Mi­rati and MD An­der­son team up on KRAS; Sanofi inks deal with a Har­vard spin­out for oral bi­o­log­ics

Mi­rati, the oth­er KRAS-fo­cused biotech, is get­ting some ex­tra am­mo as it looks to leapfrog Am­gen in the hunt for the first, most ef­fec­tive and most broad­ly ef­fec­tive for one of the most ubiq­ui­tous and hard-to-hit onco­genes.

The Cal­i­for­nia biotech has signed a re­search and de­vel­op­men­tal with MD An­der­son to help ad­vance both Mi­rati’s lead KRAS G12C mol­e­cule — the one that’s now proven ef­fec­tive in cer­tain lung can­cer pa­tients — and a pre­clin­i­cal mol­e­cule that goes af­ter an­oth­er KRAS mu­ta­tion, G12D. The Texas-based re­search in­sti­tute will help Mi­rati de­sign and run pre­clin­i­cal and clin­i­cal stud­ies for sev­er­al sol­id tu­mors, in­clud­ing non-small cell lung, pan­cre­at­ic, col­orec­tal and gy­ne­co­log­i­cal can­cers, over the next 5 years.

Al­though trail­ing Am­gen for the first ap­proval, both com­pa­nies are still work­ing to show how ef­fec­tive a KRAS drug can be in lung can­cer and, cru­cial­ly, in oth­er tu­mors, where Am­gen has on­ly shown mixed re­sults

“Ef­fec­tive tar­get­ed ther­a­pies against mu­tant KRAS could ad­dress a ma­jor un­met need for many pa­tients,” Christo­pher Flow­ers, in­ter­im head of Can­cer Med­i­cine at MD An­der­son, said in a state­ment. “Our col­lab­o­ra­tion with Mi­rati rep­re­sents an im­por­tant op­por­tu­ni­ty to work to­ward ad­vanc­ing new treat­ment op­tions for pa­tients us­ing nov­el KRAS in­hibitors that tar­get two of the most fre­quent KRAS mu­ta­tions in com­mon can­cers.” — Ja­son Mast

Sanofi en­lists i2O in the hunt for re­al­ly small drugs

Just ten months af­ter they spun out from Har­vard, i2O Ther­a­peu­tics has a big name col­lab­o­ra­tor: Sanofi.

The French phar­ma en­list­ed the small­er biotech to help them de­liv­er nanobod­ies — tiny ver­sions of an­ti­bod­ies that can be just as ac­cu­rate but po­ten­tial­ly con­fer a range of ad­van­tages, in­clud­ing bet­ter abil­i­ty to pen­e­trate tis­sues, greater scal­a­bil­i­ty, and a longer half-life. They’ve got at­ten­tion as po­ten­tial Covid-19 drugs, but Sanofi sees po­ten­tial in a wide va­ri­ety of fields, in­clud­ing di­a­betes and im­muno-on­col­o­gy.

i2O makes oral forms of bi­o­log­ics by en­cas­ing them in a kind of ion­ized slush as op­posed to wa­ter. They will help Sanofi re­search ways of mak­ing pill forms of the nanobod­ies.  — Ja­son Mast

As­traZeneca lends a hand with Hong Kong’s new biotech in­cu­ba­tor

Af­ter carv­ing out a valu­able fran­chise in Chi­na’s on­col­o­gy mar­ket, As­traZeneca is now help­ing set up a new in­cu­ba­tor in Hong Kong for biotech star­tups look­ing to hatch new can­cer drugs and di­ag­nos­tics.

The phar­ma gi­ant teamed with Hong Kong Sci­ence and Tech­nol­o­gy Parks, of­fer­ing sup­port ser­vices to the star­tups that are ac­cept­ed. The fledg­lings in turn gain ac­cess to cash and lab ser­vices as well as an ex­tend­ed set of sup­port ser­vices to help guide the ini­tial phase of their work.

Al­fred Sit, the sec­re­tary for in­no­va­tion and tech­nol­o­gy, had this to say:

This co-in­cu­ba­tion col­lab­o­ra­tion is a great leap in the biotech field of the lo­cal ecosys­tem. By cre­at­ing, as I would de­scribe, a unique “one-stop so­lu­tion in­te­gra­tion plat­form” for the whole course of ac­tion in com­bat­ing can­cer from di­ag­no­sis, to mon­i­tor­ing and treat­ment, the col­lab­o­ra­tion is a break­through in re­search on on­col­o­gy, which is cur­rent­ly con­duct­ed in a seg­men­tal man­ner pre­dom­i­nant­ly by look­ing in­to dis­crete ac­tions against can­cer. This will make re­search on on­col­o­gy more con­sol­i­dat­ed, stand­ing a high­er chance of ac­tu­al­is­ing new and bet­ter op­tions to fight against the life-threat­en­ing dis­ease.

