Tem­pest Ther­a­peu­tics jumps on­to Nas­daq in merg­er with strug­gling Mil­len­do Ther­a­peu­tics; Bio­haven hits $100M mile­stone as it gears up for a late-stage study in mi­graine pa­tients

Less than a month af­ter land­ing a Big Phar­ma part­ner on one of its lead on­col­o­gy pro­grams, Tem­pest Ther­a­peu­tics is back­flip­ping on­to Nas­daq through a re­verse merg­er with Mil­len­do Ther­a­peu­tics.

Back in Jan­u­ary, Mil­len­do shut down the sole re­main­ing ear­ly-stage study for its NK3R an­tag­o­nist MLE-301 — just nine months af­ter pulling the plug on a piv­otal study for its lead drug liv­o­le­tide. The biotech planned some deep cuts for the re­main­ing staff, while look­ing for a sale or merg­er.

In sup­port of the merg­er, Tem­pest se­cured com­mit­ments from a slate of in­vestors for a $30 mil­lion PIPE fi­nanc­ing, in­clud­ing Ver­sant Ven­tures, Rock Springs Cap­i­tal, F-Prime Cap­i­tal, Monashee In­vest­ment Man­age­ment, Quan Cap­i­tal, Lyfe Cap­i­tal, Maven In­vest­ment Part­ners US, Lil­ly Asia Ven­tures and Eight Roads Ven­tures, the com­pa­ny said on Mon­day. Over­all, the deal should ex­tend the com­pa­ny’s cash run­way through ear­ly 2023, ac­cord­ing to a state­ment.

Tom Duben­sky

Ear­li­er this month, the com­pa­ny inked a deal with Roche to study its TPST-1120 in com­bi­na­tion with Tecen­triq and Avastin in pre­vi­ous­ly un­treat­ed pa­tients with ad­vanced he­pa­to­cel­lu­lar car­ci­no­ma (HCC). The com­pa­ny al­so has TPST-1495, which is in a Phase Ia/Ib dose and sched­ule op­ti­miza­tion study in pa­tients with ad­vanced sol­id tu­mors, specif­i­cal­ly known prostaglandin-dri­ven tu­mors like col­orec­tal can­cer, lung ade­no­car­ci­no­ma and urothe­lial can­cer.

“The tran­si­tion of Tem­pest to a pub­lic com­pa­ny en­hances our abil­i­ty to fund these po­ten­tial­ly im­por­tant prod­uct can­di­dates, as well as con­sid­er ad­di­tion­al pro­grams with ex­cit­ing new tar­gets,” CEO Tom Duben­sky said in a state­ment.

The merg­er is ex­pect­ed to close in the first half of this year, with Mil­len­do stock­hold­ers ex­pect­ed to own ap­prox­i­mate­ly 18.5% of the com­bined com­pa­ny and pre-merg­er Tem­pest stock­hold­ers to hold 81.5%.

Bio­haven hits $100M mile­stone as it gears up for a late-stage study in mi­graine pa­tients

Bio­haven has of­fi­cial­ly launched a Phase II/III tri­al for its oral mi­graine drug, trig­ger­ing a $100 mil­lion pay­off from Roy­al­ty Phar­ma.

Back in Au­gust, Bio­haven added near­ly $1 bil­lion to its bal­ance sheet through a pair of cre­ative agree­ments with Roy­al­ty Phar­ma and the in­vest­ment firm Sixth Street to bol­ster the com­mer­cial launch of its new mi­graine drug, Nurtec. As part of an agree­ment with Roy­al­ty, Bio­haven was promised up to $250 mil­lion to ad­vance its za­veg­epant in mi­graine and oth­er CGRP-me­di­at­ed dis­eases. Bio­haven had al­ready pock­et­ed $150 mil­lion, and will now re­ceive the last $100 mil­lion as it en­rolls the first pa­tient in the Phase II/III study. In to­tal, Bio­haven is look­ing for 2,900 mi­graine pa­tients to test the ef­fi­ca­cy and safe­ty of 100 mg and 200 mg dos­es of oral za­veg­epant.

“Our suc­cess­ful, mul­ti-year part­ner­ship with Bio­haven to sup­port both the reg­u­la­to­ry ap­proval and com­mer­cial­iza­tion of Nurtec as well as pipeline de­vel­op­ment is an ex­cel­lent ex­am­ple of how Roy­al­ty Phar­ma can be a col­lab­o­ra­tive part­ner to in­no­v­a­tive bio­phar­ma com­pa­nies,” Roy­al­ty CSO and co-head of re­search and in­vest­ments Jim Red­doch said in a state­ment.

An in­tranasal for­mu­la­tion of za­veg­epant, for­mer­ly known as vazegepant, achieved su­pe­ri­or ef­fi­ca­cy to place­bo in a Phase II/III study, the com­pa­ny said.

Sim­cere promis­es near­ly $300M for the rights to a for­mer Genen­tech can­di­date

There’s a new glioblas­toma can­di­date in Greater Chi­na.

