AveX­is wants $400M more for promis­ing SMA gene ther­a­py; Shang­Phar­ma part­ners with Ic­ahn School of Med­i­cine at Mount Sinai

AveX­is $AVXS is rais­ing more cash to fu­el its gene ther­a­py for spinal mus­cu­lar at­ro­phy with a pub­lic of­fer­ing of up to $400 mil­lion of shares at $117.5 per share. The plan, laid out in an SEC fil­ing, is to al­lo­cate $225 to $270 mil­lion to re­search, man­u­fac­tur­ing, clin­i­cal and reg­u­la­to­ry ac­tiv­i­ties — main­ly in SMA, but al­so for pro­grams in Rett syn­drome and ALS. Around $140 mil­lion to $175 mil­lion will go to pre-com­mer­cial ac­tiv­i­ties for the po­ten­tial launch of SMA block­buster con­tender AVXS-101, in­clud­ing med­ical af­fairs, com­mer­cial ini­tia­tives and li­cens­ing ac­tiv­i­ties. The Chica­go-based biotech is com­ing on strong in the SMA field, threat­en­ing to cut in­to Bio­gen’s Spin­raza fran­chise.

Shang­Phar­ma In­no­va­tion, a VC firm that fo­cus­es on seed in­vest­ments, has struck a deal with Ic­ahn School of Med­i­cine at Mount Sinai to fu­el re­search be­hind ear­ly-stage drugs. Un­der the mul­ti-year col­lab­o­ra­tion deal, Shang­Phar­ma will pro­vide R&D fund­ing at the school’s clin­i­cal in­sti­tutes in New York. Led by CEO Wal­ter Moos, Shang­Phar­ma will al­so pro­vide re­search ser­vices from CRO Shang­hai ChemPart­ner. “This part­ner­ship was fu­eled by the ground­break­ing tech­nolo­gies from in­no­va­tors here at Mount Sinai,” said Erik Li­um, se­nior VP of Mount Sinai In­no­va­tion Part­ners, in a state­ment. “Our ex­per­tise in com­mer­cial­ly-rel­e­vant trans­la­tion­al re­search, cou­pled with Shang­Phar­ma In­no­va­tion’s drug de­vel­op­ment ca­pa­bil­i­ties will ad­vance much need­ed med­i­cines to pa­tients world­wide.”

→ Lon­don’s Hik­ma Phar­ma­ceu­ti­cals has re­cruit­ed Suren­dera Tya­gi as its new CSO and glob­al head of R&D. Tya­gi comes from Ger­man heath­care gi­ant Fre­se­nius Kabi (ETR: FRE), where he led the com­pa­ny’s US In­no­va­tion & De­vel­op­ment Cen­ter and ex­pand­ed the Fre­se­nius’ on­col­o­gy busi­ness. At Hik­ma, Tya­gi will be first tasked with boost­ing non-in­jectable R&D to ex­pand the com­pa­ny’s pipeline. The hire comes at a time when Hik­ma’s gener­ics unit is tak­ing some hits, lead­ing to the com­pa­ny’s stock (LON:HIK) falling 47% over the past year. Tya­gi’s re­cruit­ment may be part of the com­pa­ny’s strat­e­gy to re­cov­er through di­ver­si­fi­ca­tion in the pipeline. Be­fore Fre­se­nius Kabi, Tya­gi worked at Dar­bur Phar­ma, Roche, IGEN, Ab­bott, and Schwarz Phar­ma.

Pfiz­er $PFE is team­ing up with Foun­da­tion Med­i­cine $FMI to de­vel­op, pro­vide reg­u­la­to­ry sup­port, and help com­mer­cial­ize com­pan­ion di­ag­nos­tics for Pfiz­er’s on­col­o­gy port­fo­lio. Foun­da­tion Med­i­cine makes an FDA-ap­proved ge­nom­ic pro­fil­ing test for sol­id tu­mors called Foun­da­tionOne CDx, which the col­lab­o­ra­tion hopes to im­prove on. The com­pa­ny al­so makes an­a­lyt­ics soft­ware called Foun­da­tion­In­sights, which Pfiz­er can lever­age to ac­cel­er­ate dis­cov­ery and de­vel­op­ment of can­cer drugs. Pfiz­er cur­rent­ly has 10 FDA-ap­proved can­cer med­i­cines that treat sol­id tu­mors and hema­to­log­ic ma­lig­nan­cies. Its on­col­o­gy pipeline al­so in­cludes 17 as­sets in clin­i­cal de­vel­op­ment and 19 Phase III stud­ies.

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

As­traZeneca touts Imfinzi im­munother­a­py com­bos for lung can­cer in push to dri­ve PD-L1 drug up­take

Facing the big dogs in the PD-(L)1 space, AstraZeneca has taken its own contender Imfinzi into blockbuster territory in its four years on the market but sees even bigger things for the drug. Combinations could be the key, and early results from a mid-stage test are adding some fuel to that strategy.

Imfinzi combined with one of two investigational immunotherapies — a CD73 antibody dubbed oleclumab or an anti-NGK2a named monalizumab — topped Imfinzi alone in terms of overall response and progression-free survival in patients with stage III non-small cell lung cancer whose tumors had not worsened during concurrent chemoradiation, according to interim data from the Phase II COAST trial set to be presented at #ESMO21.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,500+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,500+ biopharma pros reading Endpoints daily — and it's free.

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.