On March 30, 2021 Omega Therapeutics, Inc. ("Omega"), a development-stage biotechnology company leveraging its proprietary epigenomic programming platform to biologically engineer a new class of programmable epigenetic medicines, reported the closing of an upsized Series C financing of $126 million (Press release, Omega Therapeutics, MAR 30, 2021, View Source [SID1234577346]). Joining Flagship Pioneering, Omega’s institutional founder and principal backer, are leading life science investors including Invus, Fidelity Management & Research Company, funds and accounts managed by BlackRock, Cowen, Point72, Logos Capital, Mirae Asset Capital and other undisclosed new and returning institutional investors. With this financing, Omega has raised over $210 million since its founding in 2017.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Proceeds from the financing will be used to support the advancement of Omega’s lead epigenomic controller candidate, OTX-2002, and to advance the next wave of novel pipeline therapeutics that it expects to be generated by the company’s proprietary Omega Epigenomic ProgrammingTM platform, with an initial focus in oncology, regenerative medicine, inflammation, autoimmune, metabolic and rare genetic diseases. The proceeds will also be used to continue developing the Omega Epigenomic Programming platform and build a manufacturing footprint.

"We are grateful to our new and existing investors for the commitment to our bold vision of creating the industry’s first fully programmable epigenetic medicines," said Mahesh Karande, President and Chief Executive Officer of Omega Therapeutics. "This financing enables us to advance OTX-2002 through the required IND-enabling studies with the goal of filing an IND and entering the clinic thereafter. It also allows us to continue unlocking the potential of our Omega Epigenomic Programming platform where we expect to be unveiling several additional drug candidates addressing a wide range of high unmet need diseases during 2021."

"Omega is at a pivotal stage of its development, as it prepares to debut several new pipeline assets and advance each toward clinical trials. We look forward to partnering with our new and existing investors to build out a robust pipeline, bring exciting new medicines to patients in need, and significantly grow value for all of our stakeholders," commented Roger Sawhney, Chief Financial Officer of Omega Therapeutics.

"In a short three years since its founding, Omega has made significant progress by leveraging its deep expertise of nature’s universal operating system for gene control to power its Omega Epigenomic Programming platform," said Noubar Afeyan, Ph.D., Co-founder and Chairman of the Board for Omega Therapeutics and Chief Executive Officer of Flagship Pioneering. "We welcome this exceptional group of new investors as Omega continues to pioneer and works to establish a new class of transformative programmable medicines."

In January 2021, Omega unveiled OTX-2002, its first epigenomic controller development candidate and the industry’s first programmable epigenetic medicine. OTX-2002 is engineered to specifically control c-myc (MYC) oncogene expression. In preclinical models of hepatocellular carcinoma (HCC), OTX-2002 potently downregulated MYC expression, a result that has historically eluded many prior attempts and therapeutic approaches. The Company is currently advancing OTX-2002 into Investigational New Drug (IND)-enabling studies. Omega plans to nominate additional development candidates in 2021, with an initial focus on regenerative medicine, inflammatory diseases, acute respiratory distress syndrome (ARDS) associated with COVID-19, alopecia, neutrophilic dermatoses, non-small cell lung cancer (NSCLC) and an additional oncogene target.

About Omega Epigenomic Programming Platform and Omega Epigenomic Controllers

Omega Therapeutics leverages its pioneering Epigenomic Programming platform to identify novel targets, develop first-in-class programmable epigenetic medicines, and enable rational drug development and manufacturing. Omega examines Insulated Genomic Domains (IGDs), the three-dimensional architecture of the human genome and its accompanying regulators, and has identified and classified thousands of genomic "zip codes" across the ~15,000 IGDs as new drug targets. Omega’s new class of medicine, called Omega Epigenomic Controllers, modulate IGDs using therapeutics that can be programmed to precisely up or down regulate single or multi-gene expression with controlled durability. These epigenomic controllers intervene at the pre-transcriptional level and they function without altering the native human genetic code or nucleic acid sequences. Using a rational and robust target identification and validation process, enhanced by a strong computational and data driven foundation, Omega is able to efficiently design and optimize potential epigenomic controllers from its platform. This entirely new and breakthrough approach allows the Company’s product candidates to also drug previously ‘undruggable’ targets across a broad range of diseases.

On March 30, 2021 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported financial results for the fourth quarter and year ended December 31, 2020 (Press release, Crinetics Pharmaceuticals, MAR 30, 2021, View Source [SID1234577362]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We are entering 2021 poised to achieve several key milestones that we believe will consolidate our position as a leader in the design and development of novel small molecule drugs for endocrine diseases," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Our positive Phase 2 acromegaly data demonstrate the potential of orally administered paltusotine to replace injectable somatostatin receptor ligand (SRL) depots as the standard-of-care. With input from a recently completed FDA meeting, we have designed the paltusotine Phase 3 program to support its approval for all acromegaly patients who require pharmacotherapy, including both untreated patients and those switching from injected standard of care."