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Voting in the 2020 election (AP Images)

The right to vote is fun­da­men­tal — a let­ter from biotech­nol­o­gy in­dus­try lead­ers

Biotech Voices is a collection of exclusive opinion editorials from some of the leading voices in biopharma on the biggest industry questions today. Think you have a voice that should be heard? Reach out to senior editors Kyle Blankenship and Amber Tong.

We oppose all attempts to introduce laws that reduce the rights of US citizens to vote or that restrict them from exercising that right. The right to vote is fundamental to democracy. States that have enacted, or are proposing to enact, legislation to restrict voting are undermining our democracy and posing a threat to our nation. As leaders of the life sciences industry, we stand for what we believe is right for our country, our enterprises, our employees and those who benefit from our work. We join the first groups of business leaders who have challenged these laws and will continue to make our collective voices heard on this matter.

Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Launched by MIT grads, a small start­up gets $20M to back a ro­bot­ics rev­o­lu­tion in cell ther­a­py man­u­fac­tur­ing

As co-director of an experimental cellular therapy process development and manufacturing group at UCSF specializing in T cell therapies for autoimmune conditions, Jonathan Esensten has learned a lot about the challenges involved when his group hand-fashions a cell therapy. Esensten — who was a postdoc in Wendell Lim’s lab and counts the legendary Jeffrey Bluestone as a mentor — gives them all high marks at being great at what they do, but time and again there are variations in the treatments they construct.

Anand Shah (FDA)

For­mer head of FDA’s med­ical and sci­en­tif­ic af­fairs on Covid: ‘FDA has nev­er been test­ed like this’

Anand Shah has served the American public in a unique way, crisscrossing over the last two administrations between serving as an attending radiation oncologist focused on prostate cancer at NIH, serving as CMO at the Center for Medicare and Medicaid Innovation, and most recently, leading the FDA’s operations on medical and scientific affairs from within the commissioner’s office.

Shah, who stepped down from the FDA in January, caught up with Endpoints News in a phone interview on Tuesday afternoon, offering his thoughts on the agency’s latest decision to pause the J&J vaccinations in the US, and reflecting on his time at an agency during this once-in-a-lifetime pandemic.

UP­DAT­ED: J&J paus­es vac­cine roll­out as feds probe rare cas­es of blood clots

The FDA and CDC have jointly decided to stop administering J&J’s Covid-19 vaccine after reviewing data involving six reported US cases of a rare and severe type of blood clot in individuals after receiving the vaccine.

CDC will convene a meeting of its Advisory Committee on Immunization Practices on Wednesday to further review these cases and assess their potential significance. “FDA will review that analysis as it also investigates these cases. Until that process is complete, we are recommending a pause in the use of this vaccine out of an abundance of caution,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research and Anne Schuchat, Principal Deputy Director of the CDC, said in a joint statement Tuesday morning.

Patrizia Cavazzoni, new CDER director

Pa­trizia Cavaz­zoni named per­ma­nent di­rec­tor of CDER, adding to ques­tions around where Wood­cock will end up

Patrizia Cavazzoni on Monday became the permanent director of the FDA’s Center for Drug Evaluation and Research, which puts to rest the idea that Janet Woodcock, Cavazzoni’s predecessor, might return to lead CDER if she isn’t made permanent commissioner.

Woodcock, who’s currently serving as acting commissioner and principal medical advisor to the commissioner, a position she was detailed to last year, may not make the move to permanent commissioner because of lingering questions from Senate Democrats. She previously served as director of CDER since 1994. Cavazzoni took over as acting director of CDER when Woodcock moved over to Operation Warp Speed to run the therapeutics side of the Trump-era program.

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Barbara Weber, Tango Therapeutics CEO (Tango)

It takes two to Tan­go: The biotech us­ing CRISPR to dis­cov­er new can­cer gene tar­gets rides a $353M SPAC deal to Nas­daq

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

The latest biotech-SPAC deal has arrived, and it’s dancing its way to Nasdaq to the tune of several hundred million dollars.

Tango Therapeutics and its CRISPR-focused search for new cancer genes is reverse merging with Boxer Capital’s blank-check company, the biotech announced Wednesday morning. With a spotlight on three lead programs, Tango expects total proceeds to equal about $353 million in the deal, which includes the roughly $167 million held in the SPAC and an additional $186 million in PIPE financing.