On October 10, 2023 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, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SLS009, a novel and highly selective CDK9 inhibitor, for the treatment of acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, OCT 10, 2023, View Source [SID1234635819]).

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"We are honored to receive the ODD from the FDA. This designation underscores the potential of SLS009 to address a significant unmet medical need for patients with AML," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "SLS009 is a novel and highly selective CDK9 inhibitor that has already shown a favorable safety profile, strong initial efficacy signals, and evidence of anti-tumor activity. With the support of this ODD, we look forward to accelerating SLS009 clinical development and bringing new hope to those suffering from this devastating disease."

SLS009 is a highly selective CDK9 inhibitor, currently being evaluated in an open-label, single-arm, multi-center Phase 2a study in patients with relapsed or refractory AML. The primary objectives of the trial are to evaluate safety, tolerability, and efficacy at two dose levels of SLS009 (once weekly at 45 mg and at the recommended Phase 2 dose, 60 mg) in combination with azacitidine and venetoclax (aza/ven). Top-line data are expected by the end of this year.

The ODD designation was supported by data from the Phase 1 study of SLS009 which met all key study objectives: anti-tumor activity (cell killing) of up to 77.3% bone marrow blast reduction, durable complete remission (CR) with no minimal residual disease (MRD), desired 24 hours > IC90 peripheral blood concentrations after the first infusion, with IC90 concentrations resulting in up to 97% cancer cell killed, achievement of desired levels of MCL1 and MYC suppression in peripheral blood with decrease in MCL1 or MYC observed in 97% (66/68) of analyzed patients; and, with regard to safety, no dose limiting toxicities, no higher grade non-hematologic toxicities of any kind and some hematologic toxicities difficult to determine in patients with hematologic cancers but short in duration and reversible.

The FDA’s Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for qualified clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

On October 10, 2023 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported a portfolio update, including both increased focus on its ex vivo cell therapy product candidates and an IND submission for SC291 in autoimmune diseases (Press release, Sana Biotechnology, OCT 10, 2023, View Source [SID1234635818]). Sana is positioned to generate clinical proof of concept from multiple programs in 2023 and 2024, with a goal of better understanding its allogeneic HIP CAR T programs in blood cancers, allogeneic HIP CAR T program in autoimmune diseases, and HIP pancreatic islet cells in type 1 diabetes. The company will reduce near-term spend on its fusogen platform for in vivo gene delivery, which postpones the planned SG299 IND and decreases its expected forward operating burn.

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"We have increased confidence in the potential of our HIP platform, and near-term, we are increasing focus on three therapeutic areas that utilize this platform and have the potential to address large unmet needs with curative intent – allogeneic CAR T cells in oncology, allogeneic CAR T cells in autoimmune diseases, and pancreatic islet cell transplantation in type 1 diabetes. We plan to present clinical data in these areas at various times across 2023 and 2024," said Steve Harr, President and CEO of Sana. "The SC291 IND submission for the treatment of autoimmune diseases positions us to move into the rapidly emerging opportunity of utilizing CAR T cells in these large and underserved populations, leveraging the investments we have made to date in the HIP platform, T cell therapeutics, and scaled manufacturing that can produce hundreds of patient doses per run. We need to ensure that we have a financeable cost structure with these emerging opportunities factored in, and this strategic re-positioning enables us to deliver significant clinical data across multiple drug candidates with the current balance sheet. These changes unfortunately mean that many talented and valued colleagues will depart the company, and we thank them for their contributions and commitment to our mission."

Select Program Review

SC291 Oncology (HIP-modified CD19-directed allogeneic CAR T): Enrollment continues in Sana’s ARDENT Phase 1 study for the treatment of B-cell lymphomas and leukemias with clinical data expected in 2023 and 2024.

SC291 Autoimmune (HIP-modified CD19-directed allogeneic CAR T): Sana submitted an IND for the treatment of multiple autoimmune diseases, with preliminary clinical data expected across multiple indications in 2024.

SC262 (HIP-modified CD22-directed allogeneic CAR T): Sana expects to submit an IND in 4Q 2023 for the treatment of B-cell lymphomas and leukemias in patients who have failed CD19-directed CAR T therapies, with preliminary clinical data expected in 2024.

HIP-modified primary islet cells for the treatment of type 1 diabetes: A CTA has been submitted for an investigator sponsored trial exploring the potential of HIP modifications to allogeneic primary islet cells to enable immune evasion and overcome transplant rejection in type 1 diabetes; proof of concept data expected in 2023 and 2024.

