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Menarini Group’s Elacestrant Marketing Authorization Application Accepted for Review by the European Medicines Agency (EMA) for the Treatment of ER+/HER2- Advanced or Metastatic Breast Cancer

On August 19, 2022 The Menarini Group ("Menarini"), a privately held Italian pharmaceutical and diagnostics company, and Stemline Therapeutics ("Stemline"), a wholly-owned subsidiary of the Menarini Group, reported that EMA has validated the Marketing Authorization Application (MAA) for elacestrant, a selective estrogen receptor degrader (SERD), for patients with ER+/HER2- advanced or metastatic breast cancer (Press release, Menarini, AUG 19, 2022, View Source;Advanced-or-Metastatic-Breast-Cancer/?feedref=JjAwJuNHiystnCoBq_hl-RLXHJgazfQJNuOVHefdHP-D8R-QU5o2AvY8bhI9uvWSD8DYIYv4TIC1g1u0AKcacnnViVjtb72bOP4-4nHK5ieT3WxPE8m_kWI77F87CseT [SID1234618503]). Validation of the application confirms the submission is complete and begins EMA’s centralized review procedure.

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"There is a major unmet need in the treatment of advanced or metastatic ER+/HER2- breast cancer after resistance builds in the earlier lines of treatment," commented Elcin Barker Ergun, Chief Executive Officer of the Menarini Group. "The acceptance of our application for review by the EMA represents a significant step for our company and we look forward to working with the agency to potentially bring elacestrant to patients suffering from second- and third-line ER+/HER2- advanced or metastatic breast cancer in Europe."

The Phase 3 EMERALD study (NCT03778931) evaluated elacestrant compared to SOC endocrine monotherapy (investigators’ choice of either fulvestrant or an aromatase inhibitor) in ER+/HER2- advanced or metastatic breast cancer patients. The study results were recently published online in the Journal of Clinical Oncology (JCO) on May 18, 2022. Further post-hoc analysis from the study will be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2022 taking place September 9-13, 2022, in Paris, France.

The Menarini Group obtained global licensing rights for elacestrant in July 2020 from Radius Health, Inc, who conducted and successfully completed the EMERALD study. Based on the positive phase 3 data, Stemline submitted a MAA to EMA on July 27, 2022. The regulatory review for elacestrant is also underway in the U.S. as the Food and Drug Administration (FDA) has recently accepted a New Drug Application for elacestrant designating a Priority Review. The Menarini Group is now fully responsible for global registration, commercialization and further development activities for elacestrant.

About Elacestrant (RAD1901) and the EMERALD Phase 3 Study
Elacestrant is an investigational selective estrogen receptor degrader (SERD). In 2018, elacestrant received Fast Track designation from the FDA. Preclinical studies completed prior to EMERALD indicate that the compound has the potential for use as a single agent or in combination with other therapies for the treatment of breast cancer. The EMERALD Phase 3 trial is a randomized, open label, active-controlled study evaluating elacestrant as second- or third-line monotherapy in ER+/HER2- advanced/metastatic breast cancer patients. The study enrolled 477 patients who had received prior treatment with one or two lines of endocrine therapy, including a CDK 4/6 inhibitor. Patients in the study were randomized to receive either elacestrant or the investigator’s choice of an approved hormonal agent. The primary endpoint of the study was progression-free survival (PFS) in the overall patient population and in patients with estrogen receptor 1 gene (ESR1) mutations. Secondary endpoints included evaluation of overall survival (OS), objective response rate (ORR), and duration of response (DOR) and safety.

Accutar Biotechnology Receives NMPA Clearance of IND Application for AC0176 in Prostate Cancer

On August 19, 2022 Accutar Biotechnology, Inc., a clinical stage biotechnology company focusing on artificial intelligence (AI)-empowered drug discovery, reported that the China National Medical Products Administration (NMPA) has cleared the company’s investigational new drug application (IND) for AC0176 for the treatment of patients with metastatic Castration Resistant Prostate Cancer (mCRPC) (Press release, Accutar Biotechnology, AUG 19, 2022, View Source [SID1234618502]). AC0176 is an orally bioavailable chimeric degrader molecule designed to target Androgen Receptor (AR) protein with high potency and selectivity.

