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.”

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.

Who are the women blaz­ing trails in bio­phar­ma R&D? Nom­i­nate them for End­points' 2022 spe­cial re­port

Over the past three years, Endpoints News has spotlighted 60 women who have blazed trails and supercharged R&D across the biopharma world. And judging from the response we’ve received, to both our special reports and live events, telling their stories — including any obstacles they may have had to overcome — has inspired our readers in many different ways.

But change takes time, and the fact remains that women are still underrepresented at the upper ranks of the drug-making world.

Up­dat­ed: 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 subbleases are “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,” Biogen spokesperson Ashleigh Koss wrote in a statement to Endpoints News, adding that the company has had subleases across several buildings for years.

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Lorenz Mayr, Vector BioPharma CEO (PhoreMost)

Up­dat­ed: 'Close to the mid­dle': Ver­sant launch­es Vec­tor with 'gut­less ade­n­ovirus' de­liv­ery method

Most of the gene therapy world centers around viral vectors, but a few biotechs have sprung up in recent years to try and avoid the safety issues that can come with adeno-associated viruses.

A new biotech has emerged to straddle somewhere “close to the middle,” in the words of Alex Mayweg, Versant managing director and board member of the incubator’s latest startup, Vector BioPharma.

Pfiz­er launch­es re­bate pro­gram for rare dis­ease pa­tients who have to stop tak­ing Panzy­ga

Pfizer is launching its second-ever rebate program, this time for Panzyga, its treatment for a rare neurological disease of the peripheral nerves.

The program began last month, according to STAT which first reported the news, and offers a refund of out-of-pocket costs for patients who must discontinue their course before the fifth treatment for “clinical reasons.”

Panzyga was approved back in 2018 to treat primary immunodeficiency (PI) in patients two years and older and chronic immune thrombocytopenia (cITP) in adults. It has since picked up an indication in chronic inflammatory demyelinating polyneuropathy (CIDP), a condition that’s characterized by weakness of the arms or legs, tingling or numbness, and a loss of deep tendon reflexes, according to the NIH.

Horizon's back-to-school campaign for children with cystinosis includes an all about me poster as part of a care package box.

Hori­zon read­ies kids and fam­i­lies for back to school with week­long ac­tiv­i­ties around rare dis­ease cysti­nosis

Going back to school is usually a bumpy readjustment from summer freedom for all kids, but especially for kids with chronic health conditions. Horizon Therapeutics is hoping to help smooth the way for some who have the rare disease cystinosis. Cystinosis is a genetic disease that causes the amino acid cystine to build up in different tissues and organs.

The “Gear Up” for school campaign is running all week with different online and at-home events and activities for families and children with cystinosis. Each family who signed up receives a care package mailed to their home including an activity coloring book “Michael’s Show-and-Tell.” The book tells Michael’s story about living with cystinosis while offering kids matching, coloring and finding object games along with information.

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Alessandro Maselli, Catalent CEO

Catal­ent ac­quires North Car­oli­na CD­MO for $475M, boost­ing oral solids work

As Catalent has been expanding its reach in the US this year, as well as recently completing a C-suite shuffle, the company announced last night that it has acquired the CDMO Metrics Contract Services for $475 million from Mayne Pharma Group.

The acquisition will increase Catalent’s capabilities in oral solid formulation development, manufacturing and packaging as well as expand its capacity to handle more highly potent compounds.

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Phar­mas spend mil­lions on di­a­betes ad­ver­tis­ing, but few pa­tients can re­call brand names — sur­vey

While many Big Pharma diabetes brands spend millions of dollars on TV ads every year, most people with type 2 diabetes don’t recognize specific drug brand names, according to a new study.

No brand garnered more than 30% recognition in Phreesia Life Science’s latest in-office patient survey of more than 4,000 adults with type 2 diabetes. Eli Lilly’s Trulicity topped the list as the most recognized brand with 29% of those surveyed recalling it, followed by Boehringer Ingelheim and Lilly’s Jardiance at 27% and Merck’s Januvia and Novo Nordisk tying for the third spot with 24%. Meanwhile, 76% of the patients surveyed were familiar with the generic treatment metformin.

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Faced with thou­sands of opi­oid law­suits, En­do says it will like­ly file for bank­rupt­cy 'im­mi­nent­ly'

Endo International will likely be the next pharma company to file for bankruptcy under a mountain of opioid lawsuits.

The Dublin, Ireland-based company revealed in its Q2 results on Tuesday that it’s in talks with first lien creditors, and that “these negotiations will likely result in a pre-arranged filing under Chapter 11 of the U.S. Bankruptcy Code by Endo International plc and substantially all of its subsidiaries, which could occur imminently.”