On November 14, 2022 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), reported financial results for the third quarter ended September 30, 2022 (Press release, Crinetics Pharmaceuticals, NOV 14, 2022, View Source [SID1234623976]).

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"This has been yet another successful quarter of executing our strategy to build a leading endocrinology company by advancing our clinical pipeline of internally discovered drug candidates and preparing to add to that pipeline with our highly productive drug discovery efforts. Progress includes completion of enrollment in the Phase 3 PATHFNDR-1 trial and reporting Phase 2 open-label extension data demonstrating maintenance of IGF-1 as well as a strong preference among acromegaly patients for oral paltusotine over the injected standard-of-care," said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. "We remain on track for two Phase 3 readouts in acromegaly next year. Beyond acromegaly, we maintained strong momentum with our efforts to demonstrate paltusotine’s utility in carcinoid syndrome with a Phase 2 readout expected next year. We also continue to advance CRN04777 and CRN04894 toward patient trials based on the positive proof-of-pharmacology data we reported earlier this year."

THIRD QUARTER 2022 AND RECENT HIGHLIGHTS
Completed enrollment in Phase 3 PATHFNDR-1 study. PATHFNDR-1 is one of two ongoing, placebo-controlled Phase 3 clinical trials evaluating the safety and efficacy of paltusotine in acromegaly patients. Topline data from PATHFNDR-1 are expected in the third quarter of 2023 and topline data from paltusotine’s second Phase 3 trial, PATHFNDR-2, are expected in the fourth quarter of 2023. If successful, Crinetics plans to submit data from the PATHFNDR trials to regulatory authorities in support of applications seeking approval for the broad use of paltusotine for all acromegaly patients who require pharmacotherapy, including untreated patients and those switching from other therapies.
Reported new long-term safety and efficacy data from ACROBAT Advance open-label extension trial of paltusotine in acromegaly. Results showed that once-daily oral paltusotine lowered and maintained IGF-1 at levels comparable to prior injected somatostatin receptor ligand (SRL) therapy for up to 103 weeks. In addition, paltusotine was well tolerated in the study and 89% of study participants surveyed selected paltusotine as their preferred treatment option over injected SRLs.
Received a UK Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Passport for CRN04777 for the treatment of congenital hyperinsulinism. The Innovation Passport enables Crinetics to access the Innovative Licensing and Access Pathway (ILAP). The ILAP aims to reduce the time to market for designated medicines by enabling enhanced coordination between sponsors and MHRA leading up to Marketing Authorization Application (MAA) submissions and by providing the opportunity for accelerated MAA reviews.
Reviewed analyses from preclinical and Phase 1 clinical studies of CRN04894 in an oral presentation at the 10th International Congress of Neuroendocrinology. CRN04894 is an adrenocorticotropic hormone (ACTH) antagonist being developed as a treatment for Cushing’s disease, congenital adrenal hyperplasia (CAH) and other conditions of ACTH excess. The presentation was delivered by the Crinetics’ vice president of clinical endocrinology, Dr. Peter J. Trainer, and featured the results of a Phase 1 healthy volunteer study demonstrating pharmacologic proof-of-concept for CRN04894. These results showed dose-dependent increases in CRN04894 plasma concentrations as well as reductions of both basal cortisol and elevated cortisol following an ACTH challenge.
Strengthened leadership team with appointment of Dana Pizzuti, M.D. as chief development officer. Dr. Pizzuti is a board-certified physician with more than 30 years of pharmaceutical industry experience in clinical development, pharmacovigilance, medical and regulatory affairs.
THIRD QUARTER 2022 FINANCIAL RESULTS
Research and development expenses were $32.0 million for the three months ended September 30, 2022, compared to $21.6 million for the same period in 2021. The increase was primarily attributable to an increase in spending on manufacturing and development activities of $5.2 million associated with our clinical and nonclinical activities for paltusotine, CRN04777, CRN04894 and our other clinical and preclinical programs and an increase in personnel costs of $4.2 million.
General and administrative expenses were $11.9 million for the three months ended September 30, 2022, compared to $6.2 million for the same period in 2021. The increase was primarily attributable to an increase in personnel costs of $3.0 million.
Net loss for the three months ended September 30, 2022, was $41.9 million, compared to a net loss of $27.9 million for the same period in 2021.
Revenues were $0.5 million for the three months ended September 30, 2022, consisting of license revenue recognized from the license agreement entered into with Sanwa Kagaku Kenkyusho Co., Ltd. in February 2022.
Unrestricted cash, cash equivalents and investments totaled $368.4 million as of September 30, 2022, compared to $333.7 million as of December 31, 2021.
The company had 53,810,476 common shares outstanding as of November 9, 2022.

