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Celcuity Announces Clinical Trial Collaboration with University of Rochester Wilmot Cancer Center and Puma to Study Patients with Breast Cancer Brain Metastases

On October 18, 2021 Celcuity Inc. (Nasdaq: CELC), a clinical-stage biotechnology company pursuing an integrated companion diagnostic and therapeutic strategy for treating patients with cancer, reported a clinical trial collaboration with the University of Rochester Wilmot Cancer Center and Puma Biotechnology (Nasdaq: PBYI), a biopharmaceutical company, to conduct a Phase 2 clinical trial (Press release, Celcuity, OCT 18, 2021, View Source [SID1234591471]).

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This open-label Phase 2 trial will evaluate the efficacy and safety of Puma’s drug, NERLYNX (neratinib), and Xeloda (capecitabine), a Genentech/Roche drug, in previously treated patients selected with Celcuity’s CELsignia HER2 Activity Test who have metastatic HER2-negative breast cancer. Under the agreement, The University of Rochester Wilmot Cancer Center will serve as the sponsor and Ajay Dhakal, M.D., a medical oncologist at the University of Rochester Medical Center, will serve as the principal investigator of this study. The University of Rochester Wilmot Cancer Center is one of the 51 NCI-Designated Comprehensive Cancer Centers in the U.S. tasked with developing new approaches to diagnosing and treating cancer.

"This clinical trial could play a key role in creating a new treatment paradigm for metastatic HER2-negative breast cancer patients with brain metastases," said Dr. Dhakal. "We are eager to begin working with Celcuity’s cutting-edge CELsignia technology to identify a new subset of patients who may respond to NERLYNX."

Puma will supply NERLYNX, its pan-HER inhibitor currently approved by the U.S. Food and Drug Administration (FDA) for early and late-stage HER2-positive breast cancer. Celcuity will provide its CELsignia HER2 Activity Test to select HER2-negative metastatic breast cancer patients with brain metastases who have hyperactive HER2-driven signaling pathways for the trial and will fund the patient-related trial costs. Based on its estimates of patient enrollment rates, Celcuity expects to obtain interim results 12 to 15 months after initiation of the trial followed by the final results 12 to 15 months later. Celcuity expects enrollment to begin by early to mid-2022.

The goal of the trial is to demonstrate that previously treated HER2-negative metastatic breast cancer patients with brain metastases who have hyperactive HER2 signaling tumors, as identified by the CELsignia test, respond to treatment with NERLYNX in combination with XELODA, a chemotherapy commonly used in metastatic breast cancer patients. Celcuity believes there is significant clinical interest in finding new diagnostic tests and targeted therapies for metastatic HER2- negative breast cancer patients with brain metastases.

"We are excited about the opportunity to collaborate with Dr. Dhakal, The University of Rochester Wilmot Cancer Center, and Puma Biotechnology on this important clinical trial," said Brian Sullivan, CEO and co-founder of Celcuity. "This will be our first collaboration to study metastatic breast cancer patients with brain metastases selected for treatment using our CELsignia HER2 Activity Test. Patients with HER2-negative breast cancer who have brain metastases have few good options today. Approximately 20% of the 280,000 HER2-negative breast cancer patients receiving drug treatment annually have tumors with hyperactive HER2 signaling.1 For these patients, this trial represents a critical step towards a potential new therapeutic option."

Phase Genomics Announces Next Generation Cytogenomics Platform to Advance Precision Diagnosis and Treatment in Reproductive Genetics and Oncology

On October 18, 2021 Phase Genomics, Inc., a biotech company leading advancements in next-generation sequencing (NGS) solutions for genome assembly and analysis, reported the launch of the beta version of its new platform for next generation cytogenomic applications in the reproductive genetics and oncology spaces (Press release, Phase Genomics, OCT 18, 2021, View Source [SID1234591470]).

The platform enables rapid, efficient sample processing and does not require culturing of live, dividing cells nor high-molecular-weight DNA extraction. "This is a fast and inexpensive platform that is capable of providing tremendous benefit towards answering complex genomic questions that existing methods are unable to fully resolve,” said Shawn Sullivan, Co-founder and CTO of Phase Genomics. "The flexibility of this method allows us to use a low starting volume of cells from fresh and frozen material and, most notably, paraffin-embedded tissue. We can deliver an invaluable compendium of genetic information from a single sample without having to wait for results from multiple tests. With our robust chemistry and the machine learning underpinnings of our analytic technology, our platform offers the potential to complement or replace incumbent technologies like cytogenetics, FISH, and CMA in both solid and liquid cancers and in reproductive health."

