Geron Announces PDUFA Date for Imetelstat NDA in Lower Risk MDS

  Geron Corporation (Nasdaq: GERN), a late-stage clinical biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has assigned a standard review and a Prescription Drug User Fee Act (PDUFA) action date of June 16, 2024 for Geron’s New Drug Application (NDA) for imetelstat for the treatment of transfusion-dependent anemia in patients with lower risk myelodysplastic syndromes (MDS). In addition, the FDA informed the Company that it is currently planning to hold an advisory committee meeting as part of the NDA review.

“We look forward to working with the FDA to complete the NDA review process on a timely basis,” said John A. Scarlett, M.D., Geron’s Chairman and Chief Executive Officer. “With the PDUFA date now set, we continue to expect an imetelstat launch in the United States by the end of the first half of 2024, subject to FDA approval.”

https://www.biospace.com/article/releases/geron-announces-pdufa-date-for-imetelstat-nda-in-lower-risk-mds/

Sellas: Independent Data Monitoring Committee Recommends Phase 3 Leukemia Trial Continue

 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced a positive review of the ongoing Phase 3 REGAL clinical trial of galinpepimut-S (GPS) in acute myeloid leukemia (AML) by the Independent Data Monitoring Committee (IDMC). The IDMC performed a routine, prespecified risk-benefit assessment of unblinded data from the study and has recommended that the trial continue without modifications. While the interim analysis is event driven per the protocol (60 events), based upon the Company’s statistical assumptions, the interim analysis is expected to occur by late 2023 or early 2024. The Company anticipates that enrollment in the study will be completed during Q4 2023. The IDMC is scheduled to meet again within Q4 2023.

https://www.biospace.com/article/releases/sellas-life-sciences-independent-data-monitoring-committee-recommends-galinpepimut-s-regal-trial-to-continue-without-any-modifications/

Fulcrum’s Sickle Cell Trial Given Greenlight to Proceed After Hold

 Fulcrum Therapeutics will continue testing FTX-6058 as a treatment for sickle-cell disease now that the FDA has lifted a clinical hold placed on the candidate in February, the company announced today. 

“Based on the initial data from the Phase 1b trial, which showed increasing levels of HbF with each dose escalation, we believe in the potential of FTX-6058 to not only shift the current standard of care but importantly, offer these patients a differentiated oral option,” Alex Sapir, Fulcrum’s president and chief executive officer, said in the release. 

Fulcrum shares rose 30% in the hours following the announcement.

Original story posted March 10:

Clinical Hold on Fulcrum’s Sickle Cell Hopeful Triggered by Hematologic Malignancies Profile

In its decision to put Fulcrum Therapeutics’ sickle cell disease (SCD) candidate under full clinical hold, the FDA cited non-clinical instances of hematological malignancies associated with the drug, the company revealed in its 2022 financial report.

The FDA noted that “the profile of hematologic malignancies observed in the non-clinical studies of FTX-6058 is similar to that observed with other inhibitors of PRC2,” Robert Gould, Ph.D., interim president and CEO, Fulcrum, said during an investor call Thursday.

The regulator is asking Fulcrum to further define the population where FTX-6058’s benefits would clearly outweigh its potential risks.

FTX-6058 is a potent and orally available small molecule inhibitor of the Embryonic Ectoderm Development (EED), a subunit of the PRC2 protein complex. In preclinical studies, Fulcrum has shown that using FTX-6058 to target EED induces an increase in fetal hemoglobin. In December 2022, the FDA granted the candidate Fast Track designation for SCD.

In February, flagging issues with “previously reported preclinical data,” the FDA placed FTX-6058 under full clinical hold. At the time, no specific reason was given for the hold.

Prior to the clinical hold, FTX-6058 was in a Phase Ib study, in which a 6-mg dose of the candidate elicited a 9.5% increase in absolute fetal hemoglobin levels from baseline.

Gould said the hold was not the result of any clinical finding in that trial.

