EREG

On December 23, Regor Therapeutics, a clinical-stage biotech company, announced authorization from the US Food and Drug Administration (FDA) to proceed with Regor's Phase 1 clinical development plans for RGT-419B. RGT-419B is a new generation CDK2/4/6, small molecule inhibitor with an optimized kinase activity spectrum. It is expected to improve the safety of and combat the resistance to currently approved CDK4/6 inhibitors that are given in combination with endocrine-based therapy for treatment of pre/perimenopausal or postmenopausal women with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-) advanced or metastatic breast cancer. In cell model experiments, RGT-419B has demonstrated single agent activity with greater suppression of ER+ tumor cell growth when compared to abemaciclib and palbociclib. In ER+ breast cancer cells with acquired resistance to currently approved CDK4/6 inhibitors, RGT-419B has demonstrated full suppression of cancer cell proliferation. In these experiments, RGT-419B's tumor cell suppression was further augmented when combined with a selective estrogen receptor degrader or a PI3K signaling pathway inhibitor. As a single agent and in combination with other approved therapies, RGT-419B will initially be developed for the treatment of patients with HER2-, ER+ breast cancer who have primary or acquired resistance to currently approved CDK4/6 inhibitors. As CDK4/6 inhibitors are widely adopted in clinical practice, refractory and relapsed patients previously treated by CDK4/6 inhibitors are becoming a greater and clinically significant unmet medical need. RGT-419B's clinical development programs will address this resistance to CDK4/6 and other hormone receptor modulation therapy, providing a new opportunity to improve the survival and quality of life in breast cancer patients with advanced disease. RGT-419B is the first of many innovative oncology drugs being developed by Regor. Dr. Xiayang Qiu, Founder and CEO of Regor Therapeutics, said, "This milestone marks Regor's success in entering into the area of oncology therapeutics. Achieving an optimal selectivity pro several CDKs by leveraging Regor's proprietary CARD platform, this innovative drug is entering clinical stage development in the U.S. We are excited by this opportunity to bring new and differentiated treatment options to the many patients suffering from breast cancer worldwide. About breast cancer HER2-, HR+ breast cancer is the most common type of breast cancer. Globally, breast cancer is one of the most common cancers affecting approximately 2.3 million women in 2020 and is the leading cause of death due to cancer in women. In China breast cancer ranks 4th amongst solid tumor types with over 400,000 new cases annually. Breast cancer poses a tremendous healthcare burden in China and across the world as well as having a significant negative impact on patients and their families. About Regor Therapeutics Group Regor Therapeutics is a clinical stage company dedicated to the discovery and development of innovative and clinically differentiated medicines by leveraging the proprietary CARD (Computer Accelerated Rational Discovery) Platform. The company focuses mainly on three therapeutic areas, oncology, immunology, and metabolic disorders. By seamlessly integrating CARD with structural biology, computational chemistry, therapeutic biology, medicinal chemistry, and clinical development. Regor has successfully assembled a world-class scientific team and established a highly efficient new drug innovation engine to enable the discovery and development of best- and first-in-class molecules.

One day after Bay Area-based Olema Pharmaceuticals announced that falsified information regarding its Phase I/II breast cancer data was circulating across the Internet, the company released its topline data for OP-1250, a potential best-in-class complete estrogen receptor antagonist (CERAN). This morning, Olema, which launched last year, said it successfully completed the first portion of the Phase I/II study of OP-1250, a selective ER degrader (SERD) being developed to treat metastatic breast cancer and other women’s cancers. Not only was the dose-escalation successful, but the company also said it saw clear efficacy signals in the early data. Data from this portion of the study will be presented at the San Antonio Breast Cancer Symposium later this month. According to Olema, as of a month ago, the data show strong proof of concept for OP-1250. OP-1250 has shown “highly attractive pharmacokinetics” that supports once-daily dosing. Olema said OP-1250 has also shown favorable “tolerability and clear evidence of anti-tumor activity.” The Phase I portion of the study is seeing some positives. The company said there have been three partial responses, two of which have been confirmed, as well as “robust target lesion reduction up to 100%.” The company noted patients participating in the study have been heavily pretreated. In the recommended Phase II dose range of 60–120 mg of OP-1250 once per day, the overall response rate (ORR) was 17%, and the clinical benefit rate (CBR) was 46%. The company said all three responses occurred in patients with ESR1 mutations who previously received CDK4/6 and aromatase inhibitors and fulvestrant, a selective estrogen receptor degrader. Olema added that four response-eligible patients had target lesion reductions of 30% or greater. As of the Nov. 1 data cut-off date, 32% of patients remained on treatment. Olema reported that efficacy data is continuing to mature, including both patients with confirmed partial responses. Sean P. Bohen, M.D., Ph.D., president and chief executive officer of Olema, said the dose-escalation stage of the Phase I/II study has been successfully met. Additionally, he touted the favorable tolerability and early efficacy signals witnessed in the data. “We are particularly encouraged by the responses observed in patients who had received multiple prior lines of therapy and harbored ESR1 activating mutations, demonstrating that OP-1250 is an active drug and achieved sufficient exposure levels to block the ER-mediated cancer cell growth and proliferation signal,” Bohen said in a statement. “These data give us confidence to rapidly advance our development program in both monotherapy and combination settings, as we work to further position OP-1250 as a differentiated, potential best-in-class CERAN that we believe could become the backbone endocrine therapy of choice for ER+ breast cancer.” Pamela Klein, chief medical officer at Olema, added that the interim data suggest OP-1250 may provide a meaningful benefit to patients with advanced or metastatic breast cancer, some of whom may have limited treatment options remaining. Klein said the Phase II portion of the study is expected to examine OP-1250 as a monotherapy and in combination with a CDK4/6 inhibitor. The Phase II portion will begin in the first quarter of 2022. One day before the company released its data, a poster that contained what Olema called falsified information circulated on the Internet. The data appears to have been shared by a patient who claimed to have participated in the study but did not receive a higher dose of the drug and did not see benefits. The individual who posted it claimed to have been sent a summary of the study and shared the images on Twitter. After the company put out its data, which was not as robust as the information shared on Twitter, the company stock has taken a considerable hit. As of this writing, the stock is down more than 47% to $11.17 per share.

