On September 21, 2023 Biocept, Inc. (Nasdaq: BIOC), a leading provider of molecular diagnostic assays, products and services, reported the full enrollment of 40 subjects with breast or non-small cell lung cancer (NSCLC) who have suspicious or confirmed leptomeningeal metastases (LM) in the feasibility phase of its prospective FORESEE clinical trial (NCT0414123) (Press release, Biocept, SEP 21, 2023, View Source [SID1234635329]). This trial is evaluating the performance of Biocept’s proprietary CNSide assay in monitoring the response to therapy of LM, a cancer in the membranes that surround the brain and spinal cord, and assessing its impact on treatment decisions made by physicians.

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"Early adoption and reorder rates are encouraging and suggest that neuro-oncologists are finding the information generated by CNSide as useful in managing patients with this devastating cancer."

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"Completing enrollment in this first phase of our FORESEE trial, and doing so ahead of our internal timeline, is significant as we work toward establishing CNSide as standard of care under the National Comprehensive Cancer Network, or NCCN, guidelines," said Antonino Morales, Biocept President and CEO. "We believe obtaining standard-of-care status is the best path forward to support further physician adoption and to set reimbursement at a rate that reflects the value of our test in the clinical decision making process. The FORESEE trial is specifically designed to measure the impact of CNSide on physicians’ clinical decisions to generate the data needed to help us reach this goal.

"Our CNSide assay is the first commercially available method that has the potential to objectively measure the presence of tumor in the central nervous system (CNS), as well as help guide and monitor therapy, an area of critical need for these terminally ill patients," he added. "Early adoption and reorder rates are encouraging and suggest that neuro-oncologists are finding the information generated by CNSide as useful in managing patients with this devastating cancer."

FORESEE is a two-part prospective clinical trial designed to follow subjects and collect data from each enrollee at four key time points in their treatment, as well as to compare CNSide cell detection in the cerebrospinal fluid to that of conventional cytology. CNSide has notable advantages over current standards of care, such as cytology, clinical evaluation and MRI, which have limited sensitivity and specificity. In retrospective pilot studies, CNSide demonstrated 92% sensitivity and 95% specificity in detecting LM. Additionally, CNSide is both qualitative and quantitative, which are key to monitoring treatment response and improving the ability of physicians to make or change treatment decisions.

"The current standards of care can present a significant obstacle in patient care due to the limitations in the detection and monitoring of LM. In addition to the current methods, there is a need for a reliable tool to diagnose and to monitor response to treatments in patients with LM," said Jonathan Yang, MD, PhD at the University of Washington and principal investigator at this site of the FORESEE trial. "In my practice I’ve found that CNSide provides important information that augments the detection and monitoring of LM, which is becoming increasingly important as patients with LM are living longer with improved quality of life due to improvement in treatments."

Biocept expects to have results from the feasibility phase of the FORESEE trial in the first half of 2024 and to then begin enrolling between 40 and 100 subjects in the trial’s validation phase. Enrollment is currently open at four clinical sites with two additional sites expected to join the FORESEE trial in the near term.

About CNSide

CNSide is a laboratory developed test (LDT) based on Biocept’s proprietary quantitative tumor cell capture and detection method, paired with assays to identify actionable molecular treatment targets. Given the genetic changes that can occur as metastatic cancer spreads to the CNS, the evaluation of cerebrospinal fluid with CNSide provides a unique opportunity to identify biomarkers in patients with metastatic carcinoma or melanoma to help guide physicians in therapy selection. In addition, the quantitative tumor cell count assay can be used in a serial fashion to monitor the response to therapy more effectively than other current methods.

On September 21, 2023 JURA Bio, a biotechnology company developing immune-based therapeutics using machine learning and synthetic biology, reported a research collaboration with Syena, a cell therapy product company and subsidiary of Replay, a genome writing company, to develop T cell receptor (TCR) based therapies (Press release, JURA Bio, SEP 21, 2023, View Source [SID1234635328]).

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"We’re thrilled to announce our first partnership with Replay and its product company Syena. This partnership validates our approach of using synthesized human T cell repertoires to generate safe and effective libraries to discover antigen specific TCRs at scale," said Elizabeth Wood, Ph.D., Founder & CEO, JURA Bio. "The human immune system is a powerful source of safe and effective immune receptors, and while one patient might lack a TCR necessary to fight cancer, it may be present in another. By leveraging machine learning to rewrite the gene synthesis process from the ground up, we can produce extraordinarily high-quality immune receptors libraries to discover and train probabilistic machine learning models to ensure a faster development process that identifies TCRs recognizing the most challenging therapeutic targets."

"JURA Bio’s highly differentiated TCR discovery platform has transformative potential and combines the power of synthetic biology with that of machine learning. We look forward to working with them to identify high performing TCRs recognizing challenging but therapeutically important targets such as KRAS G12D," said Adrian Woolfson, Executive Chairman, President, and Co-Founder of Replay. "JURA Bio’s unique AI tools and expertise in synthetic libraries offers a differentiated approach for discovering TCRs. This will be invaluable in helping Replay’s cell therapy product company Syena to advance novel TCR-NK therapies into the clinic."

