On September 13, 2022 AnBogen Therapeutics, a clinical-stage biotech company designing and developing precision oncology therapies to improve the life of patients, reported that U.S. Food and Drug Administration (FDA) has approved an investigational new drug (IND) protocol amendment for its phase Ib/II trial of ABT-101, an orally administered, irreversible, mutant selective tyrosine kinase inhibitor targeting oncogenic mutations of HER2 Exon20 insertion expressed in non-small cell lung cancer (NSCLC) (Press release, Anbogen Therapeutics, SEP 13, 2022, https://www.prnewswire.com/news-releases/anbogen-therapeutics-receives-approval-from-us-fda-for-phase-ibii-protocol-amendment-for-abt-101-301621795.html [SID1234619518]).

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"We are pleased to receive approval from the FDA on the protocol amendment, and continue to drive ABT-101 clinical development program forward. With the mission in mind, we want to make impact to transform the treatment for patients with non-small cell lung cancer harboring exon 20 insertions (e20i) in HER2," said John Tsu-An Hsu, Ph.D, Founder and Chief Executive Officer of Anbogen Therapeutics. "We look forward to working closely with FDA and all of our investigators as we enroll and dose the patients with non-small cell lung cancer (NSCLC). ABT-101 demonstrates excellent therapeutic potential and superior safety profile compared to competitors that are under clinical development, and it meets challenges of unmet medical needs for cancer drugs targeting exon 20 insertions (e20i) in HER2."

ABT-101

ABT-101 is a potent small molecule kinase inhibitor against several oncogenic kinases including epidermal growth factor receptor (EGFR), and human epidermal growth factor receptor 2 (HER2, also known as ERBB2). Approximately 10% to 12% of EGFR mutations and about 2% to 4 % of HER2 mutations in NSCLC are present in exon 20. Importantly, 90% of HER2 mutations in NSCLC are exon 20 mutations. It is estimated that the global annual patient population is greater than 56,000 who harbor HER2 exon insertion mutation. In the pre-clinical pharmacological studies, Anbogen has demonstrated that ABT-101 has superior selectivity against HER2 e20i mutation and significantly inhibits the growth of tumor harboring HER2-e20i.

On September 13, 2022 Regen BioPharma, Inc. (OTC PINK: RGBP) and (OTC PINK: RGBPP) reported initiation of a series of phased experiments to begin the process of moving its CAR-T cell de-differentiation approach through pre-clinical validation (Press release, Regen BioPharma, SEP 13, 2022, View Source;begins-experiments-validating-its-proprietary-car-t-cell-therapy-301623585.html [SID1234619517]). CAR-T cells are T cells (the lymphoid cells of the body that kill tumors) isolated from a cancer patient that have been modified by expressing a chimeric antigen receptor (CAR) which is specific for the patient’s tumor.

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While CAR-T cells are effective at treating certain lymphomas and leukemias, solid tumors such as liver, breast and colon remain resistant to CAR-T therapies for several reasons. One reason is "T cell exhaustion", a term that means the T cells that are initially recruited to the tumor to kill it end up losing their effectiveness. The company believes that NR2F6, a checkpoint that puts the brakes on T cell activity, is a key player in the T Cell exhaustion phenomenon. Inhibiting NR2F6 is expected to prevent these T cells from becoming dysfunctional.

The Company has engaged the contract research organization, ProMab Biotechnologies, Inc. of Richmond, California, to embark on a series of experiments using the Company’s proprietary shRNA NR2F6-inhibiting technology to validate this approach.

"We are extremely excited to be using our cutting-edge genetic approach to create long-lasting CAR-T cells," says David Koos, Chairman and CEO of the Company. "By partnering with a well-qualified organization such as ProMab Biotechnologies, Inc., we expect to quickly move this program forward to the clinic."

On September 13, 2022 The University of Texas MD Anderson Cancer Center (‘MD Anderson’) and Radiopharm Theranostics Limited (‘Radiopharm’)(ASX: RAD) reported the launch of Radiopharm Ventures, LLC, a joint venture company created to develop novel radiopharmaceutical therapeutic products for cancer (Press release, MD Anderson, SEP 13, 2022, View Source [SID1234619516]).

