On September 8, 2021 HiberCell, a clinical stage biotechnology company developing therapeutics to treat cancer relapse and metastasis, reported that the U.S. Food and Drug Administration (FDA) has granted the company Fast Track designation for HC-5404-FU, an orally administered PERK inhibitor (PERKi) for patients with solid tumors (Press release, HiberCell, SEP 8, 2021, View Source [SID1234587387]). As reported in June 2021, the company initiated a first-in-human, Phase 1a clinical trial for HC-5404-FU in select solid tumors filed their Investigational New Drug (IND) application, and received FDA authorization to begin clinical studies in late 2020.

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A Fast Track designation from the FDA provides several benefits for HiberCell’s HC-5404-FU PERKi therapeutic, including more frequent meetings with the FDA to discuss the drug development plan, additional written communications from the FDA regarding the design of the proposed clinical trials, the use of biomarkers, and Rolling Review, which means HiberCell can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until all sections of the NDA are completed. Additionally, with this designation, HC-5404-FU will automatically be considered for Accelerated Approval and Priority Review. Accelerated Approval is based on a surrogate endpoint reasonably likely to predict clinical benefit; Priority Review shortens the FDA review process from ten months to six.

"The Fast Track designation is an important step as HiberCell focuses on advancing this first-in-class clinical asset," said Alan Rigby, PhD, chief executive officer at HiberCell. "Importantly, this designation will allow us to work closely with the FDA to quickly move HC-5404-FU through our ongoing clinical trial, HC-404-FCP-2011. The trial is a first in-human, Phase 1a, multi-center, open-label study to establish the maximum tolerated dose (MTD) and evaluate the safety and tolerability following oral dosing of HC-5404-FU in a dose-escalating fashion. Up to 24 qualified subjects at five US sites, who have specific solid tumors, including renal cell carcinoma (RCC), gastric cancer (GC), and other solid tumors are planned for enrollment in this study. This Phase 1a study will be expanded into a Phase 1b/2a through a protocol amendment with the dose and tumor type(s) identified in Phase 1a as the most appropriate for further clinical development."

On September 8, 2021 GlycoMimetics, Inc. (Nasdaq: GLYC) reported that Chief Executive Officer Harout Semerjian will present at the upcoming H.C. Wainwright & Co 23rd Annual Global Investment Virtual Conference taking place September 13-15, 2021 (Press release, GlycoMimetics, SEP 8, 2021, https://ir.glycomimetics.com/news-releases/news-release-details/glycomimetics-present-upcoming-hc-wainwright-co-annual-global-0 [SID1234587386]). The presentation will be available on the company’s website at the "Investors" tab for 30 days, beginning Monday, September 13 at 7:00 a.m. EDT.

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To access the live webcast and subsequent archived recordings for the presentation, please visit the GlycoMimetics website at View Source

On September 8, 2021 XBiotech Inc. (NASDAQ: XBIT) ("XBiotech") reported that Benjamin Musher M.D., will chair XBiotech’s clinical program for pancreatic cancer (Press release, XBiotech, SEP 8, 2021, View Source [SID1234587376]). The Company is developing a novel cancer drug, Natrunix, to be used in combination with cytotoxic anti-cancer agents. Natrunix specifically blocks a substance that has been shown to stimulate tumor blood supply and break down connective tissue, which can support tumor growth and destruction of healthy tissue. The anti-inflammatory activity of Natrunix is also being evaluated for its ability to reduce the toxicity and injury caused by the cytotoxic anti-cancer agents themselves.

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XBiotech’s pancreatic cancer program, 1-BETTER, launched with a randomized, double-blind, placebo-controlled Phase 1-2 study, allows the Company to evaluate dose levels, drug interaction and anti-cancer activity for Natrunix. Dr. Musher is Medical Director of Medical Oncology at Dan L. Duncan Comprehensive Cancer Center and Associate Professor at Baylor College of Medicine. Dr. Musher is a member of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) and has authored numerous peer-reviewed articles in oncology, including treatments for pancreatic cancer. In addition to Natrunix, Dr. Musher has investigated cutting-edge pancreatic cancer therapies, including the use of tumor-targeting viruses. He has also researched and published on clinical practices relating to the treatment of pancreatic cancer, to better understand the decision-making processes and challenges faced by oncologists in the treatment of this extremely challenging form of cancer.

As chair of the 1-BETTER pancreatic cancer program, Dr. Musher will lead the clinical program and personally treat pancreatic cancer patients at the Dan L. Duncan Comprehensive Cancer Center, at St. Luke’s Hospital in Houston, Texas. Dr. Musher stated, "Pancreatic cancer is a devastating disease and remains the third leading cause of cancer-related death in the United States. Most patients with metastatic pancreatic cancer are eligible for only cytotoxic chemotherapy, which generally yields short-lived responses and can cause significant toxicity. More effective and rationally designed therapies are therefore desperately needed. By targeting cancer-related inflammation, Natrunix may facilitate better control of tumor growth, reduce toxicity from chemotherapy, and improve well-being of patients with advanced pancreatic cancer."

Dr. Musher will oversee the 1-BETTER study that will involve at least 20 other leading cancer centers around the country. The study will evaluate Natrunix in combination with ONIVYDE and 5-fluorouracil and generate preliminary data on overall survival, progression-free survival, time-to-treatment failure, and objective response rates. Numerous quality-of-life measures will also be explored, hopefully showing a reduction in chemotherapy-related toxicity.

John Simard, President and CEO of XBiotech commented, "We are honored to have Dr. Musher chair this study for which we are eagerly anticipating results."

About True Human Therapeutic Antibodies
XBiotech’s True Human antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.

On September 8, 2021 Sprint Bioscience AB (publ) reported that the company is broadening its pipeline with an additional drug program for the treatment of solid tumors (Press release, Sprint Bioscience, SEP 8, 2021, View Source [SID1234587375]). The program goes by the program name NIMA and aims to develop drugs to inhibit a naturally occurring protein that is associated with poor survival in several different forms of cancer.

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"We have once again been able to identify a target protein that constitutes a very attractive starting point for a new drug program. The ambition is to influence the microenvironment of the tumors so that the growth of cancer cells is inhibited and the body’s immune system can more easily fight them. We will in a time- and resource-efficient way drive the project to a point where it can dock into an international pharmaceutical company’s portfolio and be taken further to clinic development towards the market and cancer patients who need new treatment option"
Erik Kinnman, CEO, Sprint Bioscience

An important aspect of the development of a tumor is that the cancer cells can affect their environment to facilitate the growth of the tumor. This environment is called the tumor microenvironment and differs from the environment around the healthy cells in the body. The ability of cancer cells to reprogram the healthy cells present in the tumor facilitates tumor growth and can inhibit the body’s immune system.

The NIMA program focuses on a target protein that produces metabolites that are important for this reprogramming of the tumor’s microenvironment. Sprint Bioscience develops inhibitors of this target protein to restore balance and thereby prevent the growth of cancer cells while increasing the immune system’s ability to attack them. High levels of the current target protein have been found in tumors from, amongst others, ovarian cancer, breast cancer, and colorectal cancer. Patients with high levels of this protein have a poorer prognosis.

"We have once again been able to identify a target protein that constitutes a very attractive starting point for a new drug program. The ambition is to influence the microenvironment of the tumors so that the growth of cancer cells is inhibited and the body’s immune system can more easily fight them. We will in a time- and resource-efficient way drive the project to a point where it can dock into an international pharmaceutical company’s portfolio and be taken further to clinic development towards the market and cancer patients who need new treatment option,", says Erik Kinnman, CEO, Sprint Bioscience.

The initiation of the NIMA program is part of the company’s accelerating strategy to broaden its project portfolio in the cancer area and thereby create opportunities for further licensing deals that can generate additional multiple revenue streams.

On September 8, 2021 Sanofi reported that it has entered into a definitive merger agreement with Kadmon Holdings, Inc. (NASDAQ: KDMN) a biopharmaceutical company that discovers, develops, and markets transformative therapies for disease areas of significant unmet medical needs (Press release, Sanofi, SEP 8, 2021, View Source [SID1234587374]). The acquisition supports Sanofi’s strategy to continue to grow its General Medicines core assets and will immediately add Rezurock(belumosudil) to its transplant portfolio. Rezurock is a recently FDA-approved, first-in-class treatment for chronic graft-versus-host disease (cGVHD) for adult and pediatric patients 12 years and older who have failed at least two prior lines of systemic therapy.

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Shareholders of Kadmon common stock will receive $9.50 per share in cash, which represents a total equity value of approximately $1.9 billion (on a fully diluted basis). The Sanofi and Kadmon Boards of Directors unanimously approved the transaction.

"We are transforming and simplifying our General Medicines business and have shifted our focus on differentiated core assets in key markets," said Olivier Charmeil, Executive Vice President General Medicines. "We are thrilled to add Kadmon’s Rezurock to our well-established transplant portfolio. Our existing scale, expertise, and relationships in transplant create an ideal platform to achieve the full potential of Rezurock, which will address the significant unmet medical needs of patients with chronic graft-versus-host disease around the world."

"We are excited that Sanofi has acknowledged the value of Rezurock and the deep potential of our pipeline," said Harlan Waksal, M.D., President and Chief Executive Officer, Kadmon. "By leveraging Sanofi’s global resources and long-standing expertise in developing and commercializing innovative medicines, Rezurock is now well positioned for global accessibility, faster. I want to thank the entire Kadmon team, including management and the Board of Directors, and the Sanofi organization, for their ongoing commitment to patients and their caregivers."

Sanofi’s transplant business mainly consists of Thymoglobulin (anti-thymocyte globulin), a polyclonal, anti-human thymocyte antibody preparation that acts as a broad immunosuppressive and immunomodulating agent and Mozobil (plerixafor), a hematopoietic stem cell mobilizer. Both products are among General Medicines core assets and are currently registered and marketed in more than 65 countries.

In July 2021, the FDA approved Rezurock for the treatment of adult and pediatric patients 12 years and older with cGVHD after the failure of at least two prior lines of systemic therapy. Rezurock was launched in August in the United States. It is the first and only approved small molecule therapy that inhibits the Rho-associated coiled-coil kinase 2 (ROCK2), a signaling pathway that modulates inflammatory response and fibrotic processes. Sanofi will work closely with regulatory authorities across different geographies to ensure that patients suffering from cGVHD can benefit from belumosudil treatment as early as possible. Kadmon is also developing Rezurock for the treatment of diffuse cutaneous systemic sclerosis, with an open-label Phase 2 clinical trial currently ongoing.

Kadmon’s pipeline includes drug candidates for immune and fibrotic diseases as well as immuno-oncology therapies.

The transaction is expected to be modestly dilutive to Sanofi’s EPS in 2022.

Transaction Terms

Under the terms of the merger agreement, holders of Kadmon’s common stock will receive $9.50 per share in an all-cash transaction, reflecting a total equity value of Kadmon of approximately $1.9 billion. The offer price represents a premium of 79% over the closing price on September 7, 2021 and a premium of approximately 113% over the 60 trading days volume weighted average price.

The consummation of the transaction is subject to customary closing conditions, including the approval of holders of a majority of the outstanding shares of Kadmon voting stock, the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and other customary conditions. Following the successful completion of the merger, a wholly owned subsidiary of Sanofi will merge with Kadmon and the outstanding Kadmon shares will receive $9.50 per share in cash. Sanofi plans to fund the transaction with available cash resources. Subject to the satisfaction or waiver of customary closing conditions, Sanofi expects to complete the acquisition in the fourth quarter of 2021.

Weil, Gotshal & Manges LLP is acting as legal counsel to Sanofi. Cantor Fitzgerald & Co. and Moelis & Company LLC are acting as exclusive financial advisors to Kadmon in the transaction, while DLA Piper LLP (US) is acting as legal counsel.