Lachlan MacKinnon, CEO and Co-Founder of Replay, said: "We are delighted to be partnering with JURA Bio, and their innovative team of synthetic and computational biologists to develop highly differentiated TCR-NK cell therapies. This new partnership is an exciting opportunity for Replay’s cell therapy product company Syena to leverage JURA Bio’s powerful toolkit of machine learning and synthetic protein design coupled with their extensive expertise and to use this to advance the development of our engineered TCR-NK cell therapy programs."

JURA Bio will receive an upfront payment as well as research funding for the period of the partnership. The details of the financial terms of the agreement were not disclosed. If the option is exercised, Replay and its cell therapy product company Syena will be responsible for global development and hold exclusive worldwide commercialization rights on all TCR-NK therapies resulting from the partnership. JURA Bio will be eligible to receive development, regulatory, and commercial milestone payments in addition to tiered deferred option payments on global net sales for products based on TCRs arising from the collaboration, as well as royalties on products utilizing at least one of the licensed technologies.

Replay launched Syena, an oncology-focused product company, with The University of Texas MD Anderson Cancer Center in February 2023. The engineered TCR-NK technology in Syena is based on the scientific discoveries of Katy Rezvani, M.D., Ph.D., professor of Stem Cell Transplantation & Cellular Therapy at MD Anderson.

"In areas where conventional methods are insufficient, machine learning emerges as the catalyst that unlocks the full potential of cell-based therapy and personalized medicine," said Julie Norville, Founder and Chief Technical Officer, JURA Bio. "We founded the company in 2017 as a synthetic biology and probabilistic machine learning entity. Our team has built all the tools necessary to define machine-learning driven candidates at an incredible scale, and has been able to test and develop these with incredible speed. We look forward to working with Replay and its cell therapy product company Syena to develop new treatments to address cancers with high unmet medical need."

In addition to the partnership with Replay and Syena, JURA Bio also announced a $16.1M financing led by Michael Chambers, John Ballantyne, Fontus Capital, and Josh Elkington. The funding will accelerate the mapping of the adaptive immune system. JURA Bio’s aims to complete a predictive map of TCR-antigen-HLA binding powered by an off-the-shelf library of >100B synthesized human T cells and their cognate antigens and HLAs by the end of 2024. It will during this timeframe also expand its machine-learning based gene synthesis into the design and discovery of B cell receptors.

Most existing generative machine learning workflows face a fundamental bottleneck in their ability to synthesize and test their candidates of choice. JURA has built an ML-first workflow that incorporates the relevant underlying chemistry, physics, and biology, as well as developing a statistical framework for evaluating model quality that allows them to build and train flexible and reliable models from huge amounts of data. JURA Bio is able to propose, build, and physically assay candidates at a scale previously unimaginable.

JURA Bio’s ML-improved gene synthesis technology has generated an off-the-shelf library of 100 billion potential human and improved TCR candidates that are ready for antigen-specific TCR discovery and development. The company has already discovered high-value TCRs for prostate cancer and other key neoantigen targets that have never been before recorded in key HLA-types.

In another example, JURA has generated a polyclonal candidate pool for six melanoma patients that were refractory to MART-1 specific adoptive cell therapy. Six refractory melanoma patients failed to respond to adoptive transfer of autologous cells from healthy donors. Using an off-the-shelf HLA-matched variational synthesis library of TCR candidates, JURA Bio identified 10 candidates for expansion and engineering to create a truly personalized therapeutic polyclonal candidate pool for each individual. The company has also manufactured peptidome and virodome-scale libraries of antigens to help determine the underlying mechanisms of autoimmunity.

"It is remarkable how little we know about the cell identities underlying and driving autoimmune disease. We are not able to reliably say how many are restricted to specific individuals or shared by common mechanisms population-wide. In having the ability to screen at the scale of human peptidomes, we will not only learn a lot about potential therapeutics, but also better understand the underlying heterogeneity of autoimmune disease," said Everett Meyer, Associate Professor of Medicine, Blood and Marrow Transplantation and Cellular Therapy, Director Stanford Cellular Immune Tolerance Program.

"AI-ML and multiplex libraries are useful tools individually, but when combined together they can generate remarkable synergy, known as ML-ML variational synthesis. This has the potential to generate billions to trillions-fold of potential candidates. The mastery of this strategy for TCR, MHC, and T-cell engineering at JURA is without competition and in huge demand," said George Church, Ph.D., Founder & Scientific Advisory Board Chair, JURA Bio.

Additional members of JURA Bio’s scientific advisory board include Debora Marks, PhD (Harvard), David Hafler, MD (Yale), Jay Fishman, MD (Harvard/MGH), Melina Fan, PhD (Addgene), Kevan Herold (Yale), Lester Mackey (Microsoft Research/Stanford), and Everett Meyer, MD/PhD (Stanford).