Also during its financial report, Fulcrum announced that Santiago Arroyo has resigned as the company’s chief medical officer, effective March 7. Iain Fraser will fill the post on an interim basis.

The company will continue working with the FDA to address all of the regulator’s concerns regarding FTX-6058, Gould said during the call. Still, in view of the clinical hold, Fulcrum is suspending its guidance to complete the Phase Ib study and to arrive at a registration-enabling dose by the fourth quarter of 2023.

The Problem with PRC2

The clinical hold on FTX-6058 highlights the difficulty of targeting the PRC2 complex.

Epizyme has seen such a drug through to the market. In January 2020, the biotech won FDA accelerated approval for Tazverik (tazemetostat), an orally available and potent first-in-class inhibitor of the EZH2 sub-unit of the PRC2 complex, for use in patients with metastatic or locally advanced epithelioid sarcoma who are ineligible for resection.

Later that year, in June, Tazverik’s indication was expanded to include two specific follicular lymphomas. Ipsen now owns the rights to Tazverik after it bought Epizyme in June 2022 for $247 million.

While Tazverik made it through regulatory rigor, the drug carries a warning for secondary malignancies. Patients treated with Tazverik are at a higher risk of developing T-cell lymphoblastic lymphoma, myelodysplastic syndrome and acute myeloid leukemia.

https://www.biospace.com/article/clinical-hold-of-fulcrum-s-scd-hopeful-triggered-by-risk-of-hematologic-malignancies-/

Novavax Says Updated COVID-19 Vaccine Candidate Neutralizes Emerging Subvariants

 Novavax’s vaccine candidate against new sublineages of COVID-19 is showing promising results in small animal and non-human primate studies. The company announced Tuesday that its XBB COVID vaccine candidate induced neutralizing antibody responses in the EG.5.1 and XBB.1.16.6 subvariants, dubbed Eris and Fornax, respectively.

Data from the CDC and European Union Agency for Disease Prevention and Control point to the XBB variant as a major concern, with a majority of recent cases being XBB variants in both the U.S. and the European Union.

Currently, Novavax is in the process of submitting applications for its XBB.1.5 COVID vaccine candidate to regulatory authorities. If approved, the shot would be the only protein-based non-mRNA vaccine option available for the fall vaccination season, according to the company.

Filip Dubovsky, Novavax’s president of research and development, said in a statement that the company has “a lot of confidence” in its updated COVID vaccine and is “working diligently with global regulatory bodies to ensure our protein-based vaccine is available this fall.”

The Novavax COVID-19 Vaccine, Adjuvanted has been neither approved nor licensed by the FDA. It was authorized for emergency use by the FDA in July 2022, with the agency at the time saying the available data “support that the known and potential benefits of the vaccine outweigh its known and potential risks in people 18 years of age and older, and that this vaccine may be effective in preventing COVID-19.”

Data available at the time from 17,200 patients given the vaccine showed 90.4% effectiveness in preventing mild, moderate, or severe COVID-19, with the most commonly reported side effects being headaches, fatigue, joint pain, redness or swelling at the injection site, nausea/vomiting, muscle pain, and fever. The agency did note that clinical trial data “provide evidence for increased risks” of myocarditis and pericarditis in patients given the vaccine.

Pfizer and Moderna both recently announced that their respective updated vaccines are showing responsiveness to Eris and Fornax. This came after a June 2023 announcement from the FDA that the fall season’s vaccines should focus on the XBB.1.5 variant which, according to data from February 2023, accounted for 65% of all new cases of COVID in the U.S.

The unanimous recommendation by the FDA’s June advisory panel, with no abstentions, set the stage for Novavax to make a comeback of sorts. The company released data that month showing its monovalent vaccine had a neutralizing effect against both XBB.1.5 and XBB.1.16.6, the then-dominant and emerging subvariants of XBB, respectively.

Earlier this month, Novavax posted a surprise $58 million net income in the second quarter of 2023 compared to a net loss of $510 million in the same period last year, thanks to aggressive cost-cutting in the first quarter and laying off 25% of its staff in May. However, the company faces significant challenges as its turnaround strategy is dependent on the successful launch of its updated COVID vaccine this fall.

