Tyme flops on can­cer drug da­ta; Lil­ly IND fil­ing trig­gers $8M pay­ment to part­ner Zymeworks

Tyme Tech­nolo­gies on Fri­day tout­ed “sub­stan­tial­ly im­proved sur­vival ben­e­fit” from its ex­per­i­men­tal drug SM-88 in a sin­gle arm study in ad­vanced pan­cre­at­ic can­cer pa­tients with pro­gres­sion de­spite treat­ment with at least two pri­or ther­a­pies, but in­vestors did not buy in­to the com­pa­ny’s en­thu­si­asm, caus­ing the stock $TYME to crater about 36%. The tri­al was de­signed to en­roll 115 pa­tients, in­clud­ing 36 pa­tients in the ini­tial stage of the tri­al, ac­cord­ing to clin­i­cal­tri­als.gov, and the pri­ma­ry end­point of the study is re­sponse rate — sug­gest­ing Tyme was be­ing mis­lead­ing about the “sur­vival ben­e­fit” it was bran­dish­ing. The com­pa­ny elect­ed to high­light that 68% of pa­tients treat­ed with SM-88, (19 of 28) re­mained alive af­ter a me­di­an of 4.3 months of fol­low-up. But, on­ly 17 pa­tients had un­der­gone tu­mor scans, and the re­sponse rate, that would come from tu­mor scans, was re­vealed much low­er down in the re­lease — 12% of pa­tients (2 out of 17) saw tu­mor re­duc­tions of more than 30%. As point­ed out by STAT’s Matthew Her­p­er, the study raised im­por­tant red flags, most im­por­tant­ly that it was im­pos­si­ble to con­clude sur­vival ben­e­fit based on the avail­able da­ta.

Zymeworks has col­lect­ed $8 mil­lion in mile­stone pay­ments as Eli Lil­ly sub­mits an IND for an im­muno-on­col­o­gy bis­pe­cif­ic an­ti­body de­vel­oped on its plat­form — the sec­ond with­in a six-month pe­ri­od, the com­pa­ny high­lights. The part­ners’ li­cens­ing and col­lab­o­ra­tion agree­ment date back to 2014.

→ Pulling off the biggest biotech IPO ($604 mil­lion) of 2018 was enough for Mod­er­na $MR­NA pres­i­dent John Mendlein, who is tran­si­tion­ing to the role of ex­ec­u­tive part­ner at Flag­ship Pi­o­neer­ing.  

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Bomb squad called to As­traZeneca vac­cine plant; Lu­men nabs CARB-X award for low-cost an­tidiar­rheal

A plant located in Wrexham, Wales that is packing the Oxford/AstraZeneca Covid-19 vaccine into vials was surrounded by a bomb squad after officials called police to report a suspicious package.

The alert caused a partial evacuation of the plant, the BBC was among those to report Wednesday. The owner of the plant, British drugmaker Wockhardt UK, said it was cooperating with local authorities and that there were no reports of any injuries.

Pascal Soriot, AP

As­traZeneca CEO Pas­cal So­ri­ot sev­ers an un­usu­al board con­nec­tion, steer­ing clear of con­flicts while re­tain­ing im­por­tant al­liances

CSL Behring chief Paul Perreault scored an unusual coup last summer when he added AstraZeneca CEO Pascal Soriot to the board, via Zoom. It’s rare, to say the least, to see a Big Pharma CEO take any board post in an industry where interests can simultaneously connect and collide on multiple levels of operations.

The tie set the stage for an important manufacturing connection. The Australian pharma giant agreed to supply the country with 10s of millions of AstraZeneca’s Covid-19 vaccine, once it passes regulatory muster.

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Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Anthony Fauci, NIAID director (AP Images)

As new Covid-19 task force gets un­der­way, threat looms of vac­cine, mon­o­clon­al an­ti­body-re­sis­tant vari­ants

Hours before President Biden’s Covid-19 team gave their first virtual press conference, the famed AIDS researcher David Ho delivered concerning news in a new pre-print: SARS-CoV-2 B.1.351, the variant that emerged in South Africa, is “markedly more resistant” to antibodies from convalescent plasma and vaccinated individuals.

The news for several monoclonal antibodies, including Eli Lilly’s bamlanivimab, was even worse: Their ability to neutralize was “completely or markedly abolished,” Ho wrote. Lilly’s antibody cocktail, which was just shown to dramatically reduce the risk of hospitalizations or death, also became far less potent.

Florian Brand (L) and Srinivas Rao (ATAI)

Psy­che­del­ic biotech ATAI hopes to ex­pand port­fo­lio through re­search part­ner­ship with Mass Gen­er­al

Psychedelics have made a comeback for mental health research, with companies like startup biotech ATAI Life Sciences raising millions and earning the backing of prominent investors like Peter Thiel, but there’s a hole at the heart of the resurgence: Researchers still don’t fully understand how they work.

A new partnership between ATAI and world-renowned Mass General Hospital hopes to change that.