TG Ther­a­peu­tics touts safer PI3Kδ in­hibitor as ex­ecs tout PhII da­ta, hunt quick OK for a tough niche

Eye­ing an ex­pe­dit­ed FDA ap­proval, TG Ther­a­peu­tics un­veiled da­ta from a Phase II PI3Kδ tri­al. They say it shows ef­fec­tive­ness against fol­lic­u­lar lym­phoma with­out the se­vere ad­verse ef­fects that have plagued PI3Kδ treat­ments to date.

Michael Weiss

TG re­port­ed that it met the goal of hav­ing 40-50% of the 118 fol­lic­u­lar lym­phoma pa­tients treat­ed with um­bral­is­ib re­spond to the drug. The sin­gle-agent study was specif­i­cal­ly de­signed to mod­el the stud­ies that brought ac­cel­er­at­ed FDA ap­proval for oth­er PI3Kδ drugs, TG CEO Michael Weiss said in a con­fer­ence call yes­ter­day.

The re­sults sent TG’s stock up 18% Mon­day.

“At 40-50% we have a drug that is ac­tive and will pro­vide ben­e­fit to pa­tients,’ Weiss said.

Those num­bers com­pare well or sim­i­lar­ly with re­sults from sim­i­lar Phase II tri­als be­hind three ap­proved PI3Kδ in­hibitors, Ve­rastem’s strug­gling Copik­tra, Gilead’s Zy­delig, and Bay­er’s Aliqopa. Weiss, though, ar­gued that the safe­ty pro­file is the most im­por­tant ques­tion in the PI3K mar­ket and will like­ly be his com­pa­ny’s biggest ad­van­tage.

“We’re talk­ing about chron­ic pa­tients,” Weiss said.  “You need to be able to treat them for a long pe­ri­od of time. It’s not all about hit­ting them with some very ag­gres­sive ther­a­py. In fact, I think the more mild ther­a­py is the ad­van­tage in this area.”

TG said the ther­a­py was “well tol­er­at­ed,” but the ex­act rate and sever­i­ty of ad­verse ef­fects won’t be known un­til TG un­veils the da­ta next year.

As in the pre­vi­ous stud­ies TG mod­eled, the pa­tients were re­lapsed, re­frac­to­ry and had al­ready tak­en at least an an­ti-CD20 mon­o­clon­al an­ti­body and an alky­lat­ing agent.

The search for an ef­fec­tive and safer P13K in­hibitor has ex­cit­ed and re­peat­ed­ly stymied big de­vel­op­ers, with weak ef­fi­ca­cy fail­ing to jus­ti­fy the drug’s ad­verse ef­fects.

Zy­delig has been ap­proved since 2014 and Copik­tra and Aliqopa fol­lowed in sub­se­quent years, but both have come with sig­nif­i­cant side ef­fects, in­clud­ing  em­bryo-fe­tal tox­i­c­i­ty, au­toim­mune tox­i­c­i­ties and po­ten­tial­ly life-threat­en­ing in­fec­tions

Last year, Roche and No­var­tis each dumped or passed off a PI3Kδ drug cit­ing safe­ty con­cerns and in Au­gust, No­var­tis sold an­oth­er PI3Kδ in­hibitor for a mod­est $20 mil­lion. See­ing a lim­it­ed mar­ket, in 2016, Genen­tech sold its in­hibitor for $5 mil­lion up­front and In­fin­i­ty sold the drug that be­came Copik­tra for $0 up­front, while Ab­b­Vie and As­traZeneca pulled out of the mar­ket.

Weiss said TG would speak with the FDA as soon as pos­si­ble about their ap­proval op­tions.

In his­toric Covid-19 ad­comm, vac­cine ex­perts de­bate a sea of ques­tions — but of­fer no clear an­swers

The most widely anticipated and perhaps most widely watched meeting in the FDA’s 113-year history ended late Thursday night with a score of questions and very few answers.

For nearly 9 hours, 18 different outside experts listened to public health agencies and foundations present how the United States’ Covid-19 vaccine program developed through October, and they debated where it should go from there: Were companies testing the right metrics in their massive trials? How long should they track patients before declaring a vaccine safe or effective? Should a vaccine, once authorized, be given to the volunteers in the placebo arm of a trial?

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.

A top drug pro­gram at Bay­er clears a high bar for CKD — open­ing the door to an FDA pitch

Over the past 4 years, Bayer has been steering a major trial through a pivotal program to see if their drug finerenone could slow down the pace of chronic kidney disease in patients suffering from both CKD as well as Type 2 diabetes.

