Judith Shizuru (Stanford Department of Medicine via YouTube)

Jasper Ther­a­peu­tics launch­es out of Stan­ford with new ap­proach to stem cell treat­ment

The first girl in the tri­al came in with chron­ic di­ar­rhea and the im­mune sys­tem of an un­treat­ed HIV pa­tient. Born with a rare ge­net­ic dis­ease that im­ped­ed her abil­i­ty to make B and T cells, she had once been giv­en a stem cell trans­plant but it didn’t take.  Back in the hos­pi­tal, she was in­ject­ed with a new ex­per­i­men­tal an­ti­body and then giv­en a new stem cell trans­plant. Soon, she gained weight. The di­ar­rhea stopped.

“She has nor­mal T cells now,” Ju­dith Shizu­ru, the Stan­ford sci­en­tist who pi­o­neered the an­ti­body, told End­points News. “She’s in school.”

It’s the kind of med­ical sto­ry to launch a biotech around. To­day, Shizu­ru’s com­pa­ny Jasper Ther­a­peu­tics is emerg­ing out of stealth-mode with $35 mil­lion in Se­ries A fund­ing led by Abing­worth and Qim­ing, a mol­e­cule from Am­gen, and a Phase I tri­al set for its first read­out on Mon­day at ASH.

Jasper is broad­ly aimed at mak­ing stem cell trans­plants safer, more ac­ces­si­ble and more ef­fec­tive by us­ing an­ti­bod­ies as con­di­tion­ing agents. These agents clear out bone mar­row to make room for the new stem cells to graft on­to the body.

The new com­pa­ny is one of a hand­ful now us­ing an­ti­bod­ies to help ease stem cell trans­plan­ta­tion. Cal­i­for­nia-based Forty Sev­en is test­ing a com­bi­na­tion of two an­ti­bod­ies, a c-Kit and a CD-47, in mon­keys. In Cam­bridge, Mass., Ma­gen­ta Ther­a­peu­tics is work­ing on a c-Kit that re­leas­es a tox­in af­ter it binds.

William Lis

Jasper’s Phase I us­es a naked an­ti­body called JSP191 to help pa­tients with se­vere com­bined au­toim­mune de­fi­cien­cy re­ceive stem cell trans­plants – the on­ly pos­si­ble cure for the life-threat­en­ing dis­ease – but such trans­plants are used in a wide va­ri­ety of con­di­tions and Jasper has broad­er aims. Those in­clude oth­er au­toim­mune dis­eases, acute myeloid leukemia and cell-di­rect­ed gene ther­a­py.

“There’s a sig­nif­i­cant amount of progress be­ing made in gene ther­a­py,” in­ter­im CEO William Lis told End­points, “but no progress be­ing made in a con­di­tion­ing agent that will help graft gene ther­a­py.”

Shizu­ru path to the new an­ti­body was long and for­tu­itous. In 1987, Arl Arzst, the leg­endary ad ex­ec­u­tive and pres­i­dent of Proc­tor and Gam­ble in­ter­na­tion­al flew in on a re­cruit­ing trip for Stan­ford busi­ness stu­dents. There he vis­it­ed Shizu­ru, a young bi­ol­o­gy PhD can­di­date, be­cause he knew her room­mate. Arzst’s daugh­ter had di­a­betes and as Shizu­ru ex­plained the work she was do­ing on pan­cre­at­ic islet cell trans­plants, he told her to come to Eu­rope.

Shizu­ru had nev­er been to Eu­rope, but there Ar­szt in­tro­duced her to Ken Far­ber and the oth­er founders of the Ju­ve­nile Di­a­betes Foun­da­tion (now the JDRF). The founders struck a years-long cor­re­spon­dence and en­cour­aged Shizu­ru to go to med­ical school, where she de­cid­ed that if sci­en­tists were ever go­ing to de­vel­op trans­plants that didn’t trig­ger an im­mune re­sponse, it would be through stem cell work. She con­tin­ued her work at the Irv Weiss­man’s Stan­ford re­gen­er­a­tive lab, where even­tu­al­ly a grad­u­ate stu­dent made a dis­cov­ery that piqued her in­ter­est.

To put new stem cells in, you have to get the old stem cells out. That’s not al­ways easy. The cells sit in these pock­ets in the bone mar­row, and they’re pret­ty com­fort­able there. Doc­tors have to force them out, of­ten us­ing chemother­a­py or ra­di­a­tion, which dam­age DNA and cause se­vere side ef­fects. The costs some­times out­weigh the ben­e­fits.