On September 21, 2023 Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to alisertib, a selective, small-molecule, orally administered inhibitor of aurora kinase A, for the treatment of patients with small cell lung cancer (SCLC) (Press release, Puma Biotechnology, SEP 21, 2023, View Source [SID1234635327]). SCLC is an aggressive form of lung cancer with a poor prognosis, and with limited treatment options for patients whose cancer has progressed on or after platinum-based chemotherapy.

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"Obtaining Orphan Drug Designation from the FDA signifies our continued progress and commitment to the development of alisertib for the treatment of small cell lung cancer," said Alan H. Auerbach, Chief Executive Officer, President and Founder of Puma. "There is an urgent need for new treatments for patients with small cell lung cancer, and we look forward to the initiation of our Phase II trial (Study PUMA-ALI-4201) of alisertib in small cell lung cancer."

The FDA grants Orphan Drug Designation to investigational therapies being developed to treat, diagnose or prevent a rare disease or condition affecting fewer than 200,000 people in the United States. Further, Orphan Drug Designation provides benefits to drug developers, including assistance in the drug development process, tax credits for qualified trials, waiver of certain FDA fees, and the potential for seven years of post-approval marketing exclusivity.

Puma received FDA clearance of its Investigational New Drug application for the clinical development of alisertib monotherapy for the treatment of patients with extensive stage SCLC in August 2023 and anticipates initiating the Phase II trial in the second half of 2023.

On September 21, 2023 Leucid Bio ("Leucid" or the "Company"), a privately-held biotechnology company pursuing the development of innovative Chimeric Antigen Receptor T-cell (CAR-T) therapies using the Company’s proprietary Lateral CAR platform, reported that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted clinical trial authorisation (CTA) to commence the Phase 1/2 AERIAL clinical trial evaluating the safety and tolerability of LEU011 for the treatment of adults with relapsed or refractory solid tumours (Press release, Leucid Bio, SEP 21, 2023, View Source [SID1234635326]).

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In September 2022, LEU011 received the innovative medicine designation, the Innovation Passport, as part of the Innovative Licensing and Access Pathway (ILAP) from the MHRA for the treatment of solid tumours expressing NKG2D ligands. Established in 2021, ILAP is a pathway supporting innovative approaches to the safe, timely and efficient development of medicines to improve patient access.

"The MHRA’s authorisation and the ILAP designation for LEU011 highlight the strong potential of our proprietary lateral CAR T candidate LEU011 for the treatment of solid tumours," commented Filippo Petti, Chief Executive Officer, Leucid Bio. "The development of LEU011 demonstrates our commitment to pushing the boundaries in cell therapy research and underpins our dedicated focus on pursuing novel, durable therapies for cancer patients with limited treatment options."

Dr. John Maher, Chief Scientific Officer, Leucid Bio, stated, "The design of the AERIAL trial, which was inspired by our exciting preclinical data for LEU011, is aimed at assessing the broad potential of the innovative cell therapy across several solid tumours expressing NKG2D ligands. We look forward to initiating the AERIAL study over the next few months and providing preliminary first-in-human data for LEU011 in 2024."

About LEU011 and AERIAL Trial

LEU011 is an autologous, lateral CAR T cell therapy targeting NKG2D ligands. The NKG2D receptor enables immune recognition of one or more of the eight human NKG2D ligands expressed on transformed, infected or damaged cells. LEU011 has the potential for the treatment of multiple cancer indications as NKG2D ligands are reported to be expressed on more than 80% of human tumours.

The Phase 1/2 AERIAL trial will evaluate the safety and clinical activity of LEU011 in patients with relapsed or refractory solid tumours following preconditioning chemotherapy. The Phase 1/2 trial consists of an open-label, single-ascending dose design which will identify the maximum tolerated dose for LEU011. Following the dose-escalation portion of the trial, LEU011 will further be evaluated in a dose expansion open-label segment with enrolment of patients with solid tumours expressing one or more NKG2D ligands.

On September 21, 2023 PAVmed Inc. (Nasdaq: PAVM, PAVMZ) ("PAVmed"), a diversified commercial-stage medical technology company operating in the medical device, diagnostics, and digital health sectors, and its majority-owned subsidiary Lucid Diagnostics Inc. (Nasdaq: LUCD) ("Lucid" or "Lucid Diagnostics"), a commercial-stage cancer prevention diagnostics company, reported their participation in the 2023 Cantor Fitzgerald Global Healthcare Conference in New York City (Press release, PAVmed, SEP 21, 2023, View Source [SID1234635325]).

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Lishan Aklog, M.D., PAVmed and Lucid’s Chairman & Chief Executive Officer, and Dennis McGrath, PAVmed’s President and Chief Financial Officer and Lucid’s Chief Financial Officer, will participate in a panel presentation on Wednesday, September 27, 2023, at 8:00 a.m. EDT.