On May 26, 2021 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, and Addario Lung Cancer Medical Institute (ALCMI) reported they will present on the progress of "Biomarker Analysis in High PD-L1 Expressing NSCLC Patients Treated With PD-1/PD-L1 Based Therapy With or Without the Addition of Platinum Based Chemotherapy (BEACON-Lung) (Press release, Biodesix, MAY 26, 2021, View Source [SID1234580654])." The BEACON-Lung study aims to evaluate biomarkers to predict overall survival and early progression outcomes in treatment-naïve advanced stage non-small cell lung cancer (NSCLC) patients with high PD-L1 expression. The first patient participant enrolled in the study this week.

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"The BEACON-Lung study furthers ALCMI’s mission to work with partners like Biodesix to overcome barriers to treatment in lung cancer," said Tony Addario, chair and CEO of ALCMI. "This study will enable us to develop a better understanding of how biomarkers can unlock treatment options for patients with NSCLC and improve survivorship."

The study, being conducted through ALCMI’s prestigious research consortium of leading US academic centers, launched early this year. Nearly half of the targeted 10 sites are actively enrolling with a target enrollment of 390 patients. BEACON-Lung will use Biodesix’s Primary Immune Response (PIR) test to classify patients receiving PD-1/PD-L1 therapy or PD-1/PD-L1 plus carboplatin-based chemotherapy and identify those who may benefit from more aggressive treatment regimens.

"As an oncologist, I am excited by the progress we have made for patients who are now diagnosed with non-small cell lung cancer," said Dr. Mary Jo Fidler, principal investigator for the BEACON trial and associate professor in the Department of Internal Medicine, Division of Hematology, Oncology and Cell Therapy at Rush Medical College. "However, to identify the best treatment course for an individual patient still requires the development of biomarkers, especially in the field of immunotherapy. BEACON-Lung is a study that aims address this current gap in biomarker development and treatment selection for advanced non-small cell lung cancer."

The BEACON-Lung study is designed to generate key data to further the development of immunotherapy biomarkers for treatment naïve NSCLC patients. Currently, PDL1 expression is the only clinically validated biomarker predicting patient response to front line PD-1/L1 directed immunotherapy. However, PD-L1 expression remains an imperfect marker, and this study will also help determine if patients should pursue other treatment regimens.

"We are thrilled that the BEACON-Lung study is now launched," said Dr. James Jett, CMO of Biodesix. "This study will allow us to evaluate performance of Biodesix’s PIR test in the treatment naïve NSCLC patient population receiving immunotherapy. We are committed to the advancement of biomarker research and to fulfill an unmet need in the NSCLC treatment landscape."

Presentation Title: Biomarker Analysis in High PD-L1 Expressing NSCLC Patients Treated With PD-1/PD-L1 Based Therapy With or Without the Addition of Platinum Based Chemotherapy (BEACON-Lung)
Authors: Mary J. Fidler, Victoria Meucci Villaflor, Amol Rao, Balazs Halmos, Erin Marie Bertino, Raymond U. Osarogiagbon, David Paul Carbone; Rush University Medical Center, Chicago, IL; Northwestern University Feinberg School of Medicine and Robert H. Lurie Comprehensive Cancer Center of Northwestern University, Chicago, IL; Memorial Care, Laguna Hills, CA; Albert Einstein College of Medicine and Montefiore Medical Center, Bronx, NY; The Ohio State University Comprehensive Cancer Center, Columbus, OH; Baptist Cancer Center, Multidisciplinary Thoracic Oncology Department, Memphis, TN
Abstract Number: TPS9126
Session Date/Time: June 4, 2021 / After 9am E.T.

About ALCMI
The Addario Lung Cancer Medical Institute (ALCMI, voiced as "Alchemy"), founded in 2008 as a 501c(3) non-profit organization by lung cancer survivor Bonnie J Addario, is a patient-centric, international research consortium driving research otherwise not possible. Working in tandem with its "partner" foundation, GO2 Foundation for Lung Cancer, ALCMI powers collaborative initiatives in genetic (molecular) testing, therapeutic discoveries, targeted treatments, and early detection. ALCMI combines scientific expertise found at its network of 26 member academic institutions through its network of community cancer centers to accelerate patient access to research.

