec­tors — crit­i­cal start­ing ma­te­r­i­al for edit­ing the T cells to ac­ti­vate can­cer-killing ca­pa­bil­i­ties — from con­tract man­u­fac­tur­ers, said Tim Moore, EVP of tech­ni­cal op­er­a­tions. But amid a cell ther­a­py boom, the lim­its that place on com­mer­cial sup­ply and de­vel­op­ment is be­com­ing more ap­par­ent.

It’s not about ex­it­ing those pacts all to­geth­er, Moore em­pha­sized; rather, the new, 67,000-square-foot fa­cil­i­ty be com­ple­men­tary to what’s al­ready in place.

“We can still lever­age ex­ter­nal man­u­fac­tur­ing ca­pa­bil­i­ties but it’s go­ing to help ac­cel­er­ate our ca­pa­bil­i­ties and high­light our lead­er­ship in cell ther­a­py by leav­ing no stone un­turned,” he told me.

Un­der for­mer CEO John Mil­li­gan, Gilead had ini­ti­at­ed an ag­gres­sive ex­pan­sion on the cell ther­a­py man­u­fac­tur­ing front, plot­ting sites in the Nether­lands, Mary­land and Cal­i­for­nia last May. Daniel O’Day took it a notch up, re­veal­ing in his first an­a­lyst call weeks ago that bring­ing in a new CEO for the stand­alone Kite would be a top pri­or­i­ty. And he went for an in­dus­try heavy­weight in Shaw, the re­cent pres­i­dent of Eli Lil­ly’s Bio­Med­i­cines unit who has a per­son­al sto­ry to tell about can­cer.

Kite ex­pects to have the Ocean­side site com­plete and li­censed in the sec­ond half of 2021, Moore said. There will be some hir­ing for day-to-day op­er­a­tions, though he didn’t spec­i­fy how many.

So­cial im­age: Gilead, AP Im­ages

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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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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Amid mas­sive re­struc­tur­ing, Bio­gen looks to re­duce phys­i­cal pres­ence in Boston

Biogen is putting a sizable chunk of office and research space in Kendall Square and Weston, MA up for sublease, marking another big change as the biotech grapples with the aftershock of a disastrous and controversial rollout for its Alzheimer’s drug.

The company calls it “part of Biogen’s overall implementation of the ‘Future of Work,’ which is allowing us to optimize our footprint and reduce the amount of space we occupy, taking into consideration new elements such as the hybrid work model,” the Boston Globe reported, quoting a spokesperson.

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Robert Califf, FDA commissioner (Tom Williams/CQ Roll Call via AP Images)

Hop­ing to ex­pand mon­key­pox vac­cine sup­ply, US paves the way for new route of ad­min­is­tra­tion

After making it clear that the US’ current monkeypox vaccine supply is insufficient, the FDA on Tuesday authorized a new route of administration that should increase the number of available doses by five-fold.

Regulators cleared Bavarian Nordic’s Jynneos vaccine for intradermal injection in adults older than 18. Unlike subcutaneous injection — the current method by which vaccine is delivered under the skin — an intradermal jab goes directly into the skin. It’s believed that this method requires less vaccine, since the dermis is rich in dendritic cells which specialize in taking up foreign antigens and presenting them to the immune system, according to Daniel Kuritzkes, chief of infectious diseases at Brigham and Women’s Hospital in Boston.

John Quisel, Disc Medicine CEO

Disc Med­i­cine goes pub­lic in re­verse merg­er with strug­gling Gem­i­ni Ther­a­peu­tics

After licensing a failed Roche schizophrenia drug last year, Disc Medicine is going public via a reverse merger with Gemini Therapeutics.

The combined company, while still named Disc Medicine, will trade under the stock symbol $IRON, in reference to Disc’s lineup of therapies for blood iron disorders. Alongside the merger, Disc has secured $53.5 million in another financing round, building on the $90 million Series B it raised in September.

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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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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.

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.