SG299 (in vivo CAR T with CD8-targeted fusogen delivery of a CD19-directed CAR): Sana will continue its focused research on this innovative platform but not submit an IND at this time as previously planned.

2024 Operating Burn Guidance

Sana expects 2024 operating cash burn to be below $200 million, allowing the current cash position to extend further into 2025. The strategic re-positioning will reduce headcount by 29% while allowing the company to invest in clinical capabilities across multiple indications in oncology, autoimmune diseases, type 1 diabetes, and central nervous system disorders. Sana will leverage its existing allogeneic manufacturing expertise and continue development of its GMP manufacturing facility in Bothell, Washington.

On October 10, 2023 Perspective Therapeutics, Inc. ("Perspective" or "the Company") (NYSE AMERICAN: CATX), reported that two upcoming investigator-initiated trials (IIT) were presented at the North American Neuroendocrine Tumor Society (NANETS) 2023 Symposium in Montreal, Canada, which was held October 4-6, 2023 (Press release, Perspective Therapeutics, OCT 10, 2023, https://perspectivetherapeutics.com/press-releases/perspective-therapeutics-vmt-%CE%B1-net-protocols-presented-at-the-north-american-neuroendocrine-tumor-society-nanets-2023-symposium/ [SID1234635816]).

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Presentation One: Phase 1 Trial of Pb-212-VMT-alpha-NET in Select Metastatic or Inoperable Somatostatin Receptor Positive Tumors

Summary: This presentation detailed the planned protocol for the IIT Phase 1 trial of Pb-212-VMT-alpha-NET in patients with metastatic or inoperable somatostatin receptors positive (SSTR+) tumors to investigate if this new treatment can improve management of patients naïve to prior radioligand therapy. The study is an open-label, single arm, single-center, phase 1 trial evaluating the safety, tolerability, and pharmacokinetic properties of the alpha-emitting, systemic radioligand therapy agent Pb-212-VMT-alpha-NET in five different SSTR+ tumors: gastrointestinal neuroendocrine tumors (GI-NET), pheochromocytoma and paraganglioma (PPGL), small cell lung cancer (SCLC), renal cell carcinoma (RCC), and head and neck (H&N). Enrollment for this study is expected to commence in the first quarter of 2024.

Presenter: E. Mena, MD, National Cancer Institute, National Institutes of Health

Presentation Two: Phase 1/2 Trial of Pb-212-VMT-alpha-NET in GI Neuroendocrine Tumors and Pheochromocytoma/Paraganglioma Previously Treated with Radioligand Therapy

Summary: This presentation detailed the planned protocol for the IIT Phase 1/2 trial designed to determine if such a new treatment can improve management of patients who have progressed on beta-emitting radioligand therapy. This study is an open-label, single arm, single-center, Phase 1/2 study evaluating the safety, tolerability, and pharmacokinetic properties of the alpha-emitting, systemic radioligand therapy agent Pb-212-VMT-alpha-NET in somatostatin receptors (SSTR+) metastatic GI neuroendocrine tumors (GI-NETs) and pheochromocytoma/paraganglioma (PPGL) tumors. Enrollment for this study is expected to commence in the first quarter of 2024.

Presenter: J. Del Rivero, MD, National Cancer Institute, National Institutes of Health

"There are currently no approved targeted radiopharmaceuticals for NETs patients outside of the gastroenteropancreatic indication," said Chief Executive Officer Thijs Spoor of Perspective Therapeutics. "We are excited to expand clinical studies to a wider range of cancer types in order to potentially expand therapeutic options for patients with these difficult to treat tumors."

"We are delighted to have the NIH conduct these studies to investigate the safety and efficacy of [212Pb]VMT-alpha-NET in these expanded patient populations." said Chief Medical Officer Markus Puhlmann, MD MBA, of Perspective Therapeutics. "These clinical studies are evaluating [212Pb]VMT-alpha-NET in a wide range of neuroendocrine tumors, where the unmet need is greatest."

The presentation abstracts can be accessed on the conference website at View Source The presentations will also be made available on the Company’s website at www.perspectivetherapeutics.com.

About neuroendocrine tumors

Neuroendocrine tumors form in cells that interact with the nervous system or in glands that produce hormones. They can originate in various parts of the body, most often in the gut or the lungs and can be benign or malignant. Neuroendocrine tumors are typically classified as pancreatic neuroendocrine tumors or non-pancreatic neuroendocrine tumors. According to cancer.net, it is estimated that more than 12,000 people in the United States are diagnosed with a NET each year. Importantly, neuroendocrine tumors are associated with a relatively long duration of survival compared to other tumors and as a result, there are approximately 175,000 people living with this diagnosis.

