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.”

Late Fri­day ap­proval; Trio of biotechs wind down; Stem cell pi­o­neer finds new fron­tier; Biotech icon to re­tire; 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.

I hope your weekend is off to a nice start, wherever you are reading this email. As for me, I’m trying to catch the tail of the Lunar New Year festivities.

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Pfiz­er lays off em­ploy­ees at Cal­i­for­nia and Con­necti­cut sites

Pfizer has laid off employees at its La Jolla, CA, and Groton, CT sites, according to multiple LinkedIn posts from former employees.

The Big Pharma confirmed to Endpoints News it has let go of some employees, but a spokesperson declined to specify how many workers were impacted and the exact locations affected. Earlier this month, the drug developer had confirmed to Endpoints it was sharpening its focus and doing away with some early research on areas such as rare disease, oncology and gene therapies.

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Rodney Rietze, iVexSol CEO

Bris­tol My­ers, Charles Riv­er join Se­ries A fund­ing for iVex­Sol

Massachusetts-based iVexSol has secured funding to the tune of $23.8 million in its latest Series A round. The new investors include Bristol Myers Squibb, manufacturer Charles River Laboratories and Asahi Kasei Medical.

iVexSol is a manufacturer of lentiviral vectors (LVV), used in making gene therapies, and this latest round of fundraising brings its total Series A total over $39 million, which will be used to recruit more employees and bolster its technology.

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Jake Van Naarden, Loxo@Lilly CEO

Lil­ly en­ters ripe BTK field with quick FDA nod in man­tle cell lym­phoma

Eli Lilly has succeeded in its attempt to get the first non-covalent version of Bruton’s tyrosine kinase, or BTK, inhibitors to market, pushing it past rival Merck.

The FDA gave an accelerated nod to Lilly’s daily oral med, to be sold as Jaypirca, for patients with relapsed or refractory mantle cell lymphoma.

The agency’s green light, disclosed by the Indianapolis Big Pharma on Friday afternoon, catapults Lilly into a field dominated by covalent BTK inhibitors, which includes AbbVie and Johnson & Johnson’s Imbruvica, AstraZeneca’s Calquence and BeiGene’s Brukinsa.

Tony Johnson, Goldfinch Bio CEO (Goldfinch via YouTube)

Kid­ney dis­ease drug­mak­er Goldfinch Bio shuts down

Goldfinch Bio, attempting to make treatments for kidney diseases and diabetic nephropathy, is shutting down.

President and CEO Tony Johnson confirmed to Endpoints News Friday afternoon that the biotech shut down after “fundraising challenges in the current macro-environment.” Fierce Biotech first reported the news.

Johnson, who joined in 2017 after a stint as SVP of early clinical development at AstraZeneca, said in a text that the company “entered the ABC process recently,” referring to an assignment for the benefit of the creditors, which provides a different wind-down avenue than a bankruptcy.

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Filip Dubovsky, Novavax CMO

No­vavax gets ready to take an­oth­er shot at Covid vac­cine mar­ket with next sea­son plans

While mRNA took center stage at yesterday’s FDA vaccine advisory committee meeting, Novavax announced its plans to deliver an updated protein-based vaccine based on new guidance.

Vaccines and Related Biological Products Advisory Committee (VRBPAC) members voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all future vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

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Eliot Forster, F-star CEO (Rachel Kiki for Endpoints News)

F-star gets down to the wire with $161M sale to Chi­nese buy­er as na­tion­al se­cu­ri­ty con­cerns linger

With the clock ticking on F-star Therapeutics’ takeover by a Chinese buyer, the companies are still scrambling to remove a hold on the deal from the US government’s Committee on Foreign Investment in the United States.

F-star and invoX Pharma said they are “actively negotiating” with CFIUS “about the terms of a mitigation agreement to address CFIUS’s concerns regarding potential national security risks posed by the transaction.”

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CBER Director Peter Marks (Susan Walsh/AP Images)

FDA ad­vi­so­ry com­mit­tee votes unan­i­mous­ly in fa­vor of bi­va­lent Covid shots re­plac­ing pri­ma­ry se­ries

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted unanimously in favor of “harmonizing” Covid vaccine compositions, meaning all current vaccine recipients would receive a bivalent vaccine, regardless of whether they’ve gotten their primary series.

The vote marks an effort to clear up confusion around varying formulations and dosing schedules for current primary series and booster vaccines, as well as “get closer to the strains that are circulating,” according to committee member Paul Offit, professor of pediatrics at the Children’s Hospital of Philadelphia.

In­vestor 'misalign­men­t' leads to tR­NA biotech's shut­ter­ing

A small biotech looking to carve a lane in the tRNA field has folded, an investor and a co-founder confirmed to Endpoints News.

Similar to Flagship’s Alltrna and other upstarts like Takeda-backed hC Bioscience, the now-shuttered Theonys was attempting to go after transfer RNA, seen as a potential Swiss Army knife in the broader RNA therapeutics space. The idea is that one tRNA drug could be used across a galaxy of disorders and diseases.

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