Dr. Struthers continued, "Paltusotine’s success and progress serve as a blueprint for our new therapeutic candidates in endocrinology. We plan to follow a similar path with CRN04777 and CRN04894, both of which recently entered Phase 1 proof-of-concept trials in healthy volunteers. Much like paltusotine’s Phase 1 program, we’ve designed these trials to evaluate safety, drug-like pharmacokinetics, and corresponding pharmacodynamic effects on well-validated hormonal biomarkers for their respective indications. We look forward to reporting results from the Phase 1 trials later this year, and to the continued progression of our clinical and preclinical pipelines."

Full Year 2020 and Recent Highlights

Advancing paltusotine to a Phase 3 program for acromegaly following the completion of an FDA meeting. In 1Q 2021, Crinetics met with the U.S. Food and Drug Administration (FDA) to discuss its Phase 2 acromegaly data and plans for a Phase 3 program. Based on these interactions and those with other regulators, the Company plans to initiate two Phase 3 studies in 2021. The first of these studies, entitled PATHFNDR-1, is a double-blind, placebo-controlled, nine-month clinical trial evaluating the safety and efficacy of paltusotine in acromegaly patients who are biochemically controlled (IGF-1 ≤ 1.0 × upper limit of normal [ULN]) and who are on stable doses of SRL monotherapy (octreotide LAR or lanreotide depot). The second study, PATHFNDR-2, is a double-blind, placebo-controlled, twelve-week trial in acromegaly patients with elevated IGF-1 levels who are medication naïve or who are not being treated with pharmacotherapy (untreated patients). The primary endpoint for both studies will be the proportion of patients achieving biochemical control comparted to placebo. If successful, Crinetics believes these trials could support registration of paltusotine in the United States and Europe for all acromegaly patients who require pharmacotherapy, including untreated patients and those switching from standard of care. The Company expects to initiate PATHFNDR-1 in 2Q 2021 and PATHFNDR-2 in 2H 2021.

Showcased broad clinical-stage pipeline at ENDO 2021. In March 2021, Crinetics showcased the breadth of its pipeline with presentations on its three clinical programs at the Endocrine Society’s annual ENDO 2021 congress. Posters on the company’s acromegaly program included a summary of the previously announced ACROBAT Edge Phase 2 results, as well as details of a new tablet formulation of paltusotine. Presentations related to the company’s earlier stage clinical programs included a poster with preclinical data supporting the development of CRN04777 as a treatment for congenital

hyperinsulinism (congenital HI) and a live oral presentation with preclinical evidence supporting the further evaluation of CRN04894 in Cushing’s disease and congenital adrenal hyperplasia (CAH).

Advanced ACTH antagonist CRN04894 into a Phase 1 study designed to provide clinical proof of concept. In February 2021, Crinetics initiated a Phase 1 study of CRN04894, an investigational, oral, nonpeptide adrenocorticotropic hormone (ACTH) antagonist being developed for the treatment of diseases associated with excess ACTH such as Cushing’s disease and CAH. In addition to evaluating the safety and tolerability of CRN04894 in healthy volunteers, the placebo-controlled study aims to provide clinical proof-of-concept data by measuring the effect of CRN04894 on the suppression of ACTH-stimulated adrenal secretion of cortisol and related steroids. These endocrine biomarkers are used as key endpoints in patient studies and reflect the ability of CRN04894 to block ACTH-stimulated adrenal function. This Phase 1 trial is expected to provide important information for dose selection and be predictive of efficacy in Cushing’s disease and CAH trials. Crinetics expects to report preliminary safety, pharmacokinetic, and endocrine biomarker data from the single ascending dose portion of the trial in 1H 2021.

Advanced SST5 agonist CRN04777 into a Phase 1 study designed to provide clinical proof of concept. In February 2021, Crinetics initiated a Phase 1 study of CRN04777, an investigational, oral, nonpeptide somatostatin receptor type 5 (SST5) agonist. CRN04777 is being developed as a treatment for congenital HI, a rare genetic disease in which excess insulin secretion causes life-threatening hypoglycemia (low blood glucose). In addition to evaluating the safety and tolerability of CRN04777 in healthy volunteers, the placebo-controlled study aims to provide clinical proof of concept data by measuring the ability of CRN04777 to inhibit glucose- and sulfonylurea-induced insulin secretion and observe the corresponding effects on blood glucose levels. These endocrine biomarkers are indicative of the ability of CRN04777 to prevent hypoglycemia and are expected to provide information for dose selection and be predictive of efficacy in patients with hyperinsulinism. Crinetics expects to report preliminary safety and pharmacological effect data from the single ascending dose portion of the trial in mid-2021.

