On October 5, 2020 Unum Therapeutics Inc. ("Unum") (NASDAQ: UMRX), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported its name change to Cogent Biosciences, Inc. ("Cogent") (Press release, Unum Therapeutics, OCT 5, 2020, View Source [SID1234568092]). Beginning on October 6, 2020, Cogent will trade on NASDAQ under the ticker symbol "COGT", and Cogent’s common stock will trade under a new CUSIP number, 19240Q102. The new name reflects Cogent’s mission to design rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Cogent is also pleased to provide an update on recent scientific and operational progress since its July acquisition of Kiq LLC, including the announcement of an upcoming oral presentation of Phase 1/2 clinical data in gastrointestinal stromal tumors (GIST) with Cogent’s lead asset, PLX9486, at the 2020 Connective Tissue Oncology Society (CTOS) Annual Meeting to be held virtually November 18 – 21. In connection with the change of the Company’s corporate name, Cogent anticipates that the name of PLX9486 will be changed to CGT9486.

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"Our new name reflects the evolution of our company to focus on developing precision therapies for genetically defined diseases and paves the path forward as we strive to deliver real solutions for patients in serious need of new treatment options," said Chuck Wilson, PhD, President and CEO of Cogent. "Over the past three months, we have made significant progress in shifting the focus of the company to align with our future vision, and we look forward to presenting additional data on our lead program PLX9486 at CTOS 2020 as we continue to make progress in advancing the program for the treatment of patients suffering from Systemic Mastocytosis and GIST."

Cogent remains on track to advance PLX9486 as a single agent in Advanced Systemic Mastocytosis patients in the first half of next year and Indolent Systemic Mastocytosis patients in the second half of 2021. Additionally, Cogent plans to further develop PLX9486 in combination with sunitinib in GIST patients and plans to initiate an additional clinical study in GIST in the second half of 2021.

An updated corporate presentation, which includes additional preclinical data highlighting the potency of PLX9486 against KIT D816V, is now online at Cogent’s new website, www.cogentbio.com.

PLX9486 + Sunitinib Combination data to be presented in Oral CTOS Abstract

Cogent’s lead precision therapy is PLX9486, a potent and selective precision kinase inhibitor that is designed to target KIT mutations present in Systemic Mastocytosis and GIST. New details of the open-label Phase 1/2 clinical study testing the combination of PLX9486 with sunitinib in 18 adult patients with advanced GIST, will be presented as an oral presentation at the upcoming 2020 CTOS Annual Meeting.

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: Friday, November 20, 2020 from 11:30 a.m. – 12:30 p.m. ET

Presenter: Jonathan Trent, M.D., Ph.D., University of Miami Health System, Sylvester Comprehensive Cancer Center

The oral presentation will contain detailed results from the combination of PLX9486 and sunitinib in patients with advanced GIST. Previously announced topline results demonstrated 11 months median progression free survival with the combination of PLX9486 and sunitinib in heavily treatment-experienced patients with advanced GIST, with two-thirds of patients having previously received three or more lines of therapy. PLX9486 was well-tolerated, and the most commonly occurring treatment emergent adverse events (TEAEs) were predominantly £ Grade 2. PLX9486 has been dosed in more than 50 patients both as a single agent and as part of a combination therapy. Based on the data, Cogent believes the combination of PLX9486 and sunitinib has the potential to address an unmet medical need in patients living with GIST.

Operational Update

Cogent’s current cash balance includes gross proceeds of a $104.4 million private placement concurrent with the Kiq acquisition, as well as $8.1 million from the BOXR sale with potential near-term milestones of up to $3.4 million. This funding provides Cogent with runway into 2023. More details on Cogent’s cash position will be provided in November in the Q3 earnings call.

Cogent has retained senior personnel from Unum who bring critical expertise in clinical science, clinical operations, regulatory affairs, and project management, and the company has continued to grow the team over the past three months with new hires following the completion of the Kiq acquisition.

On October 5, 2020 Invitae Corporation (NYSE: NVTA), a leading genetics company, reported that on October 2, 2020, it completed the transaction to bring ArcherDX, a leading genomics analysis company, into Invitae to create a comprehensive offering that provides testing services for disease risk, therapy optimization and personalized cancer monitoring to enable precision approaches to cancer treatment (Press release, Invitae, OCT 5, 2020, View Source [SID1234568091]).

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Invitae’s (NVTA) mission is to bring comprehensive genetic information into mainstream medical practice to improve the quality of healthcare for billions of people. www.invitae.com (PRNewsFoto/Invitae Corporation)

"With the addition of ArcherDX’s technologies, capabilities and team, Invitae is now well positioned to accelerate the utilization of genetic information throughout a cancer patient’s journey. Starting from risk profiling and diagnostic testing, moving to therapy optimization, monitoring and recurrence surveillance, Invitae can deliver the information needed to enable best-in-class personalized cancer care," said Sean George, Ph.D., co-founder and chief executive officer of Invitae. "Invitae is on a mission to increase access to molecular medicine to all who can benefit, and the addition of the ArcherDX platform builds out an important segment serving the current and future oncology landscape."

