On May 26, 2021 Oncopeptides AB (publ) (Nasdaq Stockholm: ONCO), a global biotech company focused on the development of therapies for difficult-to-treat hematological diseases, reported positive topline results from the head-to-head phase 3 OCEAN study evaluating the efficacy and safety of melphalan flufenamide versus pomalidomide in patients with relapsed refractory multiple myeloma (RRMM) (Press release, Oncopeptides, MAY 26, 2021, View Source [SID1234580651]). The randomized study was initiated in 2017 and includes 495 patients from more than 100 hospitals in 21 countries around the world.

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"Following the accelerated approval of Pepaxto in the U.S. earlier this year, the positive topline results from the OCEAN study marks another major milestone for Oncopeptides. It is very exciting news for patients and indicates that melphalan flufenamide has the potential to become part of the standard of care in relapsed refractory multiple myeloma", says Marty J Duvall, Chief Executive Officer at Oncopeptides AB. "By demonstrating that melphalan flufenamide is at least as efficacious as pomalidomide, we are now able to begin the preparation of a sNDA which could pave the way for a potential use of melphalan flufenamide in earlier lines of therapy in a substantially larger patient population."

The PFS, as assessed by the independent review committee, demonstrated a Hazard Ratio* favoring melphalan flufenamide of 0.817 (95% CI: 0.659-1.012, p=0.0640) for the primary endpoint and shows that melphalan flufenamide is non-inferior to pomalidomide. The Hazard Ratio for PFS as per investigator assessment was 0.790 (95% CI: 0.639-0.976). In both assessments, the median PFS for the melphalan flufenamide arm was more than 40% higher than for the pomalidomide arm. The Overall Response Rate for melphalan flufenamide was 32.1% vs. 26.5% for pomalidomide.

"The efficacy and safety data from the OCEAN study are very encouraging, and the results will be of importance in physicians’ treatment decisions for patients with relapsed and refractory multiple myeloma," says Pieter Sonneveld, MD, PhD, Professor of Hematology at the Erasmus University of Rotterdam, and Principal Investigator of the OCEAN study. "Melphalan flufenamide has a unique mode of action, which in addition with its tolerability profile, makes the drug an attractive treatment option for many patients."

Melphalan flufenamide and pomalidomide had similar discontinuation rates for adverse events, and the safety profile of melphalan flufenamide was in line with previous studies and consistent across age subgroups. The Company expects to present complete OCEAN data at an upcoming scientific congress.

"The outcome from the phase 3 OCEAN study is very good news for patients with relapsed refractory multiple myeloma", says Klaas Bakker, MD, PhD, Executive Vice President and Chief Medical Officer at Oncopeptides. "This head-to-head-comparison was a bold study with an optimal design for demonstrating melphalan flufenamide ‘s true isolated treatment effects. Based on these data, Oncopeptides intends to submit a supplementary New Drug Application to the US Food and Drug Administration FDA, in Q4 2021, given the OCEAN data clearly show that melphalan flufenamide may be an important therapeutic option for the increasing number of patients who need more treatment alternatives. I am truly looking forward to sharing these data with a broader audience."

Melphalan flufenamide is evaluated in a comprehensive clinical study program. In addition to the phase 3 OCEAN study, Oncopeptides is currently enrolling patients in the phase 3 LIGHTHOUSE study, with the aim to establish the efficacy and safety of a combination therapy with melphalan flufenamide plus daratumumab compared to daratumumab, based on the successful results from the ANCHOR study, presented at American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2020.

Limitation of Use

PEPAXTO is not indicated and is not recommended for use as a conditioning regimen for transplant outside of controlled clinical trials.

To view the full prescribing information please visit View Source

About phase 3 OCEAN study

The phase 3 OCEAN study is a global, randomized, head-to-head, open-label study, evaluating the efficacy and safety of melphalan flufenamide and dexamethasone, versus pomalidomide and dexamethasone in patients with relapsed refractory multiple myeloma who have received 2-4 prior therapies. The patients have previously been treated with at least an immunomodulator agent, and a proteasome inhibitor. They have all developed resistance to their last line of therapy and to lenalidomide, the most used drug in multiple myeloma. The study was initiated in 2017 and includes 495 patients from more than 100 hospitals around the world. The primary efficacy endpoint is Progression Free Survival.