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Radiopharm Ventures brings together MD Anderson’s innovative and proprietary technologies in antigen discovery and molecular imaging with Radiopharm’s expertise in developing radiopharmaceutical products. The joint venture will focus initially on developing at least four therapeutic products based on MD Anderson intellectual property.

"Radiopharmaceuticals continue to be rapidly developed as a highly promising therapeutic frontier in oncology," said Riccardo Canevari, chief executive officer at Radiopharm. "We are pleased to have this opportunity to collaborate with MD Anderson and its tremendous scientists as we work to make significant in-roads into cancer therapy for the benefit of patients."

Radiopharmaceuticals are designed to deliver small doses of radiation to specifically targeted cells for either therapeutic or diagnostic purposes. Effective cancer radiopharmaceuticals require tumor-specific targets not found in healthy tissue. MD Anderson researchers have established novel platforms to discover and validate tumor specific antigens, offering promising candidates for the development of new radiopharmaceuticals.

The first potential therapeutic candidate is a humanized immunoglobulin G (IgG) antibody against the tumor-specific antigen B7-H3, also known as CD276, which is highly expressed in several common tumors but not in healthy cells. The antibody was developed in the laboratory of David Piwnica-Worms, M.D., Ph.D., chair of Cancer Systems Imaging at MD Anderson. Pre-clinical studies suggest the candidate radiotherapeutic antibody is effective in eliminating resistant colorectal cancers in laboratory models.

"Based on our early pre-clinical data, B7-H3 represents a promising radiotherapeutic target, and we look forward to having the opportunity to work with the team at Radiopharm with the goal of advancing our therapeutic candidate toward future clinical studies," Piwnica-Worms said.

In addition, the work of Samir Hanash, M.D., Ph.D., professor of Clinical Cancer Prevention at MD Anderson, has resulted in extensive characterization of the cancer surfaceome ­- the catalogue of proteins found specifically on the surface of cancer cells across cancer types – resulting in novel targets with cancer-restricted expression. Radiopharm Ventures has an opportunity to select additional targets from this dataset and plans to prioritize selection based on unmet needs in oncology.

"The cancer surfaceome holds a wealth of information about antigens restricted to cancer," Hanash said. "Mining the data, generated at the petabyte level, has uncovered many compelling targets that have not previously been explored, and we are pleased for the opportunity to collaborate with Radiopharm in developing innovative new treatment options for some targets."

Once targets have been selected, Radiopharm will collaborate with Hanash and Piwnica-Worms at MD Anderson to advance preclinical development of potential therapeutic candidates.

On September 13, 2022 Syros Pharmaceuticals, Inc. (NASDAQ:SYRS), a leader in the development of medicines that control the expression of genes, reported that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to SY-5609 for the treatment of pancreatic cancer (Press release, Syros Pharmaceuticals, SEP 13, 2022, View Source [SID1234619515]). SY-5609, a highly selective and potent oral cyclin-dependent kinase 7 (CDK7) inhibitor, is currently being evaluated in combination with chemotherapy for the treatment of patients with relapsed metastatic pancreatic cancer.

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"This orphan drug designation underscores the urgency of our efforts to develop SY-5609 for patients with pancreatic cancer, one of the most devastating and difficult to treat malignancies," said David A. Roth, M.D., Chief Medical Officer of Syros. "Based on the early data we reported last year, which demonstrated single-agent activity in heavily pre-treated patients, as well as compelling preclinical data and a strong mechanistic rationale, we believe SY-5609 could deliver meaningful benefit to people with pancreatic cancer, whose tumors have otherwise eluded therapeutic intervention. We look forward to sharing initial data from the safety lead-in portion of our ongoing Phase 1 study later this year."

The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for the treatment of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation may provide certain benefits, including a seven-year period of market exclusivity if the drug is approved, tax credits for qualified clinical trials and an exemption from FDA application fees.