In connection with the closing of the acquisition, Jason Myers, Ph.D., has been appointed to Invitae’s Board of Directors, effective October 2, 2020, and will serve as president of oncology.

Transaction Details
Under the terms of the Agreement and Plan of Merger and Plan of Reorganization, Invitae acquired ArcherDX for upfront consideration consisting of 30.0 million shares of Invitae common stock and $325.0 million in cash, subject to certain adjustments. In addition, up to an additional 27.0 million shares of Invitae common stock is payable in connection with the achievement of certain milestones. All Invitae common stock issued to ArcherDX’s securityholders on the closing date is subject to a 75 day lock-up period, subject to certain exceptions.

In connection with the acquisition, Invitae entered into a credit agreement and guaranty with Perceptive Credit Opportunities Holdings III, LP providing for a senior secured term loan facility, and on October 2, 2020, borrowed an aggregate principal amount of $135.0 million under the credit agreement and guaranty. In addition, Invitae issued to Perceptive warrants to purchase 1.0 million shares of Invitae common stock.

In connection with the acquisition, Invitae sold $275.0 million of common stock to certain accredited investors in a private placement.

On October 5, 2020 Trillium Therapeutics Inc. ("Trillium" or the "Company") (NASDAQ/TSX:TRIL), a clinical stage immuno-oncology company developing innovative therapies for the treatment of cancer, reported that it has received Notices of Allowance from the United States Patent and Trademark Office for two patent applications covering the use of SIRPαFc for the treatment of cancer, U.S. Patent Application No. 13/320,629 (the "629 Application", exclusively licensed to Trillium) and U.S. Patent Application No. 15/962,540 (the "540 Application") (Press release, Trillium Therapeutics, OCT 5, 2020, View Source [SID1234568090]). Trillium has two SIRPαFc biologics, TTI-621 and TTI-622, in clinical development. Both therapeutics target CD47, a "don’t eat me" signal that cancer cells use to evade immune destruction.

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The ‘629 method of use Application provides coverage for the use of SIRPαFc biologics to treat all CD47+ cancer cells and tumours, including hematologic and solid cancers. Counterparts of this application have already been granted in Japan, Canada and Australia.

The ‘540 Application has claims that cover TTI-622 composition of matter comprising human SIRPα linked to an IgG4 Fc region, as well as claims covering pharmaceutical compositions that contain TTI-622. A composition of matter patent for TTI-621 (SIRPα linked to an IgG1 Fc) has already been granted in the U.S. (US 9,969,789) and other countries.

"Intellectual property remains a top priority for Trillium, and the allowance of these two important patents further strengthens our position in the SIRPαFc CD47 space," said Jan Skvarka, President and Chief Executive Officer of Trillium. "In addition to our specific composition-of-matter claims covering both of our CD47-targeting agents, we have expanded our coverage to embrace the use of the SIRPαFc class of biologics to treat cancer. We are particularly pleased that the method of use patent also covers treatment of solid tumors."

On October 5, 2020 Arch Oncology, Inc., a clinical-stage immuno-oncology company focused on the discovery and development of anti-CD47 antibody therapies, reported the expansion of AO-176’s clinical development into a Phase 1/2 chemotherapy combination trial for patients with select solid tumors (Press release, Arch Oncology, OCT 5, 2020, View Source;utm_medium=rss&utm_campaign=arch-oncology-advances-anti-cd47-antibody-ao-176-into-chemotherapy-combination-phase-1-2-trial-in-solid-tumors [SID1234568089]). AO-176 is an anti-CD47 antibody with a potential best-in-class profile that works by blocking the "don’t eat me" signal and also by directly killing tumor cells, with preferential binding to tumor versus normal cells.

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"With encouraging evidence of AO-176’s safety and anti-tumor activity as a single agent, we are expanding this trial to further evaluate this therapy in combination with chemotherapy in patients with select solid tumors," said Julie Hambleton, M.D., Interim President and Chief Executive Officer of Arch Oncology. "In the Phase 1 dose-escalation study, we established the recommended dose for Phase 2 development. In addition, we generated important clinical data on the safety and efficacy profile of AO-176 in patients with select solid tumors and we plan to submit these data to a medical meeting next year. With patient dosing underway with AO-176 in combination with chemotherapy, we are making significant progress developing AO-176 broadly for patients with solid tumors as well as hematologic malignancies."

Howard A. "Skip" Burris, III, M.D., President, Clinical Operations, Chief Medical Officer, and Principal Investigator, Sarah Cannon Research Institute, commented, "There is a growing body of preclinical and clinical data for AO-176, demonstrating this therapy’s highly-differentiated clinical profile among anti-CD47 therapeutics in development. As drugs against this target have demonstrated robust clinical activity, we are eager to assess this new combination treatment approach for patients with solid tumors who need better options for their disease."

This open-label, multi-center, dose-escalation Phase 1/2 trial is evaluating the safety, tolerability, pharmacokinetics/pharmacodynamics, and preliminary efficacy of AO-176 in combination with paclitaxel in patients with gastric, endometrial, and platinum-resistant ovarian cancers.