Novavax CEO John Jacobs said in the second-quarter earnings call that “there is still much work to be done with significant execution risk ahead of us as we seek regulatory authorizations for our first updated version of Nuvaxovid” and prepare to “compete in the U.S. market as it converts to a commercial model for the first time.”

https://www.biospace.com/article/novavax-says-updated-covid-19-vaccine-candidate-neutralizes-emerging-subvariants/

Lionsgate Brings Back Mask Mandates in Hollywood Office

A Hollywood studio has instituted a mask mandate for its employees in light of the current COVID wave. Lionsgate sent an email to staff asking them to mask up on certain floors of its Santa Monica office after several employees caught the virus. The studio is also asking employees to conduct self-screening before coming to the office.

While this is obviously just one office of one studio, the move comes amid some national buzz about whether mask mandates might be returning as variant EG.5 has become dominant in the United States.

The variety has caused an apparent boost in cases (the L.A. Public Health Twitter feed says cases are rising in the area, and the CDC’s website says hospital admissions are up 14 percent nationally this week). Yet experts have also said this omicron-descendent variant — which has been spreading domestically since April — has thus far shown to be no more of a cause of concern than previous variants. According to reporting by The New York Times, the new variety isn’t expected to cause a major wave akin to the first omicron outbreak.

Last May, the film industry relaxed its COVID protocols to no longer require masks in most workplace settings.A Hollywood studio has instituted a mask mandate for its employees in light of the current COVID wave. Lionsgate sent an email to staff asking them to mask up on certain floors of its Santa Monica office after several employees caught the virus. The studio is also asking employees to conduct self-screening before coming to the office.

While this is obviously just one office of one studio, the move comes amid some national buzz about whether mask mandates might be returning as variant EG.5 has become dominant in the United States.

The variety has caused an apparent boost in cases (the L.A. Public Health Twitter feed says cases are rising in the area, and the CDC’s website says hospital admissions are up 14 percent nationally this week). Yet experts have also said this omicron-descendent variant — which has been spreading domestically since April — has thus far shown to be no more of a cause of concern than previous variants. According to reporting by The New York Times, the new variety isn’t expected to cause a major wave akin to the first omicron outbreak.

Last May, the film industry relaxed its COVID protocols to no longer require masks in most workplace settings.A Hollywood studio has instituted a mask mandate for its employees in light of the current COVID wave. Lionsgate sent an email to staff asking them to mask up on certain floors of its Santa Monica office after several employees caught the virus. The studio is also asking employees to conduct self-screening before coming to the office.

While this is obviously just one office of one studio, the move comes amid some national buzz about whether mask mandates might be returning as variant EG.5 has become dominant in the United States.

The variety has caused an apparent boost in cases (the L.A. Public Health Twitter feed says cases are rising in the area, and the CDC’s website says hospital admissions are up 14 percent nationally this week). Yet experts have also said this omicron-descendent variant — which has been spreading domestically since April — has thus far shown to be no more of a cause of concern than previous variants. According to reporting by The New York Times, the new variety isn’t expected to cause a major wave akin to the first omicron outbreak.

Last May, the film industry relaxed its COVID protocols to no longer require masks in most workplace settings.

https://www.hollywoodreporter.com/movies/movie-news/hollywood-studio-covid-rates-mask-mandates-back-1235571466/

ProMIS in $20.4 Million Private Placement Financing

 ProMIS Neurosciences Inc. (Nasdaq: PMN) (“ProMIS” or the “Company”), a biotechnology company focused on the generation and development of antibody therapeutics targeting toxic misfolded proteins in neurodegenerative diseases such as Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA), today announced that it has entered into a securities purchase agreement with certain new and existing accredited investors to issue and sell an aggregate of approximately $20.4 million of (a) common share units, each consisting of one of the Company’s common shares (the “Common Shares”) and one warrant to purchase one Common Share (the “Warrants”) (the “Common Share Units”) and (b) pre-funded units, each consisting of one pre-funded warrant to purchase one Common Share and one Warrant (the “Pre-Funded Units”). The Common Share Units were sold at a price of $1.88 per unit and the Pre-Funded Warrants were sold at a price of $1.87 per unit through a private investment in public equity (“PIPE”) financing.