"Prostate cancer is one of the most common cancers among men in China, and the speed of increase in its incidence and death rates ranks highest in China. We are excited about the IND clearance of AC0176 in China, after its IND clearance and initiation of the first-in-human Phase 1 study in the US early this year," said Jie Fan, Ph.D., Chief Executive Officer, Accutar Biotechnology, Inc. "We look forward to accelerating the development of AC0176 and furthering our commitment to bringing innovative medicines to patients worldwide."

The Phase 1 study in China will assess the safety, tolerability, pharmacokinetics, and preliminary anti-tumor activity of AC0176 treatment in Chinese patients with mCRPC. Accutar expects to begin enrollment of this study in the second half of this year.

About AC0176

AC0176 is an investigational orally bioavailable, chimeric degrader of androgen receptor (AR) for the potential treatment of prostate cancers. AR is a hormonal transcription factor, and plays important roles during prostate cancer onset and progression. In preclinical studies, AC0176 has demonstrated potent and selective AR protein degradation with broad coverage of AR mutants, favorable pharmacological properties, as well as promising anti-tumor activities in animal models.

AskGene Announces Appointment of Chief Medical Officer and VP of Preclinical & Clinical Pharmacology

On August 19, 2022 AskGene Pharma Inc. (AskGene) reported the appointment of Barbara Hickingbottom, J.D., M.D. as Chief Medical Officer and Matt Hsu, Ph.D. as VP of Preclinical and Clinical Pharmacology (Press release, AskGene Pharmaceuticals, AUG 19, 2022, View Source [SID1234618501]). Dr. Hickingbottom will be responsible for leading the global clinical development of AskGene, including pipeline strategy, clinical development plans and operations, product registration, and guiding the global clinical collaborations with existing and potential partners. Dr. Hsu will be responsible for leading global preclinical and clinical pharmacology activities.

Jian-Feng (Jeff) Lu, Ph.D., CEO of AskGene commented: "We are pleased to welcome Dr. Hickingbottom and Dr. Hsu to our management team earlier this year. They will each bring us over two decades of global industry experience, which will help AskGene to continue to optimize our clinical development and global registration strategies for our lead biological products and to progress our promising pipeline of novel drugs positioned to address major areas of unmet clinical needs. We believe the appointment will bring strong energy and leadership to AskGene for our global clinical development and facilitate regulatory approval of lead innovative cytokine projects and other innovative molecules."

Barbara Hickingbottom J.D., M.D., CMO of AskGene commented: "I am profoundly excited to bring my experience to AskGene pipeline and join AskGene at this important time as ASKG315 is entering into the clinical stage in Australia. The early preclinical data for ASKG315 and ASKG915 are very promising, and I look forward to working alongside AskGene’s highly skilled scientific teams to bring the whole AskGene portfolio to patients globally."

Dr. Matt Hsu, VP of Preclinical and Clinical Pharmacology commented: "AskGene’s innovative SmartKine platform with a prodrug approach for cytokine therapies has demonstrated promising anti-tumor activities in preclinical species. With strong efficacy and reduced toxicity, this technology has a potential to overcome the limitation of cytokine use clinically and revolutionize immunotherapies. Therefore, I am looking forward to working with the company’s leadership team to advance novel and highly innovative clinical projects and candidates in the areas of oncology and immunological diseases, and to build a robust pipeline that has the opportunity to transform care for many more patients in need around the world."

