Jasper's cell ther­a­py con­di­tion­ing agent shows promise in small study; Vi­iV's Ruko­bia sees ap­proval in Eu­rope

Jasper Ther­a­peu­tics, broad­ly aimed at mak­ing cell ther­a­py safer, re­leased pre­lim­i­nary da­ta for its com­ple­ment to stem cell trans­plants on Mon­day.

The biotech is run­ning an open-la­bel Phase I tri­al of its lead pro­gram JSP191, an an­ti-CD117 mon­o­clon­al an­ti­body that tar­gets the stem cell fac­tor re­cep­tor, as a con­di­tion­ing agent in pa­tients aged 65 to 74 with myelodys­plas­tic syn­dromes or acute myeloid leukemia that are un­der­go­ing blood cell trans­plan­ta­tion. Six pa­tients have re­ceived the treat­ment thus far, and all six showed suc­cess­ful en­graft­ment.

Re­searchers al­so saw donor myeloid chimerism of at least 95% in five of six evalu­able pa­tients at 28 days. Three of the five al­so showed that resid­ual dis­ease could not be found, in­di­cat­ing a “com­plete erad­i­ca­tion,” Jasper said.

Jasper’s the­o­ry be­hind JSP191 is that it blocks the stem cell fac­tor from bind­ing to CD117 and dis­rupts sur­vival sig­nals, caus­ing the stem cells to die off. That cre­ates an “emp­ty space” of sorts in the bone mar­row, al­low­ing for donor or gene-cor­rect­ed trans­plant­ed stem cells to en­graft.

The com­pa­ny launched out of Stan­ford, emerg­ing from stealth in late 2019 with a $35 mil­lion Se­ries A round.

Eu­rope ap­proves Ruko­bia for drug-re­sis­tant HIV

GSK’s Vi­iV saw the FDA ap­prove its twice-dai­ly pill for HIV back in Ju­ly, but now they’ve re­ceived the green light on an­oth­er con­ti­nent.

The Eu­ro­pean Union on Mon­day grant­ed Ruko­bia the thumbs up for use in com­bi­na­tion with oth­er an­ti­retro­vi­ral ther­a­pies in adults with drug-re­sis­tant HIV, about two months af­ter the CHMP not­ed its pos­i­tive opin­ion. In the ran­dom­ized co­hort of the drug’s piv­otal tri­al, 60% of the 272 pa­tients who re­ceived Ruko­bia in ad­di­tion to op­ti­mized back­ground ther­a­py saw un­de­tectable HIV vi­ral load af­ter 96 weeks.

“There still re­mains a small sub­set of peo­ple liv­ing with mul­ti-drug re­sis­tant HIV who are at risk of hav­ing their dis­ease progress,” Vi­iV CEO Deb­o­rah Wa­ter­house said in a state­ment. “We aim to meet the di­verse needs of the HIV com­mu­ni­ty.”

Ruko­bia func­tions by tar­get­ing the first step of the HIV life cy­cle and shows no cross-re­sis­tance to oth­er cur­rent­ly li­censed an­ti­retro­vi­ral class­es, Vi­iV says. That can help HIV pa­tients who have strug­gled to sup­press their vi­ral lev­els us­ing oth­er treat­ments.

Vi­iV al­so notched an ap­proval in the US for Cabe­nu­va last month, be­com­ing the first FDA-ap­proved in­jectable for HIV. The ther­a­py will look to re­place the dai­ly oral pills that are cur­rent­ly stan­dard of care in treat­ing HIV-pos­i­tive adults.

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.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.

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.

'Messy at best': Is the US re­peat­ing the same Covid mis­steps with mon­key­pox mes­sag­ing?

When Kyle Planck first suspected he might have monkeypox in late June, he went to the CDC website and found six photos of different types of lesions. And that was about it for general public information.

Planck, who is a sixth-year PhD pharmacology researcher at Weill Cornell, kept looking though and found a separate part of the CDC website meant for healthcare professionals. There he found a medical slide deck with more pictures, professional journal articles and more details about symptoms and diagnosis.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

BioCryst re­sumes en­roll­ment for im­mune dis­eases drug; Ea­gle buys stake in res­pi­ra­to­ry dis­ease biotech

In April, the FDA hit BioCryst with a partial hold, leading the biotech to pause enrollment on three of its rare disease clinical trials and sending its shares down 30%.

Last week, BioCryst said the partial clinical hold had been resolved and that it would resume enrolling its clinical trials for its drug, dubbed BCX9930, but with new protocols at a lower 400 mg dose.

BioCryst’s drug is a factor D inhibitor, which was being tested in an array of immune diseases. The biotech is running two pivotal trials for paroxysmal nocturnal hemoglobinuria, as well as a third trial looking at a set of three diseases — C3 glomerulopathy, IgA nephropathy and primary membranous nephropathy.

Andy Jassy, Amazon CEO (Isaac Brekken/AP Images for NFL, File)

Up­dat­ed: FDA slaps Ama­zon with a warn­ing let­ter for sell­ing OTC mole re­moval prod­ucts

The FDA’s Center for Drug Evaluation and Research on Tuesday released a warning letter sent last week to Amazon CEO Andy Jassy in Seattle for selling mole removal products over-the-counter, or, as the FDA explains, “introducing, delivering, or causing the introduction or delivery into interstate commerce of products that are unapproved new drugs.”

“There are no over-the-counter (OTC) drugs that can be legally sold for mole or skin tag removal, and FDA has safety concerns about drugs marketed OTC directly to consumers for these uses,” the agency said in its Aug. 4 warning.

Craig Thompson, Cerevance CEO

UP­DAT­ED: Mer­ck makes first big splash for Alzheimer’s drug R&D since 2017 fail, ink­ing re­search pact with Cere­vance

For the first time since discontinuing its late-stage Alzheimer’s program, Merck has found promise on the path forward in the memory-robbing disease.

After a Phase III flop of its drug verubecestat, the New Jersey Big Pharma axed the study in early 2018. More than four years later, the company is ready to sign up for another pact to test the waters of the befuddling disease.

This time, there’s $1.1 billion in biobucks on the line and a target that its partner says no other biopharma is looking at en route to finding the next treatment for Alzheimer’s, a neuroscience field that has hit hurdle after hurdle for decades.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,500+ biopharma pros reading Endpoints daily — and it's free.