Reported positive topline results for the ACROBAT Phase 2 program of paltusotine in acromegaly patients. In October 2020, Crinetics reported positive top-line data from its Phase 2 program evaluating paltusotine for the treatment of acromegaly. Data showed that levels of insulin-like growth factor 1 (IGF-1), the accepted biomarker of disease control in acromegaly, were maintained in patients switching from standard-of-care injected SRL depots to once-daily oral paltusotine.

Received Rare Pediatric Disease Designation for CRN04777 for the treatment of congenital HI. In September 2020, the FDA granted CRN04777 Rare Pediatric Disease (RPD) Designation for the treatment of congenital HI. Based on this designation, Crinetics may be eligible to receive a priority review voucher (PRV) following approval of CRN04777 if the marketing application submitted for the candidate satisfies certain additional conditions. If issued, a PRV may be redeemed for priority review of a subsequent marketing application for a different product candidate, or the PRV could be sold or transferred to another sponsor.

Received Orphan Drug Designation for paltusotine for the treatment of acromegaly. In July 2020, the FDA granted paltusotine Orphan Drug Designation for the treatment of acromegaly, qualifying Crinetics for certain development incentives such as exemption from FDA prescription drug user fees, financial incentives for qualified clinical development, and seven years of market exclusivity in the U.S. upon FDA approval.

Strengthened company leadership with appointments to clinical team and Board of Directors. Throughout 2020, Crinetics continued to reinforce its in-house expertise in endocrinology, rare disease drug development, and clinical trial management through the appointments of Drs. Alessandra Casagrande, Peter Trainer, and Hjalmar Lagast to the clinical team and the appointment of Dr. Camille L. Bedrosian to the Board of Directors.

Fourth Quarter and Full Year 2020 Financial Results

Research and development expenses were $16.8 million and $57.0 million for the three months and full year ended December 31, 2020, respectively, compared to $12.1 million and $41.5 million for the same periods in 2019. The increases were primarily attributable to clinical development and manufacturing activities for paltusotine as well as the company’s preclinical programs.

General and administrative expenses were $5.0 million and $18.0 million for the three months and full year ended December 31, 2020, compared to $3.4 million and $13.5 million for the same periods in 2019. The increases were primarily due to costs to operate as a public company, as well as personnel costs to support the company’s growth.

Net loss for the three months ended December 31, 2020 was $21.6 million, compared to a net loss of $14.5 million for the same period in 2019. For the year ended December 31, 2020, the company’s net loss was $73.8 million compared to a net loss of $50.4 million for the year ended December 31, 2019.

Unrestricted cash, cash equivalents and investments totaled $170.9 million as of December 31, 2020, compared to $186.8 million as of September 30, 2020 and $118.4 million as of December 31, 2019.

As of February 26, 2021, the company had 33,028,876 common shares outstanding.

Webcast and Conference Call Information

Crinetics will hold a live webcast and conference call today, March 30, 2021 at 4:30 p.m. Eastern Time to discuss its 2021 clinical plans and 2020 financial results. To access the webcast, please visit this link to the event. To participate by phone, please dial 877-407-0789 (domestic) or 201-689-8562 (international) and refer to conference ID 13717687. Following the live event, the archived webcast will be available for 90 days.

On March 30, 2021 Lantheus Holdings, Inc. (NASDAQ: LNTH) (Lantheus), an established leader and fully integrated provider of innovative imaging diagnostics, targeted therapeutics and artificial intelligence solutions to Find, Fight and Follow serious medical conditions, reported it has acquired the exclusive, worldwide rights to develop, manufacture and commercialize NTI-1309, an innovative PET oncology imaging agent from Noria Therapeutics, Inc (Press release, Lantheus Medical Imaging, MAR 30, 2021, View Source [SID1234577378]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

NTI-1309 targets fibroblast activation protein (FAP), a target with potential broad imaging applicability and targeting implications in oncology. FAP is overexpressed in the tumor microenvironment, specifically in tumor-associated fibroblasts, which are believed to modulate tumor progression and immune response. Given its expression in tumors coupled with low expression in normal tissue, FAP has the potential to become an important biomarker for precision medicine in cancer. Already a focus of significant research by academics and the pharmaceutical industry, a FAP biomarker has potential to address unmet medical needs and to impact the clinical management of stroma-dense tumors, such as breast, colon, lung and pancreatic cancer.

Under terms of the agreement, Noria will drive the early clinical development of NTI-1309, leveraging its experience with early-stage imaging development. Upon completion of the Phase 1 study, NTI-1309 will be integrated into Lantheus’ portfolio of imaging biomarkers and included in the offering to academic centers and pharmaceutical companies for use in oncology drug development programs. Simultaneously, Lantheus will assess options for bringing this important biomarker to market through Lantheus-sponsored trials.