The PIPE included participation from Affinity Asset Advisors, LLC, Ally Bridge Group, Sphera Healthcare and other institutional and individual accredited investors.

https://finance.yahoo.com/news/promis-neurosciences-announces-20-4-201200578.html

Japan to release Fukushima water into ocean starting Aug. 24

 Japan said on Tuesday it will start releasing more than 1 million metric tonnes of treated radioactive water from the wrecked Fukushima nuclear power plant on Aug. 24, putting into motion a plan that has drawn strong criticism from China.

The plan, approved two years ago by the Japanese government as crucial to decommissioning the plant operated by Tokyo Electric Power Company (Tepco), has also faced criticism from local fishing groups, who fear reputational damage and a threat to their livelihood.

"I have asked Tepco to swiftly prepare for the water discharge in accordance with the plan approved by the Nuclear Regulation Authority, and expect the water release to start on August 24, weather conditions permitting," Prime Minister Fumio Kishida said on Tuesday morning.

The announcement comes a day after the government said it had won "a degree of understanding" from the fishing industry over the release of the water, even as a fishing group said it still feared the reputational damage would ruin livelihoods.

The first batch of water that will be released starting Thursday will total 7,800 cubic metres over about 17 days, Tepco said at a briefing held Tuesday.

That water will contain about 190 becquerels of tritium per litre, below the World Health Organisation drinking limit of 10,000 becquerels per litre, according to Tepco. A becquerel is a unit of radioactivity.

Japan has said that the water release is safe. The International Atomic Energy Agency (IAEA), the U.N. nuclear watchdog, greenlighted the plan in July, saying that it met international standards and that the impact it would have on people and the environment was "negligible".

About 56% of respondents to a survey conducted by Japanese broadcaster FNN over the weekend said they supported the release, while 37% opposed.

"The IAEA and many other countries have said it's safe, so I believe it is. But fishermen are facing so many problems so the Japanese government needs to do something to convince them," said 77-year-old NGO worker Hiroko Hashimoto.

SCEPTICISM ABROAD

Despite assurances, some neighbouring countries have expressed scepticism over the safety of the plan, with Beijing emerging as the biggest critic. Foreign ministry spokesman Wang Wenbin said in July that Japan had shown selfishness and arrogance, and had not fully consulted the international community about the water release.

China bans seafood imports from 10 prefectures in Japan, including Fukushima and the capital, Tokyo.

South Korean activists have also protested the plan, although Seoul has concluded from its own study that the water release meets international standards and said it respects the IAEA's assessment.

Kishida said on Tuesday that he believed an "accurate understanding" of the matter was spreading in the international community.

"There is an understandable perception that all radioactive materials are always and everywhere dangerous... but not all radioactive materials are dangerous," Tony Irwin, an honorary associate professor at the Australian National University, said in a note.

"Nuclear power plants worldwide have routinely discharged water containing tritium for over 60 years without harm to people or the environment, most at higher levels than the 22 TBq per year planned for Fukushima," he added.

Japan says the water will be filtered to remove most radioactive elements except for tritium, an isotope of hydrogen that is difficult to separate from water. The treated water will be diluted to well below internationally approved levels of tritium before being released into the Pacific.

The water was used to cool the fuel rods of Fukushima Daiichi after it melted down in an accident caused by a huge tsunami in 2011 that battered Japan's eastern coast.

A Japanese official said the first test results of the seawater after the discharge may be available at the start of September. Japan will also test fish in the waters near the plant, and make the test results available on the agriculture ministry's website.

https://news.yahoo.com/japan-release-fukushima-water-ocean-013436601.html