On May 26, 2021 Milestone Pharmaceuticals Inc. (Nasdaq: MIST), a biopharmaceutical company focused on the development and commercialization of innovative cardiovascular medicines, reported that Joseph Oliveto, President and Chief Executive Officer, will present at the Jefferies Virtual Healthcare Conference on Wednesday, June 2, 2021 at 4:00 PM ET (Press release, Milestone Pharmaceuticals, MAY 26, 2021, View Source [SID1234580653]). The conference will be held in a virtual meeting format.

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A live webcast of the presentation can be accessed in the News & Events section of Milestone’s website at www.milestonepharma.com. An archived replay of the webcast will be available on the same website following the presentation.

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.

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 Gracell Biotechnologies Inc. (NASDAQ: GRCL) ("Gracell"), a global clinical-stage biopharmaceutical company dedicated to discovering and developing highly efficacious and affordable cell therapies for the treatment of cancer, reported that it will host a key opinion leader (KOL) webinar on the challenges of treating High Risk Multiple Myeloma and a potential novel approach on Friday, June 11, 2021 at 8:00am Eastern Time (Press release, Gracell Biotechnologies, MAY 26, 2021, View Source;a-novel-approach-for-treatment-of-multiple-myeloma-301299911.html [SID1234580650]).

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The call will feature a presentation by Dr. Andrzej Jakubowiak, University of Chicago Medicine, who will give an overview on the current treatment landscape and unmet medical need in treating patients with high-risk multiple myeloma. Dr. Martina Sersch, Chief Medical Officer of Gracell, will present clinical data of GC012F, a BCMA/CD19 dual-targeting CAR-T therapy for multiple myeloma manufactured on Gracell’s proprietary FasTCAR overnight manufacturing platform.

Following the formal presentations, there will be a fireside chat moderated by Neil Canavan of LifeSci Advisors and Author of "The Cure Within" with Dr. Jakubowiak and members of the Gracell management team who will discuss various topics related to the FasTCAR technology platform and the lead clinical program GC012F, a BCMA/CD19 dual-targeting CAR-T therapy. FasTCAR is Gracell’s autologous CAR-T platform that tackles the most pressing challenges associated with autologous therapies, including lengthy manufacturing time, suboptimal manufacturing quality, high therapy cost, and poor T cell fitness.

A live Q&A session will follow the formal presentations and fireside chat.

To register for the event, please click here.

About the KOL

Andrzej Jakubowiak, M.D., Ph.D., is an internationally known expert on multiple myeloma, a cancer of the plasma cells in a patient’s bone marrow. He works closely with the Multiple Myeloma Research Consortium (MMRC) to bring the latest treatments to the patient’s bedside as quickly as possible.

Dr. Jakubowiak’s primary research focus is in the development of new drugs for the treatment of multiple myeloma. He is currently the lead investigator on a number of multi-site clinical trials for patients who are newly diagnosed, have relapsed, or have refractory (resistant to treatment) disease. Dr. Jakubowiak has received research funding and several grants from the MMRC. He is also the recipient of many honors, including the Myeloma Center of the Year award by the MMRC in 2008 and 2010.

A frequently invited lecturer, Dr. Jakubowiak has presented his research findings at medical meetings around the world. He has published more than 50 peer-reviewed articles as well as 14 book chapters. Additionally, he serves as an ad hoc reviewer for several scientific journals, including the Journal of Clinical Oncology, Blood and Leukemia and Lymphoma.

About GC012F

GC012F is a FasTCAR-enabled dual-targeting CAR-T product candidate that is currently being studied in an ongoing investigator-initiated Phase 1 trial across multiple centers in China for the treatment of MM. GC012F tackles MM by simultaneously targeting both malignant plasma cells expressing BCMA and early progenitor cells expressing CD19 in order to drive fast, deep and durable responses in MM patients.

About FasTCAR

CAR-T cells manufactured on Gracell’s proprietary FasTCAR platform appear younger, less exhausted and show enhanced proliferation, persistence, bone marrow migration and tumor cell clearance activities as demonstrated in preclinical studies. With overnight manufacturing, FasTCAR is able to significantly improve cell production efficiency which may result in meaningful cost savings, increasing the accessibility of cell therapies for cancer patients.