"Lantheus is committed to advancing innovative imaging biomarker solutions to find, fight and follow cancer," said Etienne Montagut, Senior Vice President of Corporate Development at Lantheus. "We believe FAP is a promising target for cancer imaging and has broad potential to inform diagnosis and staging, to guide patient selection for therapy, and to monitor response to treatment across multiple tumor types. This partnership with Noria enables us to progress this development program in a timely fashion, leverage our established leadership in imaging and state of the art artificial intelligence, and position us to monetize this biomarker offering, which has the potential to unlock deep, data-driven insights to inform R&D and clinical decision-making."

"The Noria team is excited to partner with a proven leader in imaging diagnostics with the experience and resources to advance this development program. Noria is pleased to have been entrusted by Lantheus to continue the development of NTI-1309, leveraging our platform and very experienced team," said Allan M. Green, MD, PhD, JD, Co- Founder and CEO of Noria Therapeutics, Inc.

Terms of the transaction include an upfront license and development fee to Noria, certain development and regulatory milestones, and royalties if NTI-1309 is commercialized.

On March 30, 2021 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX-V:BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company specializing in targeted immunotherapies for advanced breast cancer, reported that it has been selected to present at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2021, a virtual meeting, held over two weeks (Week 1: April 10-15; Week 2: May 17-21) (Press release, BriaCell Therapeutics, MAR 30, 2021, View Source [SID1234577404]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

BriaCell will be presenting the results of its analysis of clinical and pathological data of its lead candidate, Bria-IMT, a novel immunotherapy for advanced breast cancer, as monotherapy and a Phase I/IIa clinical study of Bria-IMT in combination with immune checkpoint inhibitors, including pembrolizumab (KEYTRUDA; manufactured by Merck & Co., Inc.) and, more recently, Incyte’s INCMGA00012 (under a corporate collaboration with Incyte Corporation). The patient data belong to previously-disclosed patients (i.e., no incremental numbers enrolled).

Details include:

Abstract Control Number: 4932
Title: Predictors of response to a modified whole tumor cell immunotherapy in patients with advanced breast cancer from two phase I/IIa trials
Session Type: E-Poster Session
Session Category: Phase I Clinical Trials
Session Title: Phase I Clinical Trials
Permanent Abstract Number: CT102
Session Date and Time: Saturday, April 10 at 8:30 a.m. ET. E-posters will remain available for viewing through Monday, June 21.

Following the presentation, a copy of the poster will be posted on View Source

Additionally, the Board of Directors of the Company (the "Board") unanimously approved the grant of an aggregate of 617,300 incentive stock options ("Stock Options") to directors, officers, employees and consultants under the Company’s stock option plan ("Stock Option Grant"). Of the Stock Option Grant, 560,000 Stock Options will be granted to Insiders, as such term is defined in the Securities Act (British Columbia). The Stock Options are exercisable at US$4.24 per common share in the capital of the Company ("Common Share"), will vest immediately, and will expire in 5 years from the date of issuance. Following the Stock Option Grant, the Company has 635,352 Stock Options outstanding.

The Stock Option Grant to certain Insiders constitutes a "related party transaction" within the meaning of Exchange Policy 5.9, which incorporates Multilateral Instrument 61-101 ("MI 61-101"). The Company is exempt from shareholder approval in accordance with section 5.7.1(a) of MI 61-101. The Stock Option Grant will not result in a new Control Person.

On March 30, 2021 Oncotelic Therapeutics, Inc. ("Oncotelic" or the "Company") (OTCQB:OTLC) (f/k/a Mateon Therapeutics, Inc.) reported that the Financial Industry Regulatory Authority ("FINRA") has confirmed the change in the Company’s name and approved the stock symbol trading on the OTC Markets (Press release, Mateon Therapeutics, MAR 30, 2021, View Source [SID1234577347]). Effective today, March 30, 2021, the ticker symbol is changed from "MATN" to "OTLC".

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On August 14, 2020, the Company filed a Current Report on Form 8-K, in which the Company reported a number of corporate actions approved by the shareholders of the Company on August 10, 2020, including the name change and symbol change. The Company formally changed its name to Oncotelic Therapeutics, Inc. with the State of Delaware in November 2020, as disclosed in its Quarterly Report on Form 10-Q for the quarter ended September 30, 2020, filed with the Securities and Exchange Commission on November 16, 2020. A notice of corporate action was filed with FINRA, requesting confirmation of its name change and approval for the new ticker symbol. On March 29, 2021, the Company received FINRA’s approval on its notice of corporate action confirming the new company name and the change in the Company’s ticker symbol, effective March 30, 2021.