While the platform can be broadly applied to constitutional genetics, Phase Genomics sees the immediate utility that it can bring to the prenatal market. According to Sullivan, the platform can help provide answers to the study of patient populations faced with infertility or unexplained repeated pregnancy loss. "For example, our platform can detect cryptic rearrangements potentially causing a couple’s fertility issues, it can uncover novel structural and copy-number changes essential to fetal development in non-viable or paraffin-embedded POC tissue samples and, most importantly, help fuel the translation of these discoveries into clinical tools that will remove emotional, financial and other burdens borne by patients working through these heartbreaking conditions."

Many of the same challenges exist in oncology. Unlocking and maximizing the use of the genetic information contained in unculturable and paraffin-embedded cancer samples is a unique property of the Phase Genomics platform. Phase Genomics’ Co-founder and CEO Dr. Ivan Liachko stated, "The Phase Genomics ultra-long-range sequencing method and machine learning-enabled analytical platform arm clinical researchers, and eventually, healthcare providers, with a cost-effective, high-throughput, sequencing-based method that delivers actionable information. This information provides insights that can be used in the development of new diagnostic and treatment options for cancer and genetic disease, ultimately leading to improved patient care and outcomes."

Today’s announcement opens RUO platform access to early beta participants. Phase Genomics is engaged in a number of active partnerships with research and commercial entities and is presenting early results in a poster at the American Society of Human Genetics Conference. The Phase Genomics platform is for research use only and is not for use in diagnostic procedures. More information on the platform is available here.

Join Phase Genomics for a webinar on Tuesday, November 30th to learn more about this technology and follow Phase Genomics on Twitter for the latest news and information.

bluebird bio Sets Record Date and Distribution Date for Planned Business Separation

On October 18, 2021 bluebird bio, Inc. (NASDAQ: BLUE) reported that October 19, 2021 has been set as the record date for the dividend of shares of common stock of 2seventy to be distributed to bluebird stockholders in order to effect the separation of bluebird bio and 2seventy bio, Inc. into two independent, publicly traded companies (Press release, bluebird bio, OCT 18, 2021, View Source [SID1234591469]).

Each bluebird bio stockholder of record as of the close of business on October 19, 2021 will receive, on the distribution date, one share of 2seventy common stock for every three shares of bluebird common stock held. The share dividend is expected to be distributed to bluebird stockholders on or about November 4, 2021. Following the separation, bluebird stockholders will also receive cash in lieu of any fractional shares of 2seventy common stock that those holders would have received after application of the 3:1 distribution ratio. No action is required by bluebird stockholders in order to receive the shares of 2seventy common stock in the dividend distribution.

Additionally, on October 18, 2021 the Securities and Exchange Commission declared 2seventy’s Registration Statement on Form 10 (the "Form 10") effective. This Form 10 contains further information regarding bluebird bio’s plans for a tax-free spin-off of its oncology programs and portfolio into 2seventy bio as a publicly traded company, including the conditions to completion of the separation.

"When-issued" trading for 2seventy common stock and "ex-distribution" trading for bluebird common stock is expected to commence on October 18, 2021 under the stock ticker symbols "TSVTV" and "BLUEV", respectively. A description of these expected trading markets is included in the Form 10. After the separation, 2seventy common stock is expected to trade on the Nasdaq Global Select Market under the stock ticker symbol "TSVT" and bluebird will continue to trade on Nasdaq Global Select Market under the stock ticker symbol "BLUE."

TAE Life Sciences to Sponsor Industry Expert Theater “New Era in Biologically-Targeted Radiation Therapy” at 2021 ASTRO Annual Meeting

On October 18, 2021 TAE Life Sciences (TLS), a biological-targeted radiation therapy company developing next-generation boron neutron capture therapy (BNCT), reported the sponsorship of an Industry-Expert Theater session focused on advancements in BNCT at the American Society for Radiation Oncology (ASTRO) Annual Meeting, occurring October 24-27, 2021 (Press release, TAE Life Sciences, OCT 18, 2021, View Source [SID1234591468]).

The session, "New Era in Biologically-Targeted Radiation Therapy: Clinical Application and Technical Advancement in Boron Neutron Capture Therapy" will feature presentations by Timothy Malouff, M.D., Mayo Clinic, Ester Orlandi, M.D., National Center of Oncological Hadrontherapy (CNAO) and Kendall Morrison, Ph.D., Chief Scientific Officer, TAE Life Sciences. They will discuss the clinical application and technical advancements in BNCT for the treatment of invasive, recurrent and difficult to treat cancers.

