Hopes fad­ing, Onco­Genex ac­knowl­edges a PhI­II flop for can­cer drug

Any dwin­dling hopes that in­vestors still had in Onco­Genex $OGXI large­ly evap­o­rat­ed Tues­day morn­ing when the Seat­tle-based biotech re­port­ed that its late-stage ef­fort for cu­stirsen failed a Phase III study for cas­tra­tion re­sis­tant prostate can­cer.

The Phase III fail­ure is spurring Onco­Genex to see if it can get a green light from the FDA to get an ear­ly look at a sep­a­rate study for non-small cell lung can­cer as it as­sess­es its re­main­ing prospects.

Onco­Genex had al­ready fall­en in­to pen­ny stock ter­ri­to­ry af­ter a se­ries of set­backs, its mar­ket cap shrink­ing to a mere $26 mil­lion ahead of to­day’s re­port. That’s less than the $39.7 mil­lion in cash it re­port­ed at the end of Q2. And now Onco­Genex has con­tract­ed MTS Health Part­ners to start ex­plor­ing its “strate­gic al­ter­na­tives,” which can be loose­ly trans­lat­ed as a grim re­view of what comes next for the fal­ter­ing biotech.

The com­pa­ny’s stock plunged 34% af­ter the news hit, drop­ping to 59 cents a share.

It’s all been down hill for Onco­Genex since Te­va pulled out of its col­lab­o­ra­tion a lit­tle more than a year ago. A Phase II study for ap­a­tors­en failed last fall. Its stock cratered at the end of last year af­ter the biotech took an ear­ly look at one sub­group pop­u­la­tion in a Phase III study of cu­stirsen and de­ter­mined there had been no im­prove­ment in the over­all sur­vival rate. The com­pa­ny fol­lowed up by slash­ing close to a third of its staff.

Onco­Genex has had a long, hard fall since the op­ti­mistic days of 2009, when it turned up at AS­CO with promis­ing re­sults from a small study of cu­stirsen and saw its shares trade around the $30 mark.

Onco­Genex CEO Scott Cor­ma­ck

Now, with its stock bat­tered and its back against the wall, Onco­Genex wants an­oth­er peek at its re­main­ing Phase III study. In­vestors will not have much con­fi­dence that the com­pa­ny’s for­tunes will turn around at this point.

“Giv­en that the EN­SPIR­IT tri­al has near­ly com­plet­ed en­roll­ment and we be­lieve there are like­ly a suf­fi­cient num­ber of events to de­ter­mine the ef­fect of cu­stirsen in NSCLC, we are ea­ger to ex­pe­dite the fi­nal da­ta analy­sis, which would al­low us to con­serve re­sources and ful­ly un­der­stand the val­ue of the as­set as we eval­u­ate our al­ter­na­tives to max­i­mize share­hold­er val­ue,” said Onco­Genex CEO Scott Cor­ma­ck.

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.

Mathai Mammen (Rob Tannenbaum, Endpoints News at BIO 2018)

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 five 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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Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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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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Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

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HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

US weighs new route of ad­min­is­tra­tion for mon­key­pox vac­cine as cas­es climb — re­port

Less than a week after HHS Secretary Xavier Becerra declared monkeypox a national health emergency, reports have emerged that the US plans to extend its vaccine supply by opting for a different route of administration.

Officials are expected to call for intradermal injection of Bavarian Nordic’s Jynneos vaccine — the only shot approved specifically for monkeypox in the US — as opposed to subcutaneous injection, unnamed sources told both the New York Times and Washington Post on Tuesday.

'Messy at best': Is the US re­peat­ing the same Covid mis­steps with mon­key­pox mes­sag­ing?

When Kyle Planck first suspected he might have monkeypox in late June, he went to the CDC website and found six photos of different types of lesions. And that was about it for general public information.

Planck, who is a sixth-year PhD pharmacology researcher at Weill Cornell, kept looking though and found a separate part of the CDC website meant for healthcare professionals. There he found a medical slide deck with more pictures, professional journal articles and more details about symptoms and diagnosis.

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David Zaccardelli, Verona Pharma CEO

Verona’s COPD drug shines in PhI­II study, po­ten­tial­ly clear­ing path to FDA — shares jump

UK-based Verona Pharma’s COPD drug, ensifentrine, has succeeded in its Phase III trial, paving the way for a possible FDA approval.

In Verona’s Phase III ENHANCE-2 study, roughly 800 patients with moderate to severe COPD received ensifentrine or placebo through a nebulizer twice a day for 24 weeks. At 12 hours post-treatment on week 12, the placebo-corrected change in forced expiratory volume (FEV1) — a standard measure of lung function that tests how much breath one can forcefully exhale in one second — was 94 mL, leading the trial to meet its primary endpoint.

John Oyler, BeiGene CEO (Paul Yeung/Bloomberg via Getty Images)

BeiGene touts pos­i­tive PhI­II da­ta on tislelizum­ab in af­ter­math of set­backs at FDA

After the FDA indefinitely delayed its decision on BeiGene’s Novartis-partnered PD-1 drug candidate tislelizumab as a monotherapy for esophageal cancer, Novartis announced that it was scrapping a second application for non-small cell lung cancer. However, BeiGene is holding out hope that there’s yet another way to expand the drug’s market outside of China.

BeiGene put out word Tuesday morning that a Phase III open-label global study, named RATIONALE 301, met its primary endpoint of non-inferior overall survival (OS) versus sorafenib as a first-line treatment in adults with unresectable hepatocellular carcinoma (HCC). The company claimed that the trial, which enrolled more than 600 patients from the US, Europe and Asia, showed a consistent safety profile from previous studies involving tislelizumab and reported no new safety signals.

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