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.

ZS Per­spec­tive: 3 Pre­dic­tions on the Fu­ture of Cell & Gene Ther­a­pies

The field of cell and gene therapies (C&GTs) has seen a renaissance, with first generation commercial therapies such as Kymriah, Yescarta, and Luxturna laying the groundwork for an incoming wave of potentially transformative C&GTs that aim to address diverse disease areas. With this renaissance comes several potential opportunities, of which we discuss three predictions below.

Allogenic Natural Killer (NK) Cells have the potential to displace current Cell Therapies in oncology if proven durable.

Despite being early in development, Allogenic NKs are proving to be an attractive new treatment paradigm in oncology. The question of durability of response with allogenic therapies is still an unknown. Fate Therapeutics’ recent phase 1 data for FT516 showed relatively quicker relapses vs already approved autologous CAR-Ts. However, other manufacturers, like Allogene for their allogenic CAR-T therapy ALLO-501A, are exploring novel lymphodepletion approaches to improve persistence of allogenic cells. Nevertheless, allogenic NKs demonstrate a strong value proposition relative to their T cell counterparts due to comparable response rates (so far) combined with the added advantage of a significantly safer AE profile. Specifically, little to no risk of graft versus host disease (GvHD), cytotoxic release syndrome (CRS), and neurotoxicity (NT) have been seen so far with allogenic NK cells (Fig. 1). In addition, being able to harness an allogenic cell source gives way to operational advantages as “off-the-shelf” products provide improved turnaround time (TAT), scalability, and potentially reduced cost. NKs are currently in development for a variety of overlapping hematological indications with chimeric antigen receptor T cells (CAR-Ts) today, and the question remains to what extent they will disrupt the current cell therapy landscape. Click for more details.

What lured Hal Bar­ron away?; Top FDA minds on ac­cel­er­at­ed ap­proval re­forms; ‘Dead wrong’ Aduhelm ad blitz; 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.

Nothing can really compete with Hal Barron’s departure from GlaxoSmithKline as the news of the week, but we do have plenty of original reporting and analysis from the Endpoints team in this edition. Enjoy and have a nice weekend.

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Mer­ck wins le­gal bat­tle over in­sur­ance cov­er­age af­ter ran­somware at­tack

Merck has emerged victorious from a years-long legal battle with insurers over the coverage of more than a billion dollars in losses from the malware NotPetya, with a New Jersey Superior Court judge concluding that the responsibility is on insurers to clarify their policies around cyber attacks.

The pharma giant was one of several victims of a global cyber attack back in 2017 that also hit Danish shipping company Maersk, American food company Mondelēz, French construction giant Saint-Gobain and even the systems monitoring the Chernobyl nuclear power stations, Bloomberg reported back in 2019.

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Crit­ics push back on Alzheimer’s As­so­ci­a­tion ad blitz to get Medicare to change its Aduhelm rul­ing: 'Dead wrong'

The latest Alzheimer’s Association advertising campaign encourages people to fight.

Not against the disease or for more research or treatments, but against the Centers for Medicare and Medicaid Services. More specifically, CMS’ recent reimbursement decision to only pay for Biogen and Eisai’s controversial Alzheimer’s drug Aduhelm for patients in clinical trials.

With CMS’ preliminary decision now in a 30-day comment period, patient advocates’ goal is to convince CMS to reverse its decision with a marketing blitz and public pressure.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Fail­ing to con­firm clin­i­cal ben­e­fit, Gilead pulls 2 ac­cel­er­at­ed ap­proval in­di­ca­tions for can­cer drug

Gilead recently decided to pull two indications for its cancer drug Zydelig — in relapsed follicular B-cell non-Hodgkin lymphoma (FL) and relapsed small lymphocytic leukemia (SLL) — after failing to complete the confirmatory trials required as part of the accelerated approvals from 2014.

“As the treatment landscape for FL and SLL has evolved, enrollment into the confirmatory study has been an ongoing challenge,” Gilead said in a statement, noting it formally notified the FDA of its decision to voluntarily withdraw these indications.

Yingli Phar­ma brings small mol­e­cule re­search to the US in new pact with MD An­der­son; UCB's pso­ri­at­ic arthri­tis drug shows pos­i­tive PhI­II re­sults

Shanghai biotech Yingli Pharma wants to bring its small molecule drug research global — and a new pact with MD Anderson will help it get there.

Yingli and MD Anderson have inked a 5-year collaboration deal that will put its cancer candidates — some of which have already generated data in China — into trials in the US. The lead program is linperlisib, a PI3Kδ inhibitor that’s in a Phase III trial in follicular lymphoma, according to Yingli’s website. In the US, MD Anderson will work with Yingli to put the candidate in a Phase II trial for peripheral T cell lymphoma (PTCL), an uncommon and aggressive type of non-Hodgkin’s lymphoma.

Richard Pazdur (via AACR)

Time lim­its on ac­cel­er­at­ed ap­provals? FDA's on­col­o­gy chief Rick Paz­dur eyes po­ten­tial re­forms via in­ter­na­tion­al ap­proach­es

The spotlight on the accelerated approval pathway continues to shine bright, with the FDA’s top oncology official writing in an opinion that the pathway may be strengthened with bits and pieces of what other regulators in Europe and elsewhere have done with their expedited approval pathways, such as adding expiration dates for these faster approvals to ensure they confirm clinical benefit in a timely manner.

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Hal Barron, Endpoints UKBIO20 (Jeff Rumans)

'Al­tos was re­al­ly a once-in-a-life­time op­por­tu­ni­ty': Hal Bar­ron re­flects on his big move

By all accounts, Hal Barron had one of the best jobs in Big Pharma R&D. He made more than $11 million in 2020, once again reaping more than his boss, Emma Walmsley, who always championed him at every opportunity. And he oversaw a global R&D effort that struck a variety of big-dollar deals for oncology, neurodegeneration and more.

Sure, the critics never let up about what they saw as a rather uninspiring late-stage pipeline, where the rubber hits the road in the Big Pharma world’s hunt for the next big near-term blockbuster, but the in-house reviews were stellar. And Barron was firmly focused on bringing up the success rate in clinical trials, holding out for the big rewards of moving the dial from an average 10% success rate to 20%.

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Executive Director of the EMA Emer Cooke (AP Photo/Geert Vanden Wijngaert)

Eu­ro­pean Par­lia­ment signs off on strength­en­ing drug reg­u­la­tor's abil­i­ty to tack­le short­ages

The European Parliament on Thursday endorsed a plan to increase the powers of the European Medicines Agency, which will be better equipped to monitor and mitigate shortages of drugs and medical devices.

By a vote of 655 to 31, parliament signed off on a provisional agreement reached with the European Council from last October, in which the EMA will create two shortage steering groups (one for drugs, the other for devices), a new European Shortages Monitoring Platform to facilitate data collection and increase transparency, and on funding for the work of the steering groups, task force, working parties and expert panels that are to be established.

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