Gaurav Shah, Rocket Pharmaceuticals CEO

Tout­ing 100% OS rate in piv­otal rare dis­ease tri­al, Rock­et Phar­ma ready to head to FDA

In drug de­vel­op­ment, you don’t throw around the word “cure” light­ly. But Rock­et Phar­ma­ceu­ti­cals’ lat­est cut of gene ther­a­py da­ta has at least one an­a­lyst won­der­ing about the cu­ra­tive po­ten­tial.

Re­port­ing topline re­sults from a Phase II piv­otal tri­al in­volv­ing pa­tients with se­vere leuko­cyte ad­he­sion de­fi­cien­cy-I (LAD-1), Rock­et said among nine par­tic­i­pants, the over­all sur­vival at one year is 100%.

Based on the re­sults, the biotech says it ex­pects to file for ap­proval ear­ly next year. But in­vestor re­ac­tion ap­pears mut­ed, with the stock $RCKT trad­ing rel­a­tive­ly flat.

LAD-I is char­ac­ter­ized by de­fects in mal­func­tion­ing white blood cells that can’t prop­er­ly trav­el to wounds or in­fec­tion sites, and pa­tients of­ten suf­fer from re­cur­rent bac­te­r­i­al in­fec­tions. While al­lo­gene­ic bone mar­row trans­plants can cure the con­di­tion, they come with their own risks and Rock­et was look­ing to de­vel­op an al­ter­na­tive with its lentivi­ral gene ther­a­py.

With­out a trans­plant, pa­tients with se­vere LAD-I usu­al­ly on­ly have a few years to live, not­ed Cowen an­a­lyst Tyler Van Bu­ren, who called the da­ta “re­mark­able.”

“Re­duc­tions in hos­pi­tal­iza­tions and in­fec­tions were high­ly stat sig, and skin rash­es/wounds re­solved,” he wrote. “Thus, RP-L201 ap­pears cu­ra­tive.”

Rock­et not­ed that pa­tients with LAD-I have mu­ta­tions in a gene en­cod­ing for the be­ta-2 in­te­grin com­po­nent CD18. And af­ter re­ceiv­ing RP-L201, all nine pa­tients in the tri­al — aged five months to nine years — showed CD18 restora­tion and ex­pres­sion on more than 10% of neu­trophils.

“At three to 24 months af­ter RP-L201 in­fu­sion, all nine pa­tients sus­tained sta­ble CD18 ex­pres­sion (me­di­an: 56%) with no ther­a­py-re­lat­ed se­ri­ous ad­verse events,” CEO Gau­rav Shah said.

The FDA pre­vi­ous­ly grant­ed it the re­gen­er­a­tive med­i­cine ad­vanced ther­a­py, or RMAT, des­ig­na­tion.

George Scangos (L) and Marianne De Backer

Pi­o­neer­ing biotech icon George Scan­gos hands in his re­tire­ment pa­pers — and this time it’s for re­al

George Scangos, one of the all-time great biotech CEOs, says the time has come to turn over the reins one last time.

The 74-year-old biotech legend spent close to three decades in a CEO post. The first was at Exelixis — which is still heavily focused on a drug Scangos advanced in the clinic. The second “retirement” was at Biogen, where he and his team were credited with a big turnaround with the now fading MS blockbuster Tecfidera. And the third comes at Vir, where he traded in his Big Biotech credentials for a marquee founder’s role back on the West Coast, hammering out a Covid-19 alliance with Hal Barron — then R&D chief at GSK — and breaking new ground on infectious diseases with some high-powered venture players.

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Jeanne Loring, director of the Center for Regenerative Medicine (Credit: Jamie Scott Lytle)

A stem cell pi­o­neer sent an ex­per­i­ment in­to space. Pa­tients are the next fron­tier

Last July, Jeanne Loring stood on a dirt road surrounded by Florida swampland and watched as a nearby SpaceX rocket blasted into the sky. The payload included a very personal belonging: cell clusters mimicking parts of her brain.

