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.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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Karyopharm taps long­time Pfiz­er, Am­gen vet to steer the ship; With Mer­ck in the rearview mir­ror, Roger Perl­mut­ter stakes his claim to a CEO job — and it's a sur­pris­ing choice

Like many who work in biopharma, Richard Paulson got started in the field because of a love of science.

Paulson had just finished business school and was looking to start a career that married his two passions. While looking for jobs, he thought of his grandmother who had struggled with Alzheimer’s disease, recalling how he saw first-hand what innovative medicines can do for patients. Ultimately, he started his first job in the space as a sales rep at Glaxo Wellcome, one of GlaxoSmithKline’s predecessors before its merger with SmithKline Beecham in 2000.

EMA safe­ty com­mit­tee seeks more in­fo on heart in­flam­ma­tion fol­low­ing Pfiz­er Covid-19 vac­cine

The European Medicines Agency’s safety committee said Friday that it’s aware of cases of inflammation of the heart muscle and inflammation of the membrane around the heart, mainly reported following vaccination with Pfizer’s Covid-19 vaccine, known in Europe as Comirnaty.

“There is no indication that these cases are due to the vaccine,” the EMA’s Pharmacovigilance Risk Assessment Committee said.

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