"Our deep commitment to innovation in clinical practice is underscored by our research in BNCT as an emerging treatment modality for traditionally difficult to treat tumors," said Dr. Timothy Malouff, Mayo Clinic. "With clinical studies in a variety of disease sites including head and neck cancers, lung cancers, and breast cancers, BNCT holds the promise of transforming the cancer treatment landscape. We look forward to sharing an update on the current clinical research at ASTRO."

"We announced a partnership with TAE Life Sciences last year to provide BNCT for the treatment of invasive, recurrent and difficult to treat cancers," said Dr. Ester Orlandi, Medical Director at CNAO. "Taking part in this session enables us to provide an update on how we are providing a new pillar in cancer therapy."

Session Title: New Era in Biologically-Targeted Radiation Therapy: Clinical Application and Technical Advancement in Boron Neutron Capture Therapy
Location: Theater 1, Innovation Hub, McCormick Place West
Date: Monday, Oct 25 2:45 PM

About BNCT

BNCT is a combination treatment based on the reaction that occurs when a non-toxic compound containing boron-10 is irradiated with a low-energy neutron beam. BNCT differs radically from other radiation therapy and shows promise in becoming the next-generation cancer treatment. Research has shown BNCT has the capability of killing cancer cells that are resistant to traditional radiation therapy with limited harm to healthy tissue. Current advances in both neutron radiation technology and medicinal boron drug targeting are enabling BNCT’s potential to improve patient care while also improving treatment economics. To date, approximately 2,000 patients have been treated with BNCT at research sites worldwide.

NFlection Therapeutics Appoints William Hodder as CEO and Expands Leadership Team to Accelerate Development of Targeted Treatments for Rare Diseases

On October 18, 2021 NFlection Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing novel drug candidates for rare RASopathies, reported that it has appointed William Hodder as CEO (Press release, NFlection Therapeutics, OCT 18, 2021, View Source [SID1234591467]). The company also named Gerd Kochendoerfer, Ph.D., as Chief Operating Officer, Libbie Mansell, Ph.D., MBA, as SVP of Global Regulatory Affairs & Quality Assurance, and Patrice Horwath as VP of Clinical Operations.

Mr. Hodder brings over 30 years of broad biopharmaceutical industry experience including fundraising, business development, corporate strategy, product marketing, sales, and product development to NFlection, most recently serving as Chief Business Officer at Escient Pharmaceuticals where he was responsible for all financing, licensing and M&A activity, and overseeing corporate operations and administrative functions while developing and implementing infrastructure to support the growth of the organization.

At NFlection, Mr. Hodder will lead efforts to discover and develop effective, targeted therapies for rare disorders driven by aberrant activation of the RAS pathway (RASopathies), initially focusing on cutaneous neurofibromas in neurofibromatosis type 1, disfiguring congenital birthmarks (including keratinocytic epidermal nevi and nevus sebaceous), and immunosuppressant-mediated squamous cell carcinoma.

"NFlection has achieved several significant milestones in 2021, under the leadership of Chris Powala, including the FDA granting orphan drug designation for NFX-179 for the treatment of cutaneous neurofibromatosis type 1," said Mr. Hodder. "It is an exhilarating time to join NFlection as the company progresses NFX-179 Gel to Phase 2b, as we believe it is an important novel therapy for the treatment of RASopathies, such as cutaneous neurofibromatosis, and other unmet medical needs."

Further strengthening the leadership team, NFlection has appointed three key executives, including:

Gerd Kochendoerfer, Ph.D., has been named COO overseeing R&D operations. Dr. Kochendoerfer has over 20 years of experience in drug development, operations, and program management in the biotech and specialty pharma industry. Before NFlection, he was SVP and Head of Operations at PellePharm, a BridgeBio company, where he was responsible for oversight of R&D and built quality and business systems/infrastructure from the ground up for the development of an orphan cancer drug candidate with Breakthrough Designation status addressing a rare genetic disease.

Libbie Mansell, Ph.D., MBA, RAC, will serve as SVP of Global Regulatory Affairs & Quality Assurance. Dr. Mansell has over 30 years of experience in Product Registration and Development Strategy who has demonstrated success in translating innovative science and technology into high-quality, commercially attractive medicines for serious and/or rare diseases. Prior to joining NFlection, she served as SVP, Regulatory Affairs, for AskBio (acquired by Bayer in December 2020).

Patrice Horwath has been appointed VP of Clinical Operations, overseeing the day-to-day operation of all clinical development. Ms. Horwath has over 20 years of experience directing global pharmaceutical, medical device and combination product development and successfully led multiple clinical programs through Phase I-IV studies, global regulatory approvals, product launch and clinical affairs management including medical education and scientific communications. She joined NFlection from Kalika Biosciences/Ralexar Therapeutics.