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Cogent Biosciences Announces Final Results from PLX9486 Phase 1/2 Study in Advanced GIST Patients at CTOS 2020

On November 18, 2020 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported the final results from its PLX9486 + sunitinib Phase 1/2 study in patients with advanced gastrointestinal stromal tumors (GIST) (Press release, Cogent Biosciences, NOV 18, 2020, View Source [SID1234571323]). The data will be presented, summarized and discussed in a live oral session on Friday, November 20th from 11:30 a.m. to 12:30 p.m. ET at the Connective Tissue Oncology Society (CTOS) 2020 virtual meeting.

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PLX9486 is a selective tyrosine kinase inhibitor designed to potently inhibit KIT exon 17 mutations, including D816V. Most patients with imatinib-resistant GIST have both primary and secondary KIT mutations, often including secondary mutations on exon 17 and exon 13, making it difficult to achieve broad therapeutic KIT inhibition. These data suggest that treatment with a combination of PLX9486, a type 1 KIT inhibitor with activity against exon 17 mutations, and sunitinib, a type 2 KIT inhibitor with activity against exon 13 mutations, may provide substantial clinical benefit over treatment with type I or type II inhibitors alone.

"We are pleased to share final results from our Phase 1/2 trial of PLX9486 + sunitinib and are excited to advance this combination into a Phase 3 GIST trial in the second half of 2021," said Andrew Robbins, President and CEO of Cogent Biosciences. "With a median progression free survival (PFS) of 12 months in a heavily pre-treated population of advanced GIST patients, this combination holds significant promise for these patients with unmet medical need."

Oral Presentation:

Abstract: 3458521

Title: The potent and selective kit inhibitor PLX9486 dosed in combination with sunitinib demonstrates promising progression free survival (PFS) in patients with advanced gastrointestinal stromal tumor (GIST): final results of a phase 1/2 study.

Date: November 20, 2020

Time: Session 8: 11:30 a.m. – 12:30 p.m. ET

"The final results from this Phase 1/2 trial are highly encouraging for GIST patients who have limited treatment options due to secondary resistance KIT mutations," said Jonathan Trent, M.D., Ph.D., Associate Director for Clinical Research, Sylvester Comprehensive Cancer Center, University of Miami Health System. "I look forward to participating in the upcoming trial of this promising combination for imatinib-resistant GIST patients."

Results:

Out of the 18 patients with advanced GIST enrolled in the trial, all patients had received prior treatment, including 67% of patients with at least three prior lines of therapy. Doses for this study included 3 levels:

Level 1: PLX9486 500mg + sunitinib 25mg (3 patients)
Level 2: PLX9486 1000mg + sunitinib 25mg (5 patients)
Level 3: PLX9486 1000mg + sunitinib 37.5mg (10 patients)
Among the 15 patients who had not previously received PLX9486 as a single agent, the median progression free survival (PFS) was 12 months, the confirmed ORR was 20% and the clinical benefit rate (CR+PR+SD) was 80%, with 27% of patients remaining on therapy out 27-34 months. Importantly, there were no dose limiting toxicities in the three dose levels tested, and the most common adverse events were anemia, hypophosphatemia, diarrhea, and lymphopenia.

Neurologist and Psychiatrist Prof. Johannes Rolf Streffer, MD Joins AC Immune SA as Chief Medical Officer

On November 18, 2020 AC Immune SA (NASDAQ: ACIU), a Swiss-based, clinical-stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, reported that Prof. Johannes R. Streffer, an authority on neurology and psychiatry, was hired as the Company’s Chief Medical Officer, and he is expected to join the Company in January 2021 (Press release, AC Immune, NOV 18, 2020, View Source [SID1234571322]).

Prof. Streffer joins AC Immune from UCB Biopharma SPRL where he currently holds the position of Vice President, Head of Translational Medicine Neuroscience. His expertise will further accelerate AC Immune’s progress in advancing its pipeline of therapies targeting neurodegenerative diseases.

Prof. Andrea Pfeifer, CEO of AC Immune SA, commented: "We welcome Prof. Streffer’s timely appointment, as his understanding of biomarkers and experience of clinical study design in Alzheimer’s disease (AD), and particularly preclinical AD, will positively impact AC Immune’s discovery efforts. His appointment is a further affirmation of our scientific leadership in the neurodegenerative disease field. Prof. Streffer’s significant expertise and standing in the scientific and medical community will be an invaluable asset as we work to develop innovative treatments for neurodegenerative diseases using our proprietary technology platforms."