On November 14, 2022 Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported that financial results for the third quarter ended September 30, 2022 (Press release, Compugen, NOV 14, 2022, View Source [SID1234623975]).

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Corporate Update
"We continue to execute and delivered encouraging clinical data in MSS-CRC patients at the recent annual SITC (Free SITC Whitepaper) conference," said Anat Cohen-Dayag, Ph.D., President, and Chief Executive Officer of Compugen. "We were excited to show an overall response rate of 12% in MSS-CRC patients with liver metastases, a patient population in whom responses to immunotherapy treatment has been rare or non-existent. The two partial responses reported were supported by potent immune activation in the tumor microenvironment, indicative of a COM701 mediated mechanism of action. The drug combination was well tolerated with no serious adverse events assessed by the investigator as related to study drugs. Based on the totality of our data supporting the DNAM-1 axis hypothesis, we plan to move ahead with the clinical evaluation of COM701 in triple combination with an anti-PD-1 and our potential best-in-class anti-TIGIT, COM902, in MSS-CRC patients."

Dr. Cohen-Dayag continued, "We are excited to be presenting new encouraging clinical data from the fully enrolled platinum resistant ovarian cancer cohorts treated with COM701 in combination with nivolumab with or without BMS-986207 at the upcoming ESMO (Free ESMO Whitepaper)-IO conference. We believe COM701 combinations warrant further investigation in this indication. This opens the door for us to evaluate our drugs in a more favorable competitive landscape compared to NSCLC. For this reason, we have decided to pursue this indication and we are evaluating the various options for the planned NSCLC studies."

Dr. Cohen-Dayag concluded, "I am excited about the progress we have made and look forward to our continued focus on execution and delivery of meaningful clinical data. We have a solid balance sheet, with cash balances of $88 million which we expect to support our operations at least through the end of 2024."

Financial Results
As of September 30, 2022, cash, cash equivalents, short-term bank deposits and restricted cash totaled approximately $88 million, compared with approximately $118 million as of December 31, 2021. The Company expects its existing cash and cash related balances to fund its operating plans at least through the end of 2024. Compugen does not have any debt.

R&D expenses for the third quarter ended September 30, 2022, were approximately $9.3 million, compared to approximately $8.7 million for the comparable period in 2021.

General and administrative expenses for the third quarter ended September 30, 2022, were approximately $2.6 million compared with approximately $2.8 million for the comparable period in 2021.

Net loss for the third quarter ended September 30, 2022, was approximately $11.7 million, or $0.14 per basic and diluted share, compared with a net loss of approximately $6.2 million, or $0.07 per basic and diluted share, in the comparable period of 2021.

Full financial tables are included below

Conference call and webcast information
The Company will hold a conference call today, November 14, 2022, at 8:30 AM ET to review its third quarter 2022 results. To access the live conference call by telephone, please dial 1-866-744-5399 from the U.S., or +972-3-918-0644 internationally. The call will be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.

On November 14, 2022 Chemomab Therapeutics, Ltd. (Nasdaq: CMMB) (Chemomab), a clinical-stage biotechnology company focused on the discovery and development of innovative therapeutics for fibrotic and inflammatory diseases with high unmet need, reported the appointment of Matthew Frankel, MD, MBA as Chief Medical Officer (CMO) and Vice President of Drug Development (Press release, Chemomab, NOV 14, 2022, View Source,-MD,-MBA-as-Chief-Medical-Officer [SID1234623974]).

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"We believe Dr. Frankel’s wealth of experience across all aspects of clinical development and medical affairs, along with his outstanding performance in helping to bring biologic and small molecule drugs to market for both rare and chronic diseases, will be invaluable as we advance the CM-101 clinical program," said Dale Pfost, PhD, Chief Executive Officer of Chemomab. "We have greatly benefitted from the singular talents of interim CMO Dave Weiner, who will be staying on as a Senior Advisor, and are fortunate now to have Matt Frankel, with his exceptional breadth of experience and track record of success, joining as our full-time CMO."