Today, their team jumped on a virtual meeting hosted by the American Society of Nephrology to offer a solid set of pivotal data to demonstrate that the drug can delay dialysis or a kidney replacement as well as cardio disease, while also adding some worrying signs of hyperkalemia among the patients taking the drug. And they’re hustling it straight to regulators in search of an approval for kidney disease and cardio patients — one of the toughest challenges in the book, as demonstrated by repeated past failures.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.

Biond­Vax stock im­plodes af­ter a big PhI­II gam­ble for its uni­ver­sal flu vac­cine fails

After flying high on Wall Street for the last few months of a pandemic, BiondVax’s stock and dreams of getting approval for its universal flu vaccine hit the windshield.

The Jerusalem-based biotech announced on Friday that its only clinical candidate, M-001, failed both primary and secondary endpoints in a Phase III study. There was no statistically significant difference in reduction of flu illness and severity between the vaccine and placebo groups, according to the company. The vaccine did prove safe, if ineffective, BiondVax said.

Pascal Soriot, AstraZeneca CEO (Zach Gibson/Bloomberg via Getty Images)

UP­DAT­ED: FDA gives As­traZeneca the thumbs-up to restart PhI­II Covid-19 vac­cine tri­als, and J&J is prepar­ing to re­sume its study

Several countries had restarted their portions of AstraZeneca’s global Phase III Covid-19 vaccine trial after the study was paused worldwide in early September, but the US notably stayed on the sidelines — until now. Friday afternoon the pharma giant announced the all clear from US regulators. And on top of that, J&J announced Friday evening that it’s preparing to resume its own Phase III vaccine trial.

Ul­tragenyx in­jects $40M to grab Solid's mi­crody­s­trophin trans­gene — while side­step­ping the AAV9 vec­tor that stirred up safe­ty fears

Since before Ilan Ganot started Solid Bio to develop a gene therapy for kids like his son, who has Duchenne muscular dystrophy, Ultragenyx CEO Emil Kakkis has been watching and advising the former investment banker as he navigated the deep waters of drug development.

Just as Solid is getting back up on its feet after a yearlong clinical hold, Kakkis has decided to jump in for a formal alliance.

With a $40 million upfront, Ultragenyx is grabbing 14.45% of Solid’s shares $SLDB and the rights to its microdystrophin construct for use in combination with AAV8 vectors. Solid’s lead program, which utilizes AAV9, remains unaffected. The company also retains rights to other applications of its transgene.

Adam Koppel and Jeffrey Schwartz, Bain

Bain ex­ecs Adam Kop­pel and Jef­frey Schwartz line up $125M for their first blank check deal as Wall Street con­tin­ues to em­brace biotech

Adam Koppel and Jeffrey Schwartz have jumped into the blank check game, raising $125 million for a stock listing in search of a company.

Their SPAC, BCLS Acquisition Corp, raised $125 million this week, with a line on $25 million more as it scouts for a biotech in search of money and a place on Wall Street.

The two principals at Bain Life Sciences have been on a romp since they set up the Bain operation 4 years ago. Their S-1 spells out a track record of 22 deals totaling $650 million for the life sciences group, which led to 9 IPOs.

Covid-19 roundup: An mR­NA play­er gets a boost out of the lat­est round of an­i­mal da­ta; Phase­Bio pulls the plug on treat­ment tri­al

The big tell for CureVac $CVAC is coming up with a looming early-stage readout on their mRNA Covid-19 vaccine in the clinic. But for now they’ll make do with an upbeat assessment on the preclinical animal data they used to get into the clinic.

Researchers for the German biotech say they got the high antibody titers and T cell activation they were looking for, lining up a hamster challenge to demonstrate — in a simple model — that the vaccine could protect the furry creatures. Like the other mRNA vaccines, the drug sends instructions to spur cells to decorate themselves with the distinctive spike on the virus to elicit an immune response.

Dan O'Day, Gilead CEO (Andrew Harnik, AP Images)

UP­DAT­ED: FDA anoints Gilead­'s remde­sivir as the Covid-19 treat­ment win­ner, hand­ing down full ap­proval — de­spite some deep skep­ti­cism

Seven months into the Covid-19 pandemic, the race to develop a treatment for the disease that’s proved to be the biggest health crisis in a century has an officially designated winner: Gilead. And they’re picking up the prize — worth billions in peak sales — despite a major study that concluded the drug was no help in reducing the number of people who die from the virus.

The FDA handed down a thumbs-up for remdesivir, the company announced Thursday afternoon, as the drug becomes the first fully approved treatment for Covid-19 in the US. Remdesivir, to be marketed as Veklury, will come with a label for treatment in adults and children older than 12 in Covid-19 cases that require hospitalization.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 92,400+ biopharma pros reading Endpoints daily — and it's free.