“There are dis­eases we’re not treat­ing be­cause it’s too dan­ger­ous,” Shizu­ru said. “And the kids we’re treat­ing, they’re so, so frag­ile.”

The grad stu­dent had shown in mice that an­ti­bod­ies could be used to de­plete the stem cells and po­ten­tial­ly elim­i­nate the need for chemother­a­py or ra­di­a­tion. Shizu­ru and her team be­gan look­ing to see if any­one had de­vel­oped a hu­man ver­sion of the an­ti­body, CD117. It turned out Am­gen had al­ready de­vel­oped a ver­sion of this an­ti­body for a dif­fer­ent use. It al­so turned out she had a for­mer post­doc and a for­mer ad­vi­sor who worked there. They be­gan a col­lab­o­ra­tion.

“We set out to cross the val­ley of death,” Shizu­ru said, us­ing an in­dus­try slang term for the jump from an­i­mal mod­els to hu­man us­es.

Af­ter mak­ing a va­ri­ety of tweaks to the treat­ment, they pub­lished a pa­per in Sci­ence Trans­la­tion­al Med­i­cine in 2016 show­ing the an­ti­bod­ies cre­at­ed a 10,000 fold re­duc­tion in the num­ber of stem cells in mice.

The same year, they be­gan a clin­i­cal tri­al on 90 SCID pa­tients. These pa­tients had re­ceived stem cell trans­plants when they were very young but hadn’t been giv­en chemo or ra­di­a­tion for fear the side ef­fects would be too se­vere. The orig­i­nal trans­plants boost­ed their num­ber of im­mune cells, but with­out chemo or ra­di­a­tion, the stem cells don’t graft in­to those pock­ets and the body won’t con­tin­ue pro­duc­ing T cells. With­out those, they are ex­tra­or­di­nar­i­ly prone to in­fec­tion. Many pass away be­fore age 2.

The hope is that the an­ti­bod­ies al­lowed the stem cells to graft, and the pre­lim­i­nary an­swer to that ques­tion will be out on Mon­day. For the first girl in the tri­al, life has im­proved but ques­tions about how long her body will make im­mune cells re­main. Still, for that girl and oth­ers, Shizu­ru is con­fi­dent.

“We see there is stem cell en­graft­ment,” Shur­izi said. “They are ac­tu­al­ly mak­ing new T cells.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

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Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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Hill­house re­casts spot­light on Chi­na's biotech scene with $160M round for Shang­hai-based an­ti­body mak­er

Almost two years after first buying into Genor Biopharma’s pipeline of cancer and autoimmune therapies, Hillhouse Capital has led a $160 million cash injection to push the late-stage assets over the finish line while continuing to fund both internal R&D and dealmaking.

The Series B has landed right around the time Genor would have listed on the Hong Kong stock exchange, according to plans reported by Bloomberg late last year. Insiders had said that the company was looking to raise about $200 million.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

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In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

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Mark Genovese (Stanford via Twitter)

Gilead woos fil­go­tinib clin­i­cal in­ves­ti­ga­tor from Stan­ford to lead the charge on NASH, in­flam­ma­to­ry dis­eases

With an FDA OK for the use of filgotinib in rheumatoid arthritis expected to drop any day now, Gilead has recruited a new leader from academia to lead its foray into inflammatory diseases.

Mark Genovese — a longtime Stanford professor and most recently the clinical chief in the division of immunology and rheumatology — was the principal investigator in FINCH 2, one of three studies that supported Gilead’s NDA filing. In his new role as SVP, inflammation, he will oversee the clinical development of the entire portfolio.

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Stephen Isaacs, Aduro president and CEO (Aduro)

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Isaacs —who forged a string of high-profile Big Pharma deals along the way — has wrapped a 13-year run at the biotech with one program for kidney disease going to the new owners at Chinook Therapeutics. A host of once-heralded assets like their STING agonist program partnered with Novartis (which dumped their work on ADU-S100 after looking over weak clinical results), the Lilly-allied cGAS-STING inhibitor program and the anti-CD27 program out-licensed to Merck will all be posted for auction under a strategic review process.

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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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No­var­tis chips in $10M for IPO-bound part­ner Pli­ant; Tenax shares soar on heart drug da­ta

Novartis is coming in with $10 million to help support the looming IPO of a partner. Pliant Therapeutics posted a new filing with the SEC showing that Novartis is buying the shares at $15, the mid-point of the range. It’s adding several million shares to the offering, bringing the total to around $135 million. Biotech companies have been enjoying quite a run on virtual Wall Street, with investors boosting new offerings to some big hauls.