Dr. Frankel most recently served as Vice President, Clinical Development and Medical Affairs, Specialty Pharma at Boehringer Ingelheim, where he led functional teams developing new drugs for oncology, immunology, pulmonary, and central nervous system diseases. Previously Dr. Frankel was Vice President & Head, Immunology and Dermatology Medical Unit at Novartis, where he oversaw medical affairs activities and late phase clinical development for Cosentyx, Ilaris, and Zortress. At the Sandoz unit of Novartis, Dr. Frankel successfully built and led the medical affairs organization supporting the biopharmaceutical, biosimilar and generics businesses for launches including Kerydin, Glatopa and Zarxio.

"Chemomab’s CM-101 is an intriguing investigational drug, with its novel CCL24 target, dual mechanism of action, breadth of application and disease-modifying potential in serious progressive diseases with few effective treatment options," said Dr. Frankel. "Chemomab scientists have assembled an impressive body of preclinical and early clinical evidence supporting the utility of their approach, and I welcome the opportunity to join this talented team as we move to rapidly expand and accelerate our CM-101 clinical programs."

Earlier in his career, Dr. Frankel held clinical development leadership roles across geographies and therapeutic disease areas at Reata, Fibrogen, Abbott Labs, and Schering Plough. Dr. Frankel received a BA degree from Vassar College, an MD degree from the University of California School of Medicine, Los Angeles and an MBA from the J. L. Kellogg Graduate School of Management at Northwestern University. He is board certified in internal medicine.

On November 14, 2022 Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage, precision-oncology biopharmaceutical company, reported its financial results for the third quarter ended September 30, 2022 (Press release, Calithera Biosciences, NOV 14, 2022, View Source,months%20ended%20September%2030%2C%202022. [SID1234623973]).

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"Earlier in the fourth quarter, we were very pleased to announce that we received FDA Fast Track designation for sapanisertib. This designation facilitates more frequent communication with the Agency, as well as a number of other benefits that could support our efforts to bring sapanisertib to patients in this area of high unmet need more quickly," said Susan Molineaux, PhD, president and chief executive officer of Calithera. "Today we also share that we have experienced site activation delays on both our sapanisertib and mivavotinib trials, leading to slower than anticipated enrollment for both these programs. We expect initial data from these studies will not be available until mid-2023."

Third Quarter 2022 and Other Recent Highlights

Received FDA Fast Track designation for sapanisertib (dual mTORC 1/2 inhibitor). In October, Calithera announced that sapanisertib has been granted Fast Track designation by the FDA for the treatment of adult patients with unresectable or metastatic squamous non-small cell lung cancer (sqNSCLC) whose tumors harbor the NRF2 mutation. Fast Track designation, which is designed to facilitate the development and expedite the review of therapeutic candidates with the potential to treat a serious or life-threatening condition where there is a major unmet medical need, provides a number of potential benefits including increased communication with the FDA, the ability to submit a marketing application on a rolling basis and the possibility of priority review.
Began enrolling patients in Phase 2 trial evaluating sapanisertib in sqNSCLC. In July 2022, the Company began enrolling patients in its phase 2 clinical trial (NCT05275673) of the dual mTORC 1/2 inhibitor sapanisertib (CB-228) in patients with relapsed/refractory NRF2 (NFE2L2)-mutated sqNSCLC. The study is designed to confirm the selective activity of sapanisertib in NRF2-mutated tumors compared to wild-type tumors, and to refine dose in this biomarker-defined population. The primary endpoints of the study are investigator-assessed overall response rate (ORR) per RECIST v1.1, and safety. Calithera presented a trial-in-progress poster detailing the study design at the North American Conference on Lung Cancer in September 2022.

