Saar Gill at ASCO 2017 via ASH on YouTube

Ris­ing Penn star Saar Gill gets fund­ing for a next-gen CAR-T start­up, adding a new weapon to the mix

Ear­li­er this month, the Uni­ver­si­ty of Penn­syl­va­nia’s Saar Gill turned up at AS­CO to present new da­ta demon­strat­ing that a com­bi­na­tion of Im­bru­vi­ca with No­var­tis’ next-gen CAR-T CTL119 proved very ef­fec­tive in treat­ing par­tic­u­lar­ly lethal cas­es of chron­ic lym­pho­cyt­ic leukemia, wip­ing out all signs of the dis­ease in 8 of 9 treat­ment-re­sis­tant pa­tients en­rolled in an ex­plorato­ry study.

But Gill’s been par­tic­u­lar­ly busy on the CAR-T front, and not just in new re­search work. He’s al­so been en­list­ing some mar­quee in­vestors on a new start­up project that will aim at mak­ing a ma­jor new ad­vance in the field.

Close­ly in­volved with CAR-T head­lin­er Carl June in Penn’s pi­o­neer­ing work with No­var­tis, which is now rac­ing to­ward a like­ly first ap­proval, Gill’s start­up is promis­ing to com­bine the proven ef­fi­ca­cy of reengi­neered T cells with macrophages, an­oth­er weapon in the fight against can­cer that could add to the fire pow­er of the first CAR-Ts with a spe­cial fo­cus on a chal­leng­ing fron­tier: sol­id tu­mors.

“We hy­poth­e­sized ear­ly on that fo­cus­ing on dif­fer­ent ef­fec­tor cells, oth­er than T cells, might be more ef­fec­tive in try­ing to tack­le sol­id tu­mors, which has been a chal­lenge for T cell ther­a­pies. The in­nate bi­ol­o­gy drove us to try macrophages,” Gill says.

CAR-T’s first and best ap­pli­ca­tion has been cen­tered on liq­uid tu­mors, with some ma­jor ob­sta­cles to over­come in ex­pand­ing in­to sol­id tu­mors. Now Gill has some un­spec­i­fied sup­port in a new round led by Ab­b­Vie Ven­tures and Health­Cap with par­tic­i­pa­tion by Grazia Eq­ui­ty and IP Group.

Gill’s com­pa­ny is called CAR­MA — a play­ful ab­bre­vi­a­tion of the chimeric anti­gen re­cep­tors added to T cells in CAR-T along with macrophages — and it’s iden­ti­fied a lead drug called CAR­MA-0508.

“Macrophages can en­gulf and kill cells through the process of phago­cy­to­sis. By ge­net­i­cal­ly en­gi­neer­ing these cells with CARs, we can specif­i­cal­ly di­rect them to tu­mor cells, such as ovar­i­an can­cer cells,” says co-founder Michael Klichin­sky, a PhD can­di­date in the de­part­ment of sys­tems phar­ma­col­o­gy and trans­la­tion­al ther­a­peu­tics at the Uni­ver­si­ty of Penn­syl­va­nia, in a pre­pared state­ment. “Our pre-clin­i­cal da­ta sup­port our hy­poth­e­sis and show tar­get­ed, se­lec­tive and ef­fec­tive killing of sol­id tu­mor cells by CAR­MA. In ad­di­tion, we ex­pect that CAR macrophages will prime a T cell im­mune re­sponse against the tu­mor.”

CAR­MA isn’t the on­ly new com­pa­ny to get start­ed out of Penn look­ing to play a big role in CAR-T’s sec­ond act. June al­so launched Tmu­ni­ty Ther­a­peu­tics, an­oth­er cell ther­a­py com­pa­ny, re­cruit­ing No­var­tis’ Us­man ‘Oz’ Azam to take the lead role at the com­pa­ny late last year.

Im­age: Saar Gill at AS­CO 2017 via ASH on YouTube

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.

Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.

Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

Biden ad­min­is­tra­tion backs a po­lar­iz­ing pro­pos­al to waive IP for all Covid-19 vac­cines

In a surprise U-turn, the Biden administration said Wednesday that it will support a proposal at the World Trade Organization to temporarily waive intellectual property protections on Covid-19 vaccines.

The proposal, backed by South Africa and India at the WTO, seeks to help developing countries with limited vaccine supplies. The US and Europe historically opposed the proposal, saying IP should be protected because it incentivizes new drug and vaccine development.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 104,800+ biopharma pros reading Endpoints daily — and it's free.