About VMT-α-NET

VMT-α-NET is a clinical stage targeted alpha particle therapy (TAT) radiopharmaceutical being developed for the treatment and diagnosis of somatostatin receptor subtype 2 (SSTR2) expressing neuroendocrine tumors, which are a rare and difficult-to-treat type of cancer. VMT-α-NET incorporates Perspective Therapeutics’ proprietary lead-specific chelator (PSC) to bind Pb-203 for SPECT imaging, and Pb-212 for alpha particle therapy.

On October 10, 2023 Oxford Vacmedix (OVM), the UK-based biopharma company focused on the development of vaccines to treat cancer reported the publication of a key research paper on its lead cancer vaccine, OVM-200 (Press release, Oxford Vacmedix, OCT 10, 2023, View Source;utm_medium=rss&utm_campaign=publication-highlights-the-importance-of-survivin-the-cancer-treatment [SID1234635815]). The paper, in the online journal Advanced Therapeutics, comprehensively reviews the rationale for targeting survivin, the advances in vaccine design using OVM’s novel recombinant overlapping peptide (ROP) platform and the compelling preclinical results for OVM-200 being used both alone and in combination with an immune oncology agent.

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OVM-200 targets survivin, a protein overexpressed by cancer cells that allow unregulated growth and stimulates an immune response. The vaccine is in a Phase 1 trial which is both the first time OVM-200 has been used in people and also the first time any ROP based vaccine has been tested in the clinic. The Phase I trial of OVM-200 is focused on safety and on establishing an immune response in advanced cancer patients in three cancer indications – non small cell lung cancer (NSCLC), prostate cancer and ovarian cancer. Patients are being treated at five leading hospitals in the UK. To date twelve patients have been treated at four dose levels in Phase 1a, the dose escalation part of the trial – the initial results show very good safety and a strong immune response. A further 24 patients are being treated in Phase 1b.

Professor Shisong Jiang, Founder and Chief Scientific Officer of OVM, said:

"We are very pleased this research paper has been published. It shows not just the rationale for selecting survivin as a target and for the design of our ROP vaccines but also our convincing preclinical results with OVM-200. We look forward to the completion of Phase 1 and to being able to combine OVM-200 with immune oncology agents in Phase 2, to help patients with advanced cancer."

Dr Mark Tuthill, Principal Investigator for the trial at the Oxford University Hospitals NHS Trust added;

"We are very excited by the Phase 1a results from the trial of OVM-200 and look forward to being able to progress with the trial. We are very pleased to be working with the team at OVM and to have the opportunity to see how these vaccine treatments can benefit patients. We strongly believe that cancer vaccine will play a major role in the future, potentially in combination with immune-oncology agents."

On October 10, 2023 Olema Pharmaceuticals, Inc. ("Olema" or "Olema Oncology," Nasdaq: OLMA), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of targeted therapies for women’s cancers, reported an amendment to our existing clinical collaboration and supply agreement with Novartis Institutes for BioMedical Research, Inc. ("Novartis") to increase the size of the ongoing Phase 1/2 clinical study testing palazestrant in combination with ribociclib to approximately 60 patients (Press release, Olema Oncology, OCT 10, 2023, View Source [SID1234635814]).

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"The amendment announced today significantly increases the size of our ongoing Phase 1/2 clinical study testing palazestrant in combination with ribociclib, in collaboration with Novartis," said Sean P. Bohen, M.D., Ph.D., President and Chief Executive Officer of Olema Oncology. "With the Phase 1b dose escalation portion now successfully completed, we are currently in Phase 2 dose expansion at the 120 mg dose of palazestrant in combination with 600 mg of ribociclib. We believe that this expanded study now has the potential to generate a clinical dataset sufficient to support the regulatory pathway for a first-line pivotal trial."

Olema first signed a clinical collaboration and supply agreement with Novartis in July 2020, the agreement was amended and restated in January 2022, and focuses on the evaluation of the safety, tolerability and efficacy of palazestrant in combination with Novartis’ proprietary cyclin-dependent kinase 4 and 6 (CDK4/6) inhibitor ribociclib and/or Novartis’ proprietary phosphatidylinositol 3-kinase (PI3Ka) inhibitor alpelisib in patients with metastatic ER+ breast cancer. The amendment adds approximately 30 patients to be enrolled in the cohort expansion phase of the palazestrant clinical study in combination with ribociclib.