Ar­row­head touts Phase Ib RNAi da­ta for ccR­CC; PureTech lines up PD-1 from BeiGene for up­com­ing com­bo study

Just days af­ter get­ting wal­loped by the news that an­i­mal tox da­ta forced them to halt an ear­ly-stage cys­tic fi­bro­sis study, Ar­row­head is tout­ing some ini­tial snap­shots from their RNAi treat­ment for clear cell re­nal cell car­ci­no­ma.

Most of the pos­i­tive signs they’re point­ing to re­late to bio­mark­er ac­tiv­i­ty, with an av­er­age 48% re­duc­tion in HIF2α pro­tein among nine evalu­able biop­sy sam­ples. Those re­duc­tions var­ied wide­ly, though, start­ing at 9% and grow­ing to 82%.

There was one par­tial tu­mor re­sponse in the small group, though re­searchers not­ed the pa­tients had failed mul­ti­ple lines of ther­a­py. — John Car­roll

PureTech lines up a PD-1 from BeiGene for up­com­ing com­bo study

PureTech has struck a sup­ply deal with BeiGene to line up their PD-1 drug tislelizum­ab for a com­bi­na­tion study in­volv­ing LYT-200, an ex­per­i­men­tal an­ti­body tar­get­ing galectin-9 for dif­fi­cult-to-treat sol­id tu­mors.

This is part of a two-step ef­fort to test PureTech’s drug as a monother­a­py first, fol­lowed by the com­bi­na­tion with tislelizum­ab and oth­er agents.

“Af­ter a decade of op­ti­miz­ing use of im­muno-on­col­o­gy ther­a­pies, such as the check­point in­hibitors that have cer­tain­ly pro­vid­ed a par­a­digm shift in treat­ing ma­lig­nant dis­eases, we in the in­dus­try are ea­ger to ad­vance nov­el break­through agents and com­bi­na­tions to serve a wider range of can­cer pa­tients. Pa­tients need new op­tions as the first wave of im­munother­a­pies work on­ly in a small per­cent­age of them. Well thought-out com­bi­na­tion im­munother­a­py reg­i­mens may be that way for­ward,” said Alek­san­dra Fil­ipovic, head of on­col­o­gy at PureTech. — John Car­roll

Bio­haven re­ports bet­ter than ex­pect­ed sales on mi­graine drug, shares jump

Bio­haven $BHVN saw its shares tick up­ward Wednes­day morn­ing af­ter sharply beat­ing sales es­ti­mates for its mi­graine drug Nurtec, fol­low­ing a re­cent new ap­proval as a pre­ven­ta­tive.

Shares of the New Haven, CT-based biotech were trad­ing high­er by as much as 13% in ear­ly Wednes­day trad­ing af­ter Nurtec reaped near­ly $93 mil­lion in sales in the sec­ond quar­ter. Orig­i­nal­ly ap­proved in Feb­ru­ary 2020 to treat acute mi­graines, Nurtec tacked on pre­ven­ta­tive treat­ment this past May.

“Our sec­ond quar­ter net rev­enue per­for­mance re­flects the over­whelm­ing­ly pos­i­tive feed­back we are re­ceiv­ing from pa­tients and health­care providers about the im­pact of Nurtec ODT on mi­graine,” CCO BJ Jones said in a state­ment.

Bio­haven says its oral CGRP drug cov­ers about 95% of avail­able mi­graine pa­tients. It’s like­ly to face com­pe­ti­tion from Ab­b­Vie’s Ubrelvy, which launched just a cou­ple of months be­fore Nurtec in ear­ly 2020. — Max Gel­man

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Amgen VP of R&D David Reese

Am­gen rolls out da­ta for KRAS in­hibitor com­bo study in col­orec­tal can­cer, hop­ing to move on from ug­ly ear­ly re­sults

With the first win for its KRAS inhibitor sotorasib in hand, Amgen is pushing ahead with an aggressive clinical plan to capitalize on its first-to-market standing. The drugmaker thinks combinations — in-house or otherwise — could offer a path forward, and one early readout from that strategy is bearing fruit.

A combination of Amgen’s sotorasib and its EGFR inhibitor Vectibix posted an overall response rate of 27% in 26 patients with advanced colorectal cancer (CRC) with the KRAS-G12C mutation, according to data from the larger Phase Ib/II CODEBREAK 101 study set to present at this weekend’s virtual ESMO Congress.