Syros’ ongoing Phase 1 trial is evaluating SY-5609 in combination with chemotherapy in pancreatic cancer patients who have progressed following treatment with FOLFIRINOX. Patients were randomized to receive either SY-5609 in combination with gemcitabine, or SY-5609 in combination with gemcitabine and nab-paclitaxel, at the approved doses of the combination agents. The study is assessing safety and tolerability, as well as efficacy measures such as disease control rate and progression free survival. Safety and clinical activity data from the safety lead-in portion of the trial are expected in the second half of 2022.

Under an existing clinical supply agreement with Roche, Syros is also supplying SY-5609 for a combination dosing cohort in Roche’s ongoing Phase 1/1b INTRINSIC trial. This cohort is evaluating the combination of SY-5609 and atezolizumab in patients with BRAF-mutant colorectal cancer. Roche is the sponsor of the INTRINSIC trial.

On September 13, 2022 SonALAsense reported Diffuse intrinsic pontine glioma (DIPG) is a rare and deadly pediatric brain cancer with a 2% five-year survival rate (Press release, SonALAsense, SEP 13, 2022, View Source [SID1234619514]). To provide therapeutic options for patients and families, SonALAsense is partnering with clinicians at Children’s National Hospital, Ivy Brain Tumor Center at Barrow Neurological Institute, Phoenix Children’s Hospital and University of California San Francisco (UCSF) Medical Center to conduct a phase 1/2 clinical trial for sonodynamic therapy (SDT) . This is the first SDT trial for children.

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Roger Packer, M.D., co-leads the Children’s National clinical trial site with Lindsay Kilburn, M.D., and Hasan Syed, M.D., who treated the study’s first patient, a 5-year-old girl, two weeks ago. The trial will assess safety, overall patient health and tumor size.

"DIPG is a relentless and sadly fatal disease afflicting children, and there’s currently very little we can do to stop it," said Dr. Packer, who directs the Brain Tumor Institute at Children’s National. "Most children survive less than a year. Sonodynamic therapy offers us an innovative and promising, noninvasive way to directly target the tumors in these children."

"With sonodynamic therapy, we are using focused ultrasound to activate SonALAsense’s drug candidate, which is administered to the patient, and selectively killing tumor cells while leaving normal cells intact," said treating neurosurgeon, Dr. Syed. "With this, we are hoping to see improved outcomes for both tumor regression and patient health during treatment."

As many as 300 children are diagnosed with DIPG in the U.S. each year and there are no effective treatments. DIPG infiltrates the pons, which is located in the brainstem. The condition is difficult to diagnose and treat because tumor cells often diffuse throughout the pons. Radiation therapy is the current standard of care, but it can only delay the cancer, not stop it.

"SDT is a targeted and nonsurgical investigational approach for kids who have almost no therapeutic options," said SonALAsense founder, Chief Medical Officer and Chief Science Officer Stuart Marcus, M.D., Ph.D. "Surgery is contraindicated, chemotherapy and directed therapies are ineffective and have significant side effects."

SDT combines SonALAsense’s drug candidate, SONALA-001, a proprietary intravenous formulation of aminolevulinic acid (ALA), with Insightec’s magnetic resonance-guided focused ultrasound to destroy cancer cells. As the SDT pioneer, SonALAsense is the first and only company to advance SDT into clinical trials in DIPG.

"SDT is a completely novel approach to an incredibly challenging pediatric cancer," said Dr. Sabine Mueller, Pacific Pediatric Neuro-oncology Consortium (PNOC) Lead, and co-Leader of the Pediatric Malignancies Program, UCSF Helen Diller Family Comprehensive Cancer Center.

"DIPG is a devastating disease and, right now, we have no viable treatments for it," said patient advocate Lisa Ward, whose son Jace died from DIPG complications in 2021 at age 22. "We need to explore encouraging new treatments, like SDT, to help patients and families who are suffering with this condition."

The trial is supported, in part, by a $2 million National Cancer Institute Small Business Innovation Research grant to SonALAsense with a subcontract to Children’s National. The trial site in Phoenix is led by Nader Sanai, M.D., at the Ivy Brain Tumor Center, who also led the first-in-human Phase 0/1 clinical trial of SDT in recurrent high-grade glioma patients, along with Lindsey Hoffman, D.O., at Phoenix Children’s. The UCSF trial site is being led by Sabine Mueller, M.D., Ph.D.