About Multiple Myeloma

Multiple myeloma is a cancer of plasma cells, a type of white blood cell which produces antibodies to help fight infection. Multiple myeloma causes cancer cells to accumulate in the bone marrow. Approximately 7 per 100,000 Americans per year are diagnosed with multiple myeloma, making it a rare disease. A growing subset of this population is becoming triple-class refractory. The number of patients diagnosed with multiple myeloma is growing and the number of cases diagnosed annually is expected to almost double in 20 years. The average age for diagnosis is 70 years of age, and there is currently no cure.

On May 26, 2021 AbbVie (NYSE: ABBV) reported that it will participate in Bernstein’s 37th Annual Strategic Decisions Conference on Wednesday, June 2, 2021. Michael Severino, M.D., vice chairman and president, and Robert A. Michael, executive vice president and chief financial officer, will present virtually at 2:30 p.m. Central time (Press release, AbbVie, MAY 26, 2021, View Source [SID1234580604]).

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A live audio webcast of the presentation will be accessible through AbbVie’s Investor Relations website at investors.abbvie.com. An archived edition of the session will be available later that day.

On May 26, 2021 Oncternal Therapeutics (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, reported that management will present a corporate overview at the Jefferies Virtual Healthcare Conference on Thursday, June 3, 2021 at 10:30 am Eastern Time (Press release, Oncternal Therapeutics, MAY 26, 2021, View Source [SID1234580620]).

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Replay Link:
A replay will be accessible on the Events & Presentations page of the Investors section on the Company’s website at View Source

On May 26, 2021 Repertoire Immune Medicines, a clinical-stage biotech company decoding the immune synapse to create novel immune therapies for cancer, immune disorders, infectious disease, and other serious diseases, reported a new collaboration with a Yale School of Medicine research team focused on identifying antigens expressed in patients with late-stage, advanced metastatic melanoma, and determining which of these antigens activate T cells in the tumor (Press release, Repertoire, MAY 26, 2021, View Source [SID1234580636]). The collaboration will seek to understand the specificity of various subsets of T cells with the goal of uncovering new antigens and their T cells, with potential for future incorporation into Repertoire’s cell therapy and other development programs.

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The study will discover new immunodominant epitopes and determine the specificity of T cells obtained from patients with metastatic melanoma. The university research team will provide human T cell receptor (TCR) sequences to Repertoire, which will use its proprietary DECODE platform to determine the novel antigens that these TCRs identify. The team’s deep understanding of translational science in oncology, coupled with Repertoire’s platform technology, may generate unique insights leading to the development of novel immune medicines.

The research team will be led by David A. Hafler, MD, FANA, William S. and Lois Stiles Edgerly Professor of Neurology and Professor of Immunobiology, Yale School of Medicine; Chair, Department of Neurology and Neurologist-in-Chief, Yale New Haven Hospital.

"We’re pleased to initiate this collaboration with Dr. Hafler and his team at Yale University, particularly given their deep expertise in pioneering translational science in difficult disease areas, including advanced melanoma," said Anthony Coyle, Ph.D., President, Research and Development, Repertoire Immune Medicines. "Our hope is that this collaboration will unveil breakthrough science that we can apply to transformational drug discovery and development of novel immune therapies for melanoma patients in need."

On May 26, 2021 NFlection Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on the development of targeted therapies for rare disorders driven by aberrant activation of the RAS pathway (RASopathies), reported positive results from a 28-day, Phase 2a, multicenter, randomized, double-blind, parallel-group, vehicle-controlled clinical trial investigating the safety, tolerability, pharmacokinetics and pharmacodynamics of NFX-179 Gel in subjects with NF1 (Press release, NFlection Therapeutics, MAY 26, 2021, View Source [SID1234580652]). Topical application of NFX-179 Gel is designed to deliver a proprietary "soft" (metabolically labile) MEK inhibitor to cNF tumors to suppress the overactivation of the Ras/Raf/MEK/ERK pathway in these tumors while avoiding the systemic toxicities of orally administered MEK inhibitors, which have not been approved for this indication.