Across the United States, leading cancer treatment clinical trial sites are participating in this trial. For additional information, please visit www.clinicaltrials.gov using the trial identification number NCT03834948.

About AO-176

AO-176 is a humanized anti-CD47 IgG2 antibody with a potential best-in-class profile. AO-176 is highly differentiated, with the potential to improve upon the safety and efficacy profile relative to other agents in this class of innate checkpoint inhibitors. AO-176 works by blocking the "don’t eat me" signal, the standard mechanism of anti-CD47 antibodies. Beyond blocking this signal, AO-176 has additional mechanisms, including directly killing tumor cells and inducing DAMPs (Damage Associated Molecular Patterns), resulting in Immunogenic Cell Death. Importantly, AO-176 binds preferentially to tumor cells, instead of to normal cells, and binds even more potently to tumors in their acidic microenvironment (low pH). Publications and presentations on AO-176 can be found at our Pubs & Posters page.

AO-176 is being evaluated in Phase 1/2 clinical trials for the treatment of patients with select solid tumors and multiple myeloma. In a Phase 1 trial in solid tumors, AO-176 demonstrated encouraging safety and evidence of anti-tumor activity when administered as a single agent. Additional information about these trials may be found at www.clinicaltrials.gov using the trial identification number NCT03834948 (solid tumors) or NCT04445701 (multiple myeloma).

On October 5, 2020 ORPHELIA Pharma, a French biopharmaceutical company dedicated to the development and marketing of pediatric and orphan drugs reported the inclusion of the first-in patient in a clinical study aimed at demonstrating the bioequivalence between Kimozo, the first pediatric formulation of temozolomide under clinical development, and Temodal capsule (Press release, ORPHELIA Pharma, OCT 5, 2020, View Source [SID1234568070]).

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Although not approved in this indication, temozolomide is an essential anticancer medicine recommended for use in treatment protocols of relapsed or refractory neuroblastoma, a condition that affects young children. However, the only oral formulations that are commercially available are in the form of capsules that are poorly adapted to children. Caretakers are thus compelled to open capsules and disperse the content into a soft food prior to administration to the child, which harbors several risks such as the uncertainty with regards to the delivered dose, the exposure of the caregiver to the cytotoxic drug and the instability of the drug in aqueous milieu. To overcome the risks this situation implies, Kimozo, a ready to use oral formulation has been specifically designed to address the needs of the pediatric population.

Kimozo has been developed in collaboration with Gustave Roussy, one of the leading cancer centers in Europe. The current clinical trial conducted by ORPHELIA Pharma aims at demonstrating, first for regulatory purposes, the bioequivalence between Kimozo and the Temodal capsules in adult patients having brain cancers, with additional clinical investigation to come in the pediatric population of interest.

"We have reached an important milestone for Kimozo with the clinical trial approval from competent authorities and the recruitment of a first patient", said Caroline Lemarchand, Chief Pharmaceutical Development Officer of ORPHELIA Pharma "We plan to enroll 30 patients by mid-2021 thanks to the support of the three neuro-oncology teams involved in the study: Professor Ducray of the Hospices Civils de Lyon (coordinating investigator), Professor Chinot of the Timone Hospital in Marseille and Dr Bronnimann of Saint-André Hospital in Bordeaux. "

"We are pleased to contribute to the development of this new pediatric formulation of temozolomide. A liquid form is unambiguously of interest for treating children.", underlines Pr. François Ducray. Hugues Bienaymé, General Manager of ORPHELIA Pharma further comments: "This first administration is a major milestone in the development of Kimozo. We are now preparing the opening of our second clinical trial, which will evaluate Kimozo in pediatric patients, by the end of the year ", he concludes.

About the Bioequivalence Study (NCT04467346)

The clinical trial entitled "Bioequivalence Study between temozolomide oral suspension (Ped-TMZ) and Temodal capsules" (NCT04467346) is an open label phase I study, randomized, crossover, 2-period study in 30 male/female patients with primary CNS malignancies. Patients will receive, under fasting conditions, 200 mg/m² of Temozolomide Oral Suspension (Ped-TMZ, the code name of Kimozo) or Temodal, as single oral administration in 2 different study periods depending on the randomization, with no wash out period between administrations. The primary objective is to evaluate the bioequivalence between Kimozo and Temodal capsules for oral administration. The secondary objectives are to define the pharmacokinetic parameters of Kimozo administration and to assess its buccal safety.

The clinical centers are Hospices Civils de Lyon, Bron, France ; CHU de Bordeaux, Bordeaux, France and Hôpital de la Timone (AP-HM), Marseille, France. The Study sponsor is ORPHELIA Pharma.

About Kimozo

Kimozo (also known as ORP-005 or Ped-TMZ) is a ready-to-use and taste-masked oral suspension of temozolomide that is currently under development to address children needs. Kimozo is an investigational medicinal product not yet approved for marketing anywhere in the world.