SAN DIEGO, Nov. 24, 2021 (GLOBE NEWSWIRE) -- eFFECTOR Therapeutics, Inc. (NASDAQ:EFTR), a leader in the development of selective translation regulator inhibitors (“STRIs”) for the treatment of cancer, announced today the publication of data highlighting the anti-tumor potential of zotatifin, the company’s clinical-stage inhibitor of eukaryotic translation initiation factor 4A (“eIF4A”), in the peer-reviewed journal Frontiers in Oncology. eIF4A is a catalytic component of the eIF4F complex, which regulates oncoprotein production at the level of mRNA translation. The published research showed that zotatifin downregulated the expression of certain receptor tyrosine kinases (“RTKs”), which are mutated and overexpressed in many cancers. The research also showed that rational combinations of zotatifin with the PI3K inhibitor alpelisib or the AKT inhibitor ipatasertib, both of which inhibit signaling downstream of RTKs, led to enhanced anti-tumor activity in vivo. “The preclinical results highlighted in this publication underscore the unique therapeutic advantages of eIF4A inhibition and further demonstrate zotatifin’s potential as a promising clinical strategy for patients with cancers driven by RTK overexpression or dysregulation,” said Steve Worland, Ph.D., president and chief executive officer of eFFECTOR. “These data and other results are informing the selection of biomarker-specific patient populations for the Phase 2a expansion cohorts we initiated earlier this year.” The Role of eIF4A in Cancer eIF4A is a strong anti-proliferative target located at the convergence of both the RAS and PI3K signaling pathways. eIF4A inhibition selectively regulates the translation of a distinct set of target “onco” mRNAs that encode a number of important oncoproteins and survival factors. Through eIF4A inhibition, there is potential to target multiple oncoproteins, including those for which there are currently no targeted therapies available, such as MYC and Cyclin D1. About Zotatifin (eFT226) Zotatifin is a highly potent and sequence-selective small molecule inhibitor of eIF4A that works by translationally down-regulating expression of select RTKs that contain zotatifin binding motifs in the 5-prime untranslated region (5’-UTR) of their mRNAs. We are currently investigating zotatifin in ongoing clinical trials for solid tumors and as a potential host-directed antiviral therapy in patients with mild to moderative COVID-19 in collaboration with the University of California, San Francisco. About eFFECTOR Therapeutics eFFECTOR is a clinical-stage biopharmaceutical company focused on pioneering the development of a new class of oncology drugs referred to as STRIs. eFFECTOR’s STRI product candidates target the eIF4F complex and its activating kinase, mitogen-activated protein kinase interacting kinase (MNK). The eIF4F complex is a central node where two of the most frequently mutated signaling pathways in cancer, the PI3K-AKT and RAS-MEK pathways, converge to activate the translation of select mRNA into proteins that are frequent culprits in key disease-driving processes. Each of eFFECTOR’s product candidates is designed to act on a single protein that drives the expression of multiple functionally related proteins, including oncoproteins and immunosuppressive proteins in T cells, that together control tumor growth, survival and immune evasion. eFFECTOR’s lead product candidate, tomivosertib, is a MNK inhibitor currently being evaluated in KICKSTART, a randomized, double-blind, placebo-controlled Phase 2b trial of tomivosertib in combination with pembrolizumab in patients with metastatic non-small cell lung cancer (NSCLC). Zotatifin, eFFECTOR’s inhibitor of eIF4A, is currently being evaluated in Phase 2a expansion cohorts in certain biomarker-positive solid tumors, including ER+ breast cancer and KRAS-mutant NSCLC. eFFECTOR has a global collaboration with Pfizer to develop inhibitors of a third target, eIF4E. In addition to the company’s oncology focus, zotatifin is being evaluated as a potential host-directed anti-viral therapy in patients with mild to moderate COVID-19 in collaboration with the University of California, San Francisco, under a $5 million grant sponsored by the Defense Advanced Research Projects Agency. Forward-Looking Statements eFFECTOR cautions you that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. The forward-looking statements are based on our current beliefs and expectations and include, but are not limited to: the future clinical development of our product candidates; and the potential therapeutic benefits of our product candidates. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in our business, including, without limitation: potential delays in the commencement, enrollment and completion of clinical trials; disruption to our operations from the COVID-19 pandemic, including clinical trial and manufacturing delays; our dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; the results of preclinical studies and early clinical trials are not necessarily predictive of future results; the success of our clinical trials and preclinical studies for our product candidates is uncertain; regulatory developments in the United States and foreign countries; unexpected adverse side effects or inadequate efficacy of our product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; our ability to obtain and maintain intellectual property protection for our product candidates; we may use its capital resources sooner than it expects; and other risks described in our prior filings with the Securities and Exchange Commission (SEC), including under the heading “Risk Factors” in our most recent quarterly report on Form 10-Q and any subsequent filings with the SEC. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and we undertake no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Contacts: Investors: Stephanie Carrington ICR Westwicke 646-277-1282 Stephanie.Carrington@westwicke.com Media: Heidi Chokeir, Ph.D. Canale Communications 619-203-5391 heidi.chokeir@canalecomm.com