For more than two decades, Loring has been at the forefront of a stem cell field that always seems on the brink of becoming the next thing in medicine, but has been slow to lift off.

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Pa­tient death spurs tri­al halt for Ma­gen­ta Ther­a­peu­tics

Magenta Therapeutics is pausing an early-stage clinical trial after a patient died. The death was deemed to be possibly related to its drug, MGTA-117.

The biotech said the pause of the Phase I/II trial is voluntary and gives it time to review all available data before deciding what to do next. It’s also reported the known information to the FDA.

The dose-escalation trial was designed to test whether MGTA-117, an antibody-drug conjugate, could serve as a more targeted alternative to high-intensity chemotherapy as a conditioning agent for cancer patients who are set to receive a stem cell transplant. It recruited patients with relapsed/refractory acute myeloid leukemia and myelodysplastic syndrome.

FDA re­ports ini­tial 'no sig­nal' for stroke risk with Pfiz­er boost­ers, launch­es con­comi­tant flu shot study

The FDA hasn’t detected any potential safety signals, including for stroke, in people aged 65 years and older who have received Pfizer’s bivalent Covid booster, one senior official told members of the agency’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Thursday.

The update comes as the FDA and CDC investigate a “preliminary signal” that may indicate an increased risk of ischemic stroke in older Americans who received Pfizer’s updated shot.

Al Gianchetti, XyloCor CEO

Xy­lo­Cor wraps up PhII for heart dis­ease gene ther­a­py, plans for piv­otal tri­al

XyloCor Therapeutics says patients with heart disease who got its gene therapy could exercise for longer and had fewer chest pain attacks. The biotech announced it completed a Phase I/II trial of the gene therapy Thursday morning, and plans to move forward with a pivotal trial.

In the Phase II portion of the trial, 28 patients with angina (or chest pain) caused by coronary artery disease and who had no other treatment options were enrolled and were given the highest tested dose from the first part of the trial. Patients were followed for six months.

Bris­tol My­ers claims win with CAR-T ther­a­py Breyanzi in leukemia

Bristol Myers Squibb is looking to expand Breyanzi into more indications — and the pharma’s newest data readout makes progress on that front.

The Big Pharma put out word Thursday that the CAR-T cell therapy met the primary endpoint of complete response rate compared to historical control in a subset of patients with relapsed or refractory chronic lymphocytic leukemia (CLL) that were refractory to a BTK inhibitor and pretreated with a BCL-2 inhibitor.

FDA cuts off use for As­traZeneca’s Covid-19 ther­a­py Evusheld

The FDA has stopped use of another drug as a result of the new coronavirus variants. On Thursday, the agency announced that AstraZeneca’s antibody combo Evusheld, which was an important prevention option for many immunocompromised people and others, is no longer authorized.

The FDA said it made its decision based on the fact that Evusheld works on fewer than 10% of circulating variants.

Evusheld was initially given emergency authorization at the end of 2021. However, as Omicron emerged, so did studies that showed Evusheld might not work against the dominant Omicron strain. In October, the FDA warned healthcare providers that Evusheld was useless against the Omicron subvariant BA.4.6. It followed that up with another announcement earlier this month that it did not think Evusheld would work against the latest Omicron subvariant XBB.1.5.

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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Vu Truong, Aridis Pharmaceuticals CEO (Aridis/Nasdaq)

Aridis' mon­o­clon­al an­ti­body fails PhI­II, but plans for sec­ond tri­al any­way

Aridis Pharmaceuticals’ monoclonal antibody missed the bar in a Phase III test in ventilator-associated pneumonia caused by the gram-positive bacteria S. aureus, the company announced Wednesday. 

But Aridis is planning for a second Phase III study anyway once it discusses the findings with the FDA and the European Medicines Agency. Execs blamed recruitment challenges stemming from Covid-19 and Russia’s invasion of Ukraine for the miss, cutting their enrollment target in half.