Months af­ter IPO, No­var­tis spin­out resTOR­bio wins on PhI­Ib da­ta — stock soars

Just six months af­ter its de­but on the Nas­daq, resTOR­bio is trot­ting out late-stage da­ta this morn­ing for its lead drug can­di­date — a TORC1 in­hibitor they’re hop­ing will pre­vent res­pi­ra­to­ry in­fec­tions in el­der­ly folks. While it flopped in a com­bo tri­al, the com­pa­ny’s drug met its pri­ma­ry end­point when test­ed by it­self.

In­vestors are thrilled with the news, with resTOR­bio’s share price $TORC fly­ing up 161% as of press time. Yes­ter­day’s close was at $9.01 per share, and to­day its so far climbed to $23.50. If it holds, that’s a de­cent jump from its IPO price of $14 to $16 per share.

Joan Man­nick

The drug — spun out of No­var­tis in­to its own start­up just last year — is now called RTB101, al­though it once went un­der the code name BEZ235. In a Phase IIb tri­al, the 10 mg dose of the drug showed a sta­tis­ti­cal­ly sig­nif­i­cant re­duc­tion in res­pi­ra­to­ry in­fec­tions when tak­en by pa­tients once a day. That co­hort saw a 30.6% de­crease in the per­cent­age of pa­tients who de­vel­oped a res­pi­ra­to­ry in­fec­tion when com­pared to the place­bo co­hort: p=0.026.

In­ter­est­ing­ly, the drug flopped big time when test­ed in com­bi­na­tion with an al­ready-ap­proved im­muno­sup­pres­sant called everolimus, an mTOR in­hibitor of­ten used to pre­vent the re­jec­tion of or­gan trans­plants. When resTOR­bio test­ed RTB101 com­bi­na­tion with everolimus, they found no de­crease in the per­cent­age of pa­tients with res­pi­ra­to­ry in­fec­tions. The drug al­so saw lousy re­sults when the 10 mg dose was tak­en twice dai­ly in­stead of once.

“We’re find­ing that less TORC1 in­hi­bi­tion works bet­ter than more TORC1 in­hi­bi­tion,” the com­pa­ny’s co-founder and CMO Joan Man­nick tells me. They saw a sim­i­lar re­sponse in their Phase IIa tri­al, she said.

Chen Schor

The com­pa­ny’s CEO Chen Schor said he wasn’t too con­cerned that the com­bo and dou­ble dos­es didn’t per­form well. In fact, the drug work­ing as a monother­a­py is ben­e­fi­cial.

“We pre­fer to move for­ward with the monother­a­py, be­cause it’s eas­i­er to man­u­fac­ture,” he said. “That was our hope.”

The com­pa­ny al­so homed in on a few spe­cif­ic pa­tient groups in which they saw more promis­ing re­sults. For ex­am­ple, in asth­ma pa­tients, they saw a 68% re­duc­tion in res­pi­ra­to­ry in­fec­tions (p=0.0002), and in pa­tients 85 years or old­er they saw 67% (p=0.007). The tri­al in­volved some 650 pa­tients, ac­cord­ing to clin­i­cal­tri­als.gov, but when resTOR­bio whit­tles down the pa­tient group to these se­lect pop­u­la­tions, we’re talk­ing much few­er peo­ple. I asked Man­nick just how many we’re talk­ing here. In asth­ma, it was on­ly 47 pa­tients, she said, and a mere 27 peo­ple were 85 years old and old­er.

But Man­nick de­fend­ed the da­ta’s sig­nif­i­cance re­gard­less of the small­er pa­tient group. “When you have few­er pa­tients, it’s much hard­er to reach sta­tis­ti­cal sig­nif­i­cance,” she said. “You have to have a big ef­fect.”

The com­pa­ny is meet­ing with the FDA at the end of this year to dis­cuss how it should move for­ward with a Phase III tri­al. Man­nick and Schor said the com­pa­ny will be shoot­ing for a sim­i­lar tri­al de­sign, in which they test co­horts of nar­row pa­tient groups with­in the same tri­al.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Gossamer Bio CEO Faheem Hasnain at Endpoints' #BIO22 panel (J.T. MacMillan Photography for Endpoints News)

Gos­samer’s Fa­heem Has­nain de­fends a round of pos­i­tive PAH da­ta as a clear win. But can these PhII re­sults stand up to scruti­ny?

Gossamer Bio $GOSS posted a statistically significant improvement for its primary endpoint in the key Phase II TORREY trial for lead drug seralutinib on Tuesday morning. But CEO Faheem Hasnain has some explaining to do on the important secondary of the crucial six-minute walk distance test — which will be the primary endpoint in Phase III — as the data on both endpoints fell short of expectations, missing one analyst’s bar on even modest success.

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Kristen Hege, Bristol Myers Squibb SVP, early clinical development, oncology/hematology and cell therapy (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: Bris­tol My­er­s' Kris­ten Hege on cell ther­a­py, can­cer pa­tients and men­tor­ing the next gen­er­a­tion

Kristen Hege leads Bristol Myers Squibb’s early oncology discovery program carrying on from the same work at Celgene, which was acquired by BMS in 2019. She’s known for her early work in CAR-T, having pioneered the first CAR-T cell trial for solid tumors more than 25 years ago.

However, the eminent physician-scientist is more than just a drug developer mastermind. She’s also a practicing physician, mother to two young women, an avid backpacker and intersecting all those interests — a champion of young women and people of color in STEM and life sciences.

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Pfiz­er and BioN­Tech look to toss Mod­er­na patent suit, call­ing claims 'unen­force­able'

Pfizer and BioNTech took a swing at Moderna’s Covid-19 patent claims in Massachusetts federal court on Monday, calling them “invalid,” “overbroad” and “unenforceable.”

The defendants also filed counterclaims against the Cambridge, MA-based biotech, seeking a dismissal of the case, recovery of court fees and an official judgment invalidating Moderna’s claims.

Moderna sued Pfizer and BioNTech back in August, alleging that the partners’ Covid-19 vaccine Comirnaty copied parts of Moderna’s vaccine technology patented before the pandemic, when it was developing an mRNA vaccine for MERS, another respiratory illness.

US sup­ports ex­ten­sion for Covid-19 IP waiv­er de­ci­sion

After much debate, the US government is now calling for a deadline extension to discuss a controversial potential IP waiver for Covid-19 diagnostics and therapeutics.

Over the last five months, the Office of the United States Trade Representative said it has consulted with members of Congress, public health advocates, organized labor groups, academics, think tanks, companies and trade associations on the WTO’s recent TRIPS agreement, which established a 5-year waiver of certain patent requirements on Covid-19 vaccines.

Klick Health is lighting the way, literally, this holiday season to encourage connection for lonely seniors in long-term care facilities.

Klick Health an­nu­al hol­i­day spot­light se­nior lone­li­ness and the pow­er of con­nec­tion

Every year Klick Health leans into a cause for the holidays, and this year it’s highlighting the sometimes lonely season for seniors. So Klicksters, as employees call themselves, decided to brighten one nursing home community in hopes of inspiring others to do the same.

Klick literally lit up the Tony Stacey Centre for Veterans Care, a long-term care home in Toronto where 75% of residents receive no visitors during the holiday season. The agency brought staff and family along with lighting crews and musicians for a “Light the Way” event, creating a video of the experience debuting on Tuesday.

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Sum­i­to­vant sub­sidiaries En­zy­vant and Al­ta­vant merge in­to com­bined com­pa­ny

Two Sumitovant Biopharma entities are merging under one name, effective immediately.

Enzyvant Therapeutics and Altavant Sciences announced they have merged to form a singular entity focused on developing therapies for patients with rare diseases. The combined company will keep the name Enzyvant and along with clinical development will eventually include in-house manufacturing.

Bill Symonds, the current CEO of both Altavant and Enzyvant, is now CEO of the merged company.

Eu­ro­pean Com­mis­sion lays ground­work to un­wind Il­lu­mi­na's $7B+ Grail merg­er

The European Commission has recommended steps that — though not yet final — would require Illumina to “swiftly” unwind its controversial $7.1 billion Grail buyout.

The Commission delivered a “statement of objections” on Monday, detailing the process Illumina would need to take in divesting Grail, its blood testing spinout launched in 2016. Illumina re-acquired Grail back in August, despite criticism from both the FTC and EU.

Rick Modi, Affinia Therapeutics CEO

Ver­tex-part­nered gene ther­a­py biotech Affinia scraps IPO plans

Affinia Therapeutics has ditched its plans to go public in a relatively closed-door market that has not favored Nasdaq debuts for the drug development industry most of this year. A pandemic surge in 2020 and 2021 opened the doors for many preclinical startups, which caught Affinia’s attention and gave the gene therapy biotech confidence in the beginning days of 2022 to send in its S-1.

But on Friday, Affinia threw in the S-1 towel and concluded now is not the time to step onto Wall Street. The biotech has put out few public announcements since the spring of this year. Endpoints News picked the startup as one of its 11 biotechs to watch last year.

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