Take­da re­ceives ad­di­tion­al FDA ap­proval for GI drug; Can­cer Re­search UK to test gem­c­itabine for ad­vanced pan­cre­at­ic can­cer

→ The FDA has giv­en the green light to Take­da to ex­tend­ed the in­di­ca­tion of its drug Gat­tex (tedug­lu­tide) for in­jec­tion to pe­di­atric pa­tients 1 year of age and old­er with short bow­el syn­drome (SBS) who need ad­di­tion­al nu­tri­tion or flu­ids from in­tra­venous (IV) feed­ing (par­enter­al sup­port).

→ More than a decade af­ter the FDA first ap­proved Frag­min to treat blood clots in adults, Pfiz­er has won an OK to ex­tend its use in pe­di­atric pa­tients as young as one month old. There’d been no FDA-ap­proved ther­a­pies to treat ve­nous throm­boem­bolism in this pop­u­la­tion, Richard Paz­dur said in a state­ment, who are of­ten suf­fer­ing from a se­ri­ous un­der­ly­ing con­di­tion such as can­cer and con­gen­i­tal heart dis­ease. The ap­proval was based on a sin­gle tri­al with 38 pa­tients, in which “21 pa­tients achieved res­o­lu­tion of the qual­i­fy­ing VTE, sev­en pa­tients showed re­gres­sion, two pa­tients showed no change, no pa­tients ex­pe­ri­enced pro­gres­sion of the VTE and one pa­tient ex­pe­ri­enced re­cur­rence of VTE.”

→ Af­ter whip­ping up some buzz around its gene ther­a­py for “bub­ble boy dis­ease” with ear­ly but hope­ful da­ta, Mus­tang Bio has scored an or­phan drug des­ig­na­tion for an­oth­er as­set in its pipeline: an on­colyt­ic virus to treat ma­lig­nant glioma, an ag­gres­sive type of brain can­cer. The New York-based biotech is li­censed the pro­gram from Na­tion­wide Chil­dren’s Hos­pi­tal, with the in­ten­tion to com­bine it with its an­ti-CD213a2 CAR-T ther­a­py.

→ The Can­cer Re­search UK an­nounced to­day that it will be­gin a clin­i­cal tri­al to test gem­c­itabine in ad­vanced pan­cre­at­ic can­cer. The or­ga­ni­za­tion says that “the study will use PLX7486 to tar­get Trk ki­nas­es, which are be­lieved to help can­cer cells in­vade nerves. It’s hoped that PLX7486, in com­bi­na­tion with the chemother­a­py drug gem­c­itabine, could im­prove pro­gres­sion-free sur­vival and re­duce pain for pan­cre­at­ic can­cer pa­tients.” The tri­al will be con­duct­ed through the Ex­per­i­men­tal Can­cer Med­i­cine Cen­tre (ECMC) net­work, sup­port­ed by Can­cer Re­search UK and the UK Health De­part­ments and let by the Man­ches­ter ECMC and spon­sored by the Uni­ver­si­ty of Glas­gow Clin­i­cal Tri­als Unit. The first pa­tients can ex­pect to start treat­ments in ear­ly June and the tri­al will span four sites across the UK.

Na­tal­ie Cook, chief in­ves­ti­ga­tor of the tri­al and clin­i­cal lead of the Man­ches­ter ECMC, said: “Pre­clin­i­cal mod­els show that giv­ing this drug com­bi­na­tion seems ef­fec­tive at stop­ping the can­cer cells grow­ing and mul­ti­ply­ing. Hope­ful­ly, this will trans­late in­to clin­i­cal ben­e­fits and en­able us to bet­ter con­trol the pain and spread of can­cer in pa­tients.”


Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology

ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development

CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

UP­DAT­ED: As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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SEC calls out lit­tle Ther­a­peu­tic­sMD for its in­sid­er con­tacts with an­a­lysts to boost share price, then halt rout

Back in May 2017, following an FDA rejection, TherapeuticsMD saw its share price plummet to the lowest levels in two years. The little Florida biotech eventually found its way back to the good side of regulators, scoring a curious OK a year later for its therapy preventing vaginal pain during sex. But the SEC is now accusing it of selectively disclosing nonpublic information in attempts to manipulate its stock.

In two instances in June and July of 2017, TherapeuticsMD allegedly violated the Regulation Fair Disclosure rule by sharing material information with certain sell-side analysts and not the public, resulting in a more favorable stock move than otherwise would be expected.

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Therapists Marcela Ot'alora and Bruce Poulter are trained to conduct MDMA-assisted psychotherapy. In this reenactment, they demonstrate how they help guide and watch over a patient who is revisiting traumatic memories while under the influence of MDMA. (Photo: Multidisciplinary Association for Psychedelic Studies)

MD­MA, now in Phase III, shows promise as a PTSD treat­ment

The first time Lori Tipton tried MDMA, she was skeptical it would make a difference.

“I really was, at the beginning, very nervous,” Tipton said.

MDMA is the main ingredient in the club drug known as ecstasy or molly. But Tipton wasn’t taking pills sold on the street to get high. She was trying to treat her post-traumatic stress disorder by participating in a clinical trial.

After taking a dose of pure MDMA, Tipton lay in a quiet room with two specially trained psychotherapists. They sat next to her as she recalled some of her deepest traumas, such as discovering her mother’s body after Tipton’s mother killed two people and then herself in a murder-suicide.

Ted Ashburn. Oncorus

Cowen, Per­cep­tive lead $79.5M Se­ries B for 's­tand­out' biotech shep­herd­ing on­colyt­ic virus to clin­ic

As several Big Pharma players secure biotech partners in the oncolytic virus space for new immuno-oncology combos, Cowen and Perceptive Advisors have come out with their own bet on a startup that promises to shine.

The marquee investors are joining MPM, Deerfield, Celgene, Astellas, Arkin Bio Ventures and UBS Oncology Impact Fund in backing the drug developer, Oncorus, which will now deploy the $79.5 million in Series B cash toward clinical development of its lead program. Other new investors include Surveyor Capital, Sphera Funds, IMM Investment, QUAD Investment Management, UTC Investment, SV Investment Corp and Shinhan Investment-Private Equity, the last five of which are Korean-based funds.

Fu­til­i­ty analy­sis au­gurs de­feat in piv­otal tri­al test­ing of Nu­Cana's lead drug in metasta­t­ic pan­cre­at­ic can­cer

Nearly two years after making its public debut, UK-based NuCana’s mission to make chemotherapies more potent and safer was dealt a blow, after a pivotal study testing its lead experimental drug halted enrollment in a hard-to-treat advanced form of cancer, following a futility analysis.

The drug, Acelarin, is being evaluated for use in metastatic pancreatic cancer patients who were not considered suitable for combination chemotherapy. In the late-stage ACELARATE study — which compared the experimental drug against the chemotherapy gemcitabine — 200 patients had been enrolled by the sponsor, Clatterbridge Cancer Centre, before an analysis from an independent safety and data monitoring panel suggested the study’s main goal would not be met.