Prof. Streffer commented: "I am humbled and proud to be invited to join AC Immune, an established pioneer at the forefront of discovering and developing treatments for neurodegenerative diseases. It is tremendously exciting to be involved at the cutting edge of research addressing an area with such a high unmet medical need and where my work on clinical studies and understanding early biomarkers in Alzheimer’s could play a critically important role. The existing and developing pipeline of AC Immune is very impressive. I look forward to working with the outstanding team at AC Immune and hope my own expertise will contribute to their continued success."

At UCB Prof. Streffer led the development of a translational space in neuroscience, integrating clinical and biomarker sciences. Prior to this he was a member of the Alzheimer Disease Area Leadership Team at Janssen, Pharmaceutical Companies of Johnson & Johnson. Here he gained experience in early experimental AD trials, including biomolecular modalities such as positron emission tomography (PET), volumetric and functional MRI, genetics, cognition and cerebrospinal fluid markers. As a member of the Experimental Medicine group and as leader of the Janssen

BACE inhibitor (BACEi) program targeting Alzheimer’s Disease he initiated a number of methodological and biomarker studies focusing on early diagnosis and translation. Prof. Streffer was also the industrial lead for European Medical Information Framework (EMIF)-AD. In the Innovative Medicines Initiative (IMI)-EMIF program researchers from 14 countries combined in a EUR 56 million project to integrate a wide variety of AD data cohorts to foster understanding of early biomarkers and change in the predementia AD spectrum.

Prof. Streffer is a graduate of the University of Tübingen, Germany, where he received his medical degree. He also completed graduate studies on neuro-oncology and is Board certified in psychiatry and neurology. He has published more than 70 manuscripts and is a visiting Professor in the Department of Biomedical Sciences at the University of Antwerp.

Synlogic to Present at Upcoming Virtual Banking Conference

On November 18, 2020 Synlogic, Inc. (Nasdaq: SYBX), a clinical stage company bringing the transformative potential of synthetic biology to medicine, reported that Aoife Brennan, M.B. Ch.B., Synlogic’s President and Chief Executive Officer, and other members of the executive team will present at the following virtual banking conference (Press release, Synlogic, NOV 18, 2020, View Source [SID1234571320]):

Piper Sandler 32nd Annual Virtual Healthcare Conference: Dr. Brennan will participate in a ‘Fireside Chat’ at 10:00 am on Monday, November 23, 2020.
These are virtual events. Presentations will be available for registered attendees via the Piper Sandler conference site from November 23 to December 3. A live webcast of the presentations can be accessed under "Event Calendar" in the Investors & Media section of the Company’s website. An archived copy of the webcast will be available on the Synlogic website for approximately 30 days after the event.

Obsidian Therapeutics & MD Anderson announce strategic collaboration to accelerate advancement of novel engineered tumor infiltrating lymphocyte therapy (cytoTIL™) for solid tumors

On November 18, 2020 Obsidian Therapeutics, Inc. and The University of Texas MD Anderson Cancer Center reported a multi-year strategic collaboration designed to expedite the research and development of novel engineered tumor infiltrating lymphocytes (TILs) for the treatment of solid tumors (Press release, Obsidian Therapeutics, NOV 18, 2020, View Source [SID1234571319]). The agreement pairs Obsidian and its novel cytoDRiVE technology platform with MD Anderson’s extensive experience and state-of-the-art capabilities in TIL cell therapy, led by the Biologics Development platform, within the Therapeutics Discovery division.

The collaboration is focused on developing TIL armored with regulated membrane-bound IL15 (referred to as cytoTIL) with the potential to enhance anti-tumor efficacy and reduce tumor burden in patients suffering from different types of solid tumors. The teams will collaborate to accelerate the development of cytoTIL, including process and analytical development and clinical readiness activities.

"TIL therapy has emerged as a promising option for treating patients with solid tumors, though its widespread use today is limited by safety and efficacy challenges," said Rodabe Amaria, M.D., associate professor of Melanoma Medical Oncology at MD Anderson. "We are pleased to work with Obsidian to advance their novel cytoTIL program, which has the potential to drive more durable treatment responses and expand TIL therapy to a broader group of our patients."