Before joining AskGene, Dr. Hickingbottom served as the Head of Clinical Development at Xencor, Inc. (Xencor). During her time at Xencor, she was deeply involved in the clinical development of 12 anti-tumor biological innovative drugs in Xencor’s pipeline and responsible for all of the company’s clinical-stage products. Prior to Xencor, Dr. Hickingbottom worked as both a consultant and a scientist in many biopharmaceutical companies, including BioMarin, Mannkind, Novartis, Xoma, Novacea, and CTL Immunotherapies. Dr. Hickingbottom is an accomplished clinical pathologist with over 25 years of life sciences experience in clinical development, medical affairs, and registration strategy. She received her Medicine Doctor degree from the Tufts University School of Medicine and completed her internship and residency in pathology at Stanford. She also received a Juris Doctor degree from the Harvard Law School.

Dr. Hsu has more than two decades of experiences in drug development, ranging from proof of concept to approval. Previously, Dr. Hsu was Senior Principal Scientist in the Clinical Pharmacology Modeling & Simulation group at Amgen, where he led the Technical and Professional Unit for clinical study data and report preparation, conducted clinical pharmacology studies in translational sciences, applied PK/PD knowledge for dose selection at IND and End-of-Phase 2 stages, and prepared NDA/BLA submission for drug approvals. Prior to Amgen, Dr. Hsu worked in Preclinical Development at Johnson & Johnson in New Jersey and received his Ph.D. in Pharmaceutical Sciences from the University of Michigan. He has published more than 35 peer-reviewed papers.

Scandion Oncology receives approvals for next parts of the CORIST trial

On August 19, 2022, Scandion Oncology (Scandion), a biotech company developing first-in-class medicines aimed at treating cancer which is resistant to current treatment options, reported that it has now received approvals from authorities and ethical committees in Denmark and Germany for the next parts of the ongoing CORIST phase II-trial (Press release, Scandion Oncology, AUG 19, 2022, View Source,c3615707 [SID1234618499]). The trial studies Scandion’s lead compound SCO-101 as a combination treatment in patients with metastatic colorectal cancer (mCRC). Thus, the development of SCO-101 can continue as planned and communicated in connection with Scandion’s recently completed rights issue.

Part 1 of the CORIST trial has been completed and part 2 is currently ongoing with topline data expected to be communicated during the third quarter of 2022. To ensure a seamless continuation of the trial, parts 3 and 4 have been planned, and part 3 is ready to start independently of part 2. Patient recruitment in part 3 is expected to commence during the third quarter of 2022, and topline results from CORIST part 3 are expected most likely within the third quarter of 2023.

CORIST part 3 represents an expansion of the development program, in order to best exploit SCO-101’s potential in mCRC. As described in connection with the rights issue the expansion is expected to approximately double the commercial potential of SCO-101 in this indication.

The trial is expanded by adding a new schedule for combining SCO-101 and chemotherapy (FOLFIRI), which will be evaluated in patients with both RAS wild-type and RAS mutated tumors. The expansion was done through an amendment of the ongoing trial, making it possible to initiate the expansion while finalizing part 2, thereby keeping the momentum in the trial and utilizing already activated trial sites.

"We are pleased to have received formal approvals so swiftly and thereby be able to advance the CORIST trial as soon as practically possible. The expansion of the trial will greatly enhance both the medical and commercial potential of SCO-101 and we are delighted to be able to invest in maximizing the value of this compound to the benefit of patients, physicians and Scandion", says Bo Rode Hansen, President & CEO of Scandion.

The CORIST trial is also carried out in Spain. Parts 3 and 4 of the trial are expected to recruit patients here as well as in Denmark and Germany pending one outstanding approval in Spain.