Sim­cere Phar­ma­ceu­ti­cal Group is putting down $11 mil­lion up­front and up to $281 in mile­stones for the de­vel­op­ment and com­mer­cial­iza­tion rights to Kazia Ther­a­peu­tics’ pax­al­is­ib — a PI3K in­hibitor li­censed from Genen­tech back in 2016. The can­di­date en­tered a piv­otal study ear­li­er this year, and is in sev­en ad­di­tion­al stud­ies for oth­er brain can­cers.

“The need for new ther­a­pies in brain can­cer is sig­nif­i­cant in Chi­na, and we share Kazia’s com­mit­ment to bring­ing for­ward new treat­ment op­tions for pa­tients,” Sim­cere se­nior VP Ren­hong Tang said in a state­ment.

Pax­al­is­ib is dis­tin­guished by its abil­i­ty to cross the blood-brain bar­ri­er, which pre­vents many drugs from ful­ly af­fect­ing brain tis­sue, the com­pa­ny said.

Sim­cere’s up­front pay­ment in­cludes $7 mil­lion in cash and a $4 mil­lion eq­ui­ty in­vest­ment. In ad­di­tion to the $281 mil­lion in mile­stones for glioblas­toma, Kazia can snag more for oth­er in­di­ca­tions, ac­cord­ing to the deal. And Sim­cere will dole out com­mer­cial roy­al­ties in the “mid-teens.” Sim­cere can de­vel­op and mar­ket the drug in Main­land Chi­na, Hong Kong, Macau, and Tai­wan, while Kazia re­tains the rights every­where else.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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BREAK­ING: Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck 5 years ago, where the soft spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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No­vavax shares shred­ded as Covid vac­cine sales fall more than 90% in Q2

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room:

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Bernhardt Zeiher, outgoing Astellas CMO (Astellas)

Q&A: Astel­las' re­tir­ing head of de­vel­op­ment re­flects on gene ther­a­py deaths

For anyone who’s been following discussions about the safety alarms surrounding the adeno-associated viruses (AAV) commonly used to deliver gene therapy, Astellas should be a familiar name.

The Japanese pharma — which bought out Audentes Therapeutics near the end of 2019 and later built a gene therapy unit around the acquisition — rocked the field when it reported three patient deaths in a trial testing AT132, the lead program from Audentes designed to treat a rare muscle disease called X-linked myotubular myopathy (XLMTM).

When the company restarted the trial, it adjusted the dose and instituted a battery of other measures to try to prevent the same thing from happening again. But tragically, the first patient to receive the new regimen died just weeks after administration. The therapy remains under clinical hold, and just weeks ago, Astellas flagged another safety-related hold for a separate gene therapy candidate. In the process of investigating the deaths, the company has also taken flak about the way it disclosed information.

Big questions remain — questions that can have big implications about the future of AAV gene therapies.

Bernhardt Zeiher did not imagine any of it when he first joined Astellas as the therapeutic area leader in inflammation, immunology and infectious diseases. But his ascent to chief medical officer and head of development coincided almost exactly with Astellas’ big move into gene therapy, putting him often in the driver’s seat to grapple with the setbacks.

As Zeiher prepares to retire next month after a 12-year tenure — leaving the unfinished tasks to his successor, a seasoned cancer drug developer — he chatted with Endpoints News, in part, to discuss the effort to understand what happened, lessons learned and the criticism along the way.

The transcript has been lightly edited for length and clarity.

Endpoints: I want to also ask you a bit about the gene therapy efforts you’ve been working on. Astellas has really been at the forefront of discovering the safety concerns associated with AAV gene therapy. What’s that been like for you?

Zeiher: Well, I have to admit, it’s been a bit of a roller coaster. We acquired Audentes. Huge amount of enthusiasm. What we saw with AT132 — that was the lead program in XLMTM — was just remarkable efficacy. I mean, kids who went from being on ventilators, not able to eat for themselves, sit up, do things like that, to off ventilators, walking, you know, really — one investigator called it this Lazarus-like effect. It was just really dramatic efficacy. And then to have the safety events that occurred. So they actually occurred within that first year of the acquisition. So we had the three patient deaths. Me and my organization became very, very much involved. In fact, Ed Conner, who had been the chief medical officer, he left after some of the deaths, but I stepped in as the kind of acting chief medical officer, we had another chief medical officer who was involved, and then we had a fourth death, and I became acting again for a period of time.

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Clo­vis says it needs mon­ey af­ter re­verse split vot­ed down; Cin­Cor touts PhII da­ta for hy­per­ten­sion drug

Clovis Oncology needs a serious leg up after posting a $71.3 million net loss in Q2.

According to the company’s Q2 report, based on its current cash, cash equivalents and liquidity the company will need to raise additional capital in the near term to continue going on beyond February of next year.

A proposed reverse stock split of Clovis’ common stock, which would have freed up more capital, was not approved at its annual meeting of stockholders. The company said in the report it is looking at other sources of funding such as strategic partnerships or licensing arrangements for one or more of its products or candidates.