The cytoTIL therapy is engineered using Obsidian’s cytoDRiVE platform technology, which precisely and reversibly controls protein expression and activity using FDA-approved orally bioavailable drugs. By leveraging regulated membrane-bound IL15 to drive antigen-independent expansion of T cells and transactivation of NK cells, cytoTIL therapy is anticipated to improve patient response to TIL treatment and expand patient eligibility to those who currently cannot benefit from this transformative therapy.

"We are delighted to work with MD Anderson’s Biologics Development team to build upon the success of first generation TIL therapies and bring the first controllable TIL therapy to patients as rapidly as possible," said Paul Wotton, Ph.D., CEO of Obsidian Therapeutics. "Through its cell therapy research platforms, deep clinical development experience, and industrial manufacturing capabilities, MD Anderson is a best-in-class collaborator to advance and accelerate cutting-edge cell therapies."

MD Anderson’s Biologics Development platform is built around an experienced team focused on pioneering impactful biologic therapeutics, including antibodies and cell therapies. With a state-of-the-art 60,000 sq. foot GMP cell-therapy manufacturing facility, the platform joins MD Anderson expertise with the rigor of industrial development. Biologics Development offers a strong starting point for early-stage companies to access the breadth of MD Anderson capabilities in cell therapy development.

MD Anderson is implementing an Institutional Conflict of Interest Management and Monitoring Plan for any research related to this relationship.

Kronos Bio Reports Recent Business Progress and Third Quarter 2020 Financial Results

On November 18, 2020 Kronos Bio, Inc. (Nasdaq: KRON), a company dedicated to transforming the lives of those affected by cancer, reported recent business progress and third quarter 2020 financial results (Press release, Kronos Bio, NOV 18, 2020, View Source [SID1234571318]).

"Kronos Bio experienced transformational growth in 2020. With the acquisition of the portfolio of SYK inhibitors from Gilead Sciences, we are positioned to initiate late-stage clinical testing next year of our lead investigational therapy entospletinib in patients with newly diagnosed NPM1-mutated acute myeloid leukemia. Furthermore, in the first half of 2021, we plan to begin clinical testing of KB-0742, our CDK9 inhibitor, in patients with advanced solid tumors," said Norbert Bischofberger, Ph.D., president and CEO. "We have expanded our leadership team and filled key roles across the organization. Additionally, with the completion of two financing rounds, including our upsized IPO, we are well-capitalized to execute on our mission to develop and deliver therapies that have the potential to change patients’ lives."

Recent Highlights

In July 2020, acquired Gilead Sciences’ portfolio of spleen tyrosine kinase (SYK) inhibitors, including entospletinib, which Kronos Bio is developing as a frontline treatment for patients with NPM1-mutated acute myeloid leukemia (AML).

Expanded the company’s leadership team with the hiring of Barbara Kosacz, chief operating officer and general counsel, and Dr. Yasir Al-Wakeel, chief financial officer and head of corporate development, and appointed Elena Ridloff to the company’s Board of Directors as chair of the audit committee.

In September 2020, closed a private financing round of approximately $155.2 million of convertible notes.

In October 2020, completed an upsized initial public offering (IPO) of 15,131,579 of common stock, including full exercise of the underwriters’ option to purchase additional shares, resulting in gross proceeds of $287.5 million, before deducting underwriting discounts and commissions and offering expenses.

In October 2020, published in Cell Chemical Biology the results of a preclinical study of KB-0742, the company’s potent oral, highly selective cyclin dependent kinase 9 (CDK9) inhibitor being developed to treat MYC-amplified solid tumors. These results were previously presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II in June 2020.
Third Quarter Financial Highlights

Cash, Cash Equivalents and Short-Term Investments: As of September 30, 2020, cash, cash equivalents and short-term investments totaled $211.3 million, which excludes approximately $263.9 million in net proceeds from the company’s October 2020 upsized IPO.

R&D Expenses: Research and development expenses were $16.8 million for the third quarter of 2020, which includes $0.2 million in stock-based compensation expense.

G&A Expenses: General and administrative expenses were $4.1 million for the third quarter of 2020, which includes $1.0 million in stock-based compensation expense.

Net Loss: Net loss was $39.7 million, or $6.48 per basic and diluted share, for the quarter ended September 30, 2020. This includes a non-recurring $15.2 million non-cash change in fair value of convertible notes.