European Commission approves Oncopeptides’ Pepaxti for the treatment of patients with relapsed refractory multiple myeloma

On August 18, 2022 Oncopeptides, a biotech company focused on research and development of therapies for difficult-to-treat hematological diseases, reported that the European Commission has granted Pepaxti[] (melphalan flufenamide, also called melflufen) marketing authorization in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy (Press release, Oncopeptides, AUG 18, 2022, View Source [SID1234646791]). For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation. The marketing authorization is valid in all EU member states, as well as in the European Economic Area (EEA) countries Iceland, Lichtenstein, and Norway.
The marketing authorization is based on data from the phase 2 HORIZON study and is supported by data from the randomized controlled phase 3 OCEAN study as confirmatory study. Oncopeptides intends to submit a type II variation in Q4 2022 to enable access to earlier lines of treatment for patients with relapsed refractory multiple myeloma (RRMM).

"The approval of Pepaxti in Europe is foundational for Oncopeptides, and brings excellent news for patients and shareholders," says Jakob Lindberg, CEO Oncopeptides AB. "Despite the introduction of novel therapies, patients with triple class refractory disease have a high unmet medical need, since their treatment options ultimately become exhausted."

Oncopeptides will now advance market access activities to pave the way for a successful launch of Pepaxti in Germany in Q4, 2022. The Company is dedicated to making the drug available for patients across Europe and is actively considering various options to commercialize the product.

For further information, please contact:

Rolf Gulliksen, Global Head of Corporate Communications, Oncopeptides AB (publ)
E-post: [email protected]
Mobil: + 46 70 262 96 28

The information in the press release is information that Oncopeptides is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person above, on August 18, 2022, at 13:00 (CET).

About HORIZON study

The HORIZON study is a pivotal phase 2 study, that evaluated melflufen in combination with dexamethasone, in heavily pre-treated patients with poor prognosis. This multi-center single arm study evaluated 157 patients with relapsed or refractory multiple myeloma, of whom 97 were triple-class refractory and had received at least four prior lines of treatment. The efficacy results for triple-class refractory patients who have received at least 3 prior lines of therapies and who had no ASCT or progressed more than 36 months after an ASCT in the HORIZON study is outlined below:

Response (n=52) HORIZON study (assessed by investigator)
Overall response rate (ORR), 95% CI (%) 28.8% (17.1%, 43.1%)
Duration of response (DOR) 95% CI (months) 7.6 (3.0-12.3)
Time to response (TTR) (months) 2.3 (1.0-10.5)
About Pepaxti

Pepaxti (melphalan flufenamide, also called melflufen) is a lipophilic peptide conjugated alkylating drug that rapidly and selectively is delivering cytotoxic agents into tumor cells. The drug is composed of a di-peptide and an alkylating moiety. The lipophilicity allows a faster cellular uptake whereas the peptide hydrolysis mediated by aminopeptidases, results in accumulation of alkylating moieties in cancer cells. This results in an improved efficacy without an increased toxicity compared to melphalan. Pepaxti inhibits proliferation and induces apoptosis of hematopoietic and solid tumor cells. It shows synergistic cytotoxicity in combination with dexamethasone in melphalan resistant and non-resistant multiple myeloma cell lines.

Pepaxti is indicated in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapy, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapies. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.

About Multiple Myeloma

Multiple myeloma is a cancer that originates in plasma cells, a type of white blood cells which produce antibodies to help fight infection, and cause cancer cells to accumulate in the bone marrow. Multiple Myeloma is the second most common hematologic malignancy, and accounts for approximately 1-2% of all new cancer cases, with a global incidence rate of 1.7 per 100,000 and an age-standardized incidence rate of 2.1-3.4 per 100,000 in France, Germany, Italy, Spain, and the UK. An estimated 35,842 patients were diagnosed in the EU27 during 2020, with an estimated 23,275 deaths due to the disease (ECIS 2020).

Patients with multiple myeloma may have symptom-free periods, but the disease always relapses, and patients may become refractory to all available treatment options due to mutations and/or clonal evolution of the tumor cells. A growing subset of patients are triple-class refractory, and develop disease refractory to immunomodulatory drugs, proteasome inhibitors, and CD38- targeting monoclonal antibodies. These patients have a very short expected overall survival.