Continued patient enrollment activities in Phase 2 trial evaluating mivavotinib (SYK inhibitor) in r/r non-GCB DLBCL. In June 2022, the Company began enrolling patients in its multicenter Phase 2 clinical trial (NCT05319028) evaluating mivavotinib (CB-659) in patients with relapsed/refractory non-germinal center B-cell like (non-GCB) diffuse large B-cell lymphoma (DLBCL). The main objectives of the study are to confirm previously observed single-agent activity in non-GCB DLBCL patients, evaluate activity according to MYD88/CD79b mutation status and refine dose/schedule in this patient population. The primary endpoints of the study are overall response rate (as assessed by an independent radiology review committee) and safety. Details of the Phase 2 study design were presented in a trial-in-progress poster at the Pan Pacific Lymphoma Conference in July.
Continued to advance VPS4 program through lead optimization. Calithera continued to advance multiple vacuolar protein sorting-associated protein 4A (VPS4A) and VPS4B inhibitors through lead optimization and plans to share updates on this program by the end of 2022.
Selected Third Quarter 2022 Financial Results

Cash and cash equivalents totaled $34.1 million at September 30, 2022. Based on its current operating plan, the Company expects it has sufficient cash to fund its operations into the second quarter of 2023. The Company is currently evaluating all options for its programs, including strategic collaboration or licensing agreements and actively considering the sale of certain programs, in order to extend its cash runway.

Research and development expenses for the third quarter 2022 were $6.5 million, compared to $11.6 million in the same period prior year. The decrease of $5.1 million was primarily due to decreases in the telaglenastat and CB-280 programs and investments in early stage research, partially offset by increases in the sapanisertib and mivavotinib programs.

General and administrative expenses for the third quarter 2022 were $3.0 million, compared to $6.3 million in the same period prior year. The decrease of $3.3 million was primarily due to decreased personnel-related costs and legal expenses.

Net loss was $9.8 million for the three months ended September 30, 2022.

Conference Call Information

Calithera will host an update conference call today, Monday, November 14, at 2:00 p.m. Pacific Time/5:00 p.m. Eastern Time. To register for dial-in access to the call, please use this link. To access the live audio webcast or the subsequent archived recording, visit the Investors section of the Calithera website at www.calithera.com. The webcast will be recorded and available for replay on Calithera’s website for 30 days.

On November 14, 2022 Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) and Metagenomi reported that the companies have entered a collaboration that will leverage Ionis’ extensive expertise in RNA-targeted therapeutics and Metagenomi’s versatile next-generation gene editing systems to pursue a mix of validated and novel genetic targets that have potential to expand therapeutic options for patients (Press release, Ionis Pharmaceuticals, NOV 14, 2022, View Source [SID1234623972]). The companies will jointly conduct research aimed initially at delivering investigational medicines for up to four genetic targets. Ionis has the right to add four more targets upon achievement of pre-determined development milestones.

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"Ionis was founded over 30 years ago to discover and develop novel, highly personalized medicines using our powerful RNA-targeting technology platform. This partnership with Metagenomi supports Ionis’ strategic objective to advance our technology and expand our capabilities to deliver precision genetic medicines. Together, we can broaden the application of gene editing by leveraging Ionis’ vast experience in nucleic acid therapeutics to optimize and extend the reach of gene editing for liver targets and to new tissues," said Ionis Chief Executive Officer Brett P. Monia, Ph.D. "This alliance brings our two companies closer to delivering next-generation therapies with the potential to revolutionize the treatment of diseases."

"Gene editing has the potential to transform chronic therapies into potentially curative treatments for patients who currently have limited options," said Brian C. Thomas, Ph.D., chief executive officer and founder of Metagenomi. "This collaboration is a strategic step to combine Metagenomi’s leading gene editing toolbox of diverse, specific and highly efficient nucleases, with Ionis’ pioneering expertise of RNA-targeted therapeutics, to catalyze the next wave of in vivo gene editing applications. We continue to follow our strategy of partnering with the most experienced teams in the genetic medicines field that will allow us to transform the lives of patients."

Under the terms of the agreement, Ionis will pay $80 million upfront to Metagenomi plus the potential for future milestone payments and royalties. Wells Fargo Securities acted as financial advisor to Ionis on the transaction.

Webcast

Ionis will conduct a webcast on Nov. 14 at 8 a.m. Eastern Time to discuss this announcement and related activities. Interested parties may access the webcast here. A webcast replay will be available for a limited time at the same address.

CRISPR Gene Editing

CRISPR (clustered regularly interspaced short palindromic repeats) gene editing is a technology that modifies the sequence of DNA in cells utilizing a specific RNA-guided nuclease (Cas enzyme). Gene editing enzymes act as molecular word processors to correct the genetic code at the precise spot where it is malfunctioning.