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In the trial, 48 subjects were randomized in a 1:1:1:1 ratio to receive once-daily NFX-179 Gel at 0.05%, 0.15%, or 0.5%, or placebo (vehicle), for 28 days. The primary endpoints were safety, tolerability and suppression of p-ERK, a key biomarker known to promote the growth of cNF tumors.

NFX-179 Gel was well tolerated. No serious adverse events were reported, and no adverse events were classified as related to NFX-179 Gel. All adverse events were mild to moderate and occurred at similar frequencies in the treatment and vehicle groups. Orally administered MEK inhibitors have common adverse effects of rash, diarrhea, peripheral edema and fatigue. Unlike systemic MEK inhibitors, which can cause severe acneiform rash, acneiform rash was not observed during treatment with NFX-179 Gel. Forty seven of the 48 subjects completed the trial; one subject withdrew from the trial due to COVID-19 infection.

NF1 subjects carry a mutation in the gene encoding neurofibromin 1, a tumor suppressor that suppresses the Ras/Raf/MEK/ERK pathway. The resulting overactivation of this pathway leads to increased levels of p-ERK, which drives the growth of cNF tumors. Treatment of cNF tumors with NFX-179 Gel for 28 days induced a dose-dependent suppression of p-ERK in the tumors. Compared to vehicle-treated lesions, tumors treated with 0.5% and 0.15% NFX-179 Gel showed a statistically significant suppression in p-ERK. A 47% reduction (p = 0.0001) in p-ERK was observed in tumors treated with 0.5% NFX-179 Gel, and a 26% reduction (p = 0.04) in p-ERK was observed in tumors treated with 0.15% NFX-179 Gel. The lowest dose group, 0.05% NFX-179 Gel, gave a 10% reduction of p-ERK that was not statistically different from vehicle (p = 0.4). In addition, exploratory secondary endpoints demonstrated a tumor size reduction despite limiting treatment to only 28 days. There was a 17% mean reduction in tumor volume from baseline in the 0.5% NFX-179 Gel group versus an 8% reduction in the vehicle group (p = 0.073). In a per-subject responder analysis, 22% of subjects in the 0.5% NFX-179 group had a 50% or greater mean reduction in tumor volume, versus 6% of subjects in the vehicle group (p = 0.051).

Dr. Guy Webster, Chief Medical Officer of NFlection, said, "We are very pleased with these data, which demonstrate that NFX-179 Gel is well tolerated and induces clinically meaningful levels of p-ERK suppression in cNF tumors. The strong p-ERK biomarker data, along with an unexpected early trend in cNF tumor volume reduction after only 28 days of treatment, support our hypothesis that NFX-179 Gel is an important novel therapy for NF1 patients. We look forward to progressing NFX-179 Gel to Phase 2b to determine the effect of longer treatment duration on cNF tumor regression, as well as to initiating trials testing NFX-179 Gel for the treatment of other cutaneous RASopathies."

"We are delighted that our partnership with NFlection is delivering such encouraging results," said Annette Bakker, Ph.D., President, Children’s Tumor Foundation. "It is really exciting to already observe tumor shrinkage in the 28-day Phase 2a study.

This study provides hope that the NFX-179 Gel could become a life-changing solution for the NF patients suffering with painful and often disfiguring cutaneous neurofibromas, for which no approved pharmacological therapies exist today."

About NFX-179 Gel
NFX-179 is an investigational mitogen-activated protein kinase kinase (MEK) inhibitor. NFX-179 is a "soft" (metabolically labile) drug, which, when formulated as NFX-179 Gel for topical application, is designed to concentrate at the dermal site of action but degrade in systemic circulation, thereby significantly reducing side effects compared to systemically available MEK inhibitors. NFlection is developing NFX-179 Gel for the treatment of RASopathies such as cutaneous neurofibromatosis type 1, immunosuppressant-mediated cutaneous squamous cell carcinoma, and congenital birthmarks.

About Cutaneous Neurofibromatosis Type 1
Cutaneous neurofibromas are tumors that grow from small nerves in the skin or just under the skin and appear as small or larger bumps typically beginning around the time of puberty. Individuals with NF1 commonly develop more cutaneous neurofibromas as they get older. They do not become malignant, but they may be disfiguring, itchy or painful when bumped. Despite their benign nature, they may cause significant problems (e.g., depression, isolation, etc.), and may require surgical removal.