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Dan O'Day, Gilead CEO (Jim Watson/AFP via Getty Images)

Eu­ro­pean study finds that Gilead­'s Covid-19 an­tivi­ral remde­sivir shows no clin­i­cal ben­e­fit

Gilead’s remdesivir — or Veklury, as it’s marketed in the US — raked in around $2.8 billion last year as the only FDA-approved antiviral to treat Covid-19. But new data from a European study suggest the drug, which has been given to about half of hospitalized Covid patients in the country, has no actual benefit.

The open-label DisCoVeRy trial enrolled Covid-19 patients across 48 sites in Europe to test a handful of treatments, including remdesivir, lopinavir–ritonavir, lopinavir–ritonavir and interferon beta-1a, and hydroxychloroquine. To participate, patients had to show symptoms for seven days and require oxygen support. A total of 429 patients were randomized to receive remdesivir plus standard of care, while 428 were assigned to standard of care alone.

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Gri­fols drops $1B on Ger­man hold­ing com­pa­ny in con­tin­ued plas­ma push

One Spanish biotech is beefing up its plasma therapy operations, and on Friday, it announced that it’s doing so in a billion-dollar deal.

Grifols is now the largest shareholder of Biotest, a company valued at more than $1.8 billion. By teaming up, the two will try to increase the number of plasma therapies available and increase patient access around the world, Grifols said in a press release.

The company did so by acquiring holding company Tiancheng Pharmaceutical, the Germany-based owner of nearly 90% of Biotest shares, for nearly $1.27 billion. Grifols now owns nearly 90% of Biotest voting rights and almost 45% of the total share capital of Biotest.

Covid-19 roundup: FDA re­veals boost­er ad­comm ques­tion; Eli Lil­ly's an­ti­body cock­tail cleared for pre­ven­tion

The FDA released briefing documents this week from the agency and Pfizer each outlining their arguments for today’s Covid-19 booster shot adcomm, but one thing conspicuously missing was the question on which panel members would be voting. But late Thursday night, regulators published that question.

Adcomm members will be asked whether or not the safety and efficacy data from Pfizer/BioNTech’s original Phase III study “support approval” of a booster shot at least six months after the second dose in individuals older than 16. The question notably excludes the real-world data from Israel and other analyses that Pfizer and the Biden administration had said would be a centerpiece of their arguments for boosters.

A Pfiz­er part­ner wel­comes ex-ADC Ther­a­peu­tics CMO Jay Fein­gold to the team; Amid tough sled­ding, Im­muno­vant choos­es Eli Lil­ly alum as CFO

→ Last week we told you about the CMO revolving door at ADC Therapeutics, as Joseph Camardo replaced the departing Jay Feingold. The next opportunity for Feingold in the CMO slot has opened up at antibody-drug conjugate and mAb developer Pyxis Oncology, which has added several new execs and scientific advisory board members in recent months, including ex-Immunovant CFO Pamela Yanchik Connealy. Before his tenure at ADC, Feingold was Daiichi Sankyo’s VP of US medical affairs and chairman of the Global Medical Affairs Oversight Committee. Within weeks in March, Pyxis struck a licensing deal with Pfizer for two of its ADCs and raked in $152 million from a Series B round.

FDA ac­cepts In­tel­li­a's IND for CRISPR and TCR-T cell ther­a­py; San­té clos­es Fund IV at $260M

Riding the coattails of a massive $600 million cash raise in June, Intellia announced that the FDA accepted their IND application for their gene editing treatment NTLA-5001, built as a treatment for acute myeloid leukemia.

The Cambridge, MA biotech said that they have plans to start patient screening in a Phase I/IIa study by the end of 2021. The study will evaluate the effects of a single dose of the treatment in adults who have detectable AML after having received standard first-line therapy. The study will contain a dose escalation and expansion phase, with up to 54 participants.

Multiple antibiotic resistant Pseudomonas aeruginosa bacterium

A new way to in­fil­trate (and de­stroy) some of the dead­liest drug-re­sis­tant bugs

About four years ago, Ruben Tommasi, the gregarious scientific chief of antibiotics startup Entasis, walked into a meeting with his top chemist and top biologist to chew over another batch of unchanging results.

“It felt like we were running the same experiment over and over,” Tommasi told Endpoints News. “We had all sort of come to that point in time where we felt like we were banging our heads against the wall.”

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