Nan­jing Leg­end wows AS­CO re­searchers with ear­ly da­ta on a BC­MA-tar­get­ed CAR-T con­tender

Chi­na’s Nan­jing Leg­end Biotech is a vir­tu­al un­known in the CAR-T field. But it gained mar­quee sta­tus at AS­CO to­day with stel­lar ear­ly mul­ti­ple myelo­ma da­ta on a drug tar­get­ed at B-cell mat­u­ra­tion pro­tein, or BC­MA.

Among 35 re­lapsed, drug-re­sis­tant pa­tients, 33 — 94% — demon­strat­ed clin­i­cal re­mis­sion rang­ing from a com­plete to a par­tial re­sponse. There was a 100% ob­jec­tive re­sponse rate, ac­cord­ing to in­ves­ti­ga­tors.

The in­ves­ti­ga­tors al­so flagged some promis­ing signs of dura­bil­i­ty.

Of the first 19 pa­tients to reach the four-month mark spec­i­fied in the tri­al, a re­mark­able 14 achieved a “strin­gent” com­plete re­sponse (sCR), which is as­so­ci­at­ed with longer sur­vival times. And 5 pa­tients who hit the one-year mark were still in the sCR stage.

Xi­ao­hu (Frank) Fan

Look­ing past the CD19 bio­mark­er, BC­MA of­fers one of the most promis­ing tar­gets in CAR-T. And this is, to date, the largest pa­tient group yet re­port­ed on. There are, how­ev­er, some sig­nif­i­cant caveats to point out. Its BC­MA ri­val blue­bird bio points out that these pa­tients ev­i­dent­ly weren’t as sick as the ones they treat­ed, with few­er ear­li­er drug fail­ures.

But you can be sure that there will be plen­ty of an­a­lysts pour­ing over the da­ta to­day to start cal­cu­lat­ing how the lead­ers all line up.

The same safe­ty is­sues that have plagued in­ves­ti­ga­tors for years now were ev­i­dent in the study. 85% of the pa­tients in the study ex­pe­ri­enced cy­tokine re­lease syn­drome, with two ex­pe­ri­enc­ing a grade 3 case that re­quired Actem­ra to tamp down on the in­flam­ma­to­ry re­sponse. All those pa­tients re­cov­ered.

And no mat­ter who is do­ing the work, CAR-T ap­pears to be more promis­ing than ever for this group of pa­tients.

“While it’s still ear­ly, these da­ta are a strong sign that CAR T-cell ther­a­py can send mul­ti­ple myelo­ma in­to re­mis­sion,” said AS­CO Ex­pert Michael S. Sabel in a state­ment. “It’s rare to see such high re­sponse rates, es­pe­cial­ly for a hard-to-treat can­cer. This serves as proof that im­munother­a­py and pre­ci­sion med­i­cine re­search pays off. We hope that fu­ture re­search builds on this suc­cess in mul­ti­ple myelo­ma and oth­er can­cers.”


Pic­tured: Nan­jing, Chi­na on April 27th, 2017 / Shut­ter­stock

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

Clockwise from left: Canaccord Genuity principal Michelle Gilson, Canaccord Genuity CSO Brian Mueller and BioMarin CSO Hank Fuchs (Canaccord Genuity webcast)

Bio­Marin CSO diss­es ri­vals for the he­mo­phil­ia A gene ther­a­py crown: Way be­hind, fac­ing big re­cruit­ment chal­lenges and at best a .6 on the gen-one scale

The leader in the race to a hemophilia A gene therapy does not like to be compared unfavorably to the competition. And when their top execs do the comparing, don’t look for any modesty — BioMarin, they say, owns the lead.

As Factor VIII expression wanes over time, quite a few analysts have raised questions about the kind of future BioMarin’s gene therapy — a supposed once-and-done treatment — faces if it stops working. But just 7 days away from their PDUFA date, with high odds of success, the top execs clearly feel that they are way out front, while promising their rivals will discover there’s a tough slog ahead trying to pursue trials where large numbers of patients are ineligible for new therapies.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.