On May 10, 2022 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported financial results for the first quarter ended March 31, 2022 (Press release, Cogent Biosciences, MAY 10, 2022, View Source [SID1234614096]).

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"We continue to make significant progress towards our strategic priorities for 2022," said Andrew Robbins, President and CEO of Cogent Biosciences. "We look forward to presenting initial clinical data from our APEX trial of bezuclastinib in advanced systemic mastocytosis patients at the annual EHA (Free EHA Whