On June 29, 2022 ALX Oncology Holdings Inc., ("ALX Oncology") (Nasdaq: ALXO) a clinical-stage immuno-oncology company developing therapies that block the CD47 checkpoint pathway, reported that the U.S. Food and Drug Administration ("FDA") granted orphan drug designation ("ODD") to evorpacept, a next-generation CD47 blocker, for the treatment of patients with acute myeloid leukemia ("AML") (Press release, ALX Oncology, JUN 29, 2022, View Source [SID1234616353]).

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"Receiving orphan drug designation in AML, and previously in gastric cancer, from the FDA is an important regulatory milestone and reflects the FDA’s recognition of evorpacept’s potential to improve clinical outcomes in patients with these advanced cancers," said Sophia Randolph, M.D., Ph.D., Chief Medical Officer, ALX Oncology. "In our ongoing Phase 1/2 ASPEN-05 study (NCT04755244), we are excited to evaluate the combination of evorpacept with venetoclax and azacitidine in patients with previously untreated AML who are not candidates for intensive induction therapy or with relapsed/refractory AML."

The FDA’s Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include assistance in the drug development process, tax credits for qualified clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

About Acute Myeloid Leukemia

AML is an aggressive blood cell cancer that can rapidly progress and lead to death if not treated promptly. AML is the most common form of acute leukemia in adults, with an estimated 20,050 new cases and 11,540 deaths from AML in the United States in 2022. Due to advanced age and comorbidities at the time of diagnosis, a significant number of patients are not considered eligible for intensive and potentially curative therapies. Despite advances in available care, the estimated 5-year survival for patients in the United States with AML remains only 31%.

On June 29, 2022 Chimeric Therapeutics Ltd (ASX:CHM) reported that it has expanded its licensing agreement with the University of Pennsylvania to include non-exclusive access to its lentiviral vector plasmid system, a viral vector technology critical for the manufacture of chimeric antigen receptor (CAR) T cells (Press release, Chimeric Therapeutics, JUN 29, 2022, View Source [SID1234616350]).

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Licensing of this technology will support Chimeric’s efforts to develop and commercialise its CHM 2101 CDH17 CAR T technology, offering improved safety as a third-generation technology.

The amended licence agreement with Pennsylvania University will allow CHM to manufacture clinical-grade lentiviral vector for use in its planned Phase 1 study of CHM2101 for gastrointestinal cancers.

Chimeric will also be able to cross-reference regulatory information on file with the US Food and Drug Administration (FDA) to support the filing of an investigational new drug (IND) designation for CHM 2101.

CDH17 CAR T technology
CHM 2101 (CDH17 CAR T) is based on a novel third-generation iteration of the CDH17 CAR T technology invented by the University of Pennsylvania.

The treatment is at a pre-clinical development stage, with plans for a Phase 1 clinical trial assessing neuroendocrine tumours, colorectal, gastroesophageal and gastric cancer already in the works.

Preclinical data has indicated a particular efficacy in solid tumours, having demonstrated the complete eradication of tumour cells with no relapse and no toxicity to normal tissue.

On July 28, 2022 VolitionRx Limited (NYSE AMERICAN: VNRX) ("Volition"), a multi-national epigenetics company, reported that it has commenced an underwritten public offering of its common stock. All of the shares of common stock are being offered by Volition (Press release, VolitionRX, JUN 28, 2022, View Source [SID1234617284]). In addition, Volition intends to grant the underwriter in the offering a 30-day option to purchase up to an additional 15% of the number of shares of common stock offered in the offering at the public offering price, less underwriting discounts and commissions. The final terms of the proposed offering will depend on market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering.

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Newbridge Securities Corporation is acting as the sole book-running manager of the offering.

The securities are being offered by Volition pursuant to a "shelf" registration statement on Form S-3 (File No. 333-259783) previously filed with the Securities and Exchange Commission (the "SEC") on September 24, 2021 and declared effective by the SEC on November 8, 2021. The offering is being made only by means of a prospectus supplement and an accompanying base prospectus, as may be further supplemented by any free writing prospectus and/or pricing supplement that the Company may file with the SEC. A preliminary prospectus supplement and an accompanying base prospectus describing the terms of the proposed offering have been filed with the SEC and are available on the SEC’s website at www.sec.gov. Copies of the preliminary prospectus supplement and the accompanying base prospectus relating to the offering can also be obtained, when available, from Newbridge Securities Corporation, Attn: Equity Syndicate Department, 1200 North Federal Highway, Suite 400, Boca Raton, FL 33432, by email at [email protected], or by telephone at (877) 447-9625. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

On June 28, 2022 Elevar Therapeutics, Inc., a fully integrated biopharmaceutical company dedicated to elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, reported the hiring of Jan M. Van Tornout, M.D., MSc., as chief medical officer, and Gordon Schooley, Ph.D., as chief regulatory officer (Press release, Elevar Therapeutics, JUN 28, 2022, View Source [SID1234616351]).

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Van Tornout, an accomplished hematologist-oncologist, genetic epidemiologist and pharma-biotech executive, joins Elevar after serving as acting chief medical officer of biotechnology company Tyme Technologies, Inc. since March 2021. He brings more than 25 years’ experience in the medical industry and related academia, including over 15 years successfully developing drugs across the full spectrum of hematological, oncological and immuno-oncological indications from pre-investigational new drug (IND) meeting through Phase 4.

"Jan Van Tornout’s broad experience and demonstrated leadership in every phase of the clinical development process, and his interest in the potential of drugs that address gaps in critical medical treatments, make him an ideal addition to the Elevar team," said Dr. Saeho Chong, chief executive officer of Elevar. "We are pleased to add him to our leadership team."

Van Tornout’s clinical management and development experience includes work for Moleculin Biotech, Inc., Bright Peak Therapeutics, Cyclacel Pharmaceuticals, GlaxoSmithKline, Puma Biotherapeutics, Maverick Therapeutics, ERT Inc., HUYA Bioscience Int’l, Gradalis Inc., Inovio Pharmaceuticals, Natera Inc., Bristol-Myers Squibb and Amgen. He played an instrumental role in two successful NDAs, led several INDs and has expertise in all phases of clinical trials.

Van Tornout previously held academic appointments at the University of Southern California (USC), with a clinical appointment as attending pediatric hematologist-oncologist at Children’s Hospital Los Angeles. He earned his Doctor of Medicine from the Katholieke Universiteit Leuven (KUL), Leuven, Belgium, a Master of Science in classical philosophy from KUL and a Bachelor of Science from the Faculté Notre-Dame de la Paix, Namur, Belgium. He obtained his certification as a pediatrician from the Gasthuisberg University Hospitals, KUL, and completed his training as a pediatric hematologist-oncologist at Children’s Hospital. Van Tornout earned a Master of Science in applied biometry from USC and completed a post-doctoral fellowship in molecular epidemiology at USC.

Schooley, a global regulatory leader with more than 30 years of drug development experience, joins Elevar after nine years as chief regulatory officer for BeyondSpring Pharmaceuticals Inc., a global, clinical-stage biopharmaceutical company that develops immuno-oncology cancer therapies.

"Careful navigation of the road to regulatory approval is so critical to the clinical development process and Gordon Schooley has demonstrated his leadership in this discipline throughout his distinguished career," said Chong. "His addition to the Elevar team adds valuable perspective with respect to both regulatory strategy and execution, and will bolster our efforts to fulfill the unmet needs of patients awaiting new therapeutic options."

Schooley has spent more than a quarter century at the vice president level or higher, working in both startup and large company environments. He’s led global development teams through every clinical development milestone; worked closely with regulatory bodies in several different countries; overseen successful breakthrough designations and accelerated approvals; and managed complex label negotiations for initial product approvals.

Prior to BeyondSpring, Schooley spent seven years as a consultant for drug development activities related to clinical development, biostatistics and regulatory affairs. He previously worked at SkyePharma PLC/Pacira Pharma, Alliance Pharmaceuticals and Allergan/Herbert Labs.

Schooley holds a doctorate in biostatistics from the University of Michigan School of Public Health. He earned a Master of Science in statistics and Bachelor of Science in business management at Brigham Young University.

On June 28, 2022 IDEAYA Biosciences, Inc. (Nasdaq: IDYA), a synthetic lethality focused precision medicine oncology company committed to the discovery and development of targeted therapeutics, reported that selection of a potential first-in-class Pol Theta Helicase development candidate (DC) (Press release, Ideaya Biosciences, JUN 28, 2022, View Source [SID1234616348]).

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The Pol Theta Helicase DC is a potential first-in-class small molecule inhibitor of the helicase domain of DNA Polymerase Theta. IDEAYA is collaborating with GSK on IND-enabling studies to support the evaluation of the Pol Theta Helicase DC in combination with niraparib, GSK’s PARP inhibitor, for patients having tumors with BRCA or other homologous recombination (HR) mutations or homologous recombination deficiency (HRD).

"We are excited about potential clinical development opportunities for this potential first-in-class Pol Theta Helicase inhibitor. Pol Theta promotes DNA repair by Microhomology-Mediated End-Joining (MMEJ), an error-prone mutagenic DNA repair pathway, which is active in BRCA mutant and other HRD cancer cells. PARP1 is also involved in MMEJ DNA repair, supporting a hypothesis for synergistic combination of our Pol Theta Helicase DC with niraparib," said Michael White, Senior Vice President and Chief Scientific Officer of IDEAYA Biosciences.

"The development candidate has demonstrated robust in vivo efficacy in combination with niraparib, with significant tumor regressions and durable responses in multiple cancer models. We believe the Pol Theta helicase and niraparib combination has the opportunity to deliver meaningful patient benefit," said Benjamin Schwartz, Ph.D., Vice President, Head of Oncology Synthetic Lethality Research Unit at GSK.

IDEAYA and GSK are targeting an IND submission for the Pol Theta Helicase DC, subject to satisfactory completion of ongoing preclinical and IND-enabling studies, to enable first-in-human studies in the first half of 2023.

IDEAYA and GSK are collaborating on the ongoing IND-enabling studies, and GSK will lead clinical development for the Pol Theta program. GSK holds a global, exclusive license to develop and commercialize the Pol Theta Helicase DC and is responsible for all research and development costs for the program, including those incurred by IDEAYA. IDEAYA is eligible to receive future development and regulatory milestones of up to $485 million aggregate, inclusive of preclinical and clinical milestones of up to $10 million aggregate for advancing this asset through IND effectiveness.

Upon potential commercialization, IDEAYA will be eligible to receive up to $475 million of commercial milestones and tiered royalties on global net sales by GSK, its affiliates and their sublicensees ranging from high single digit to sub-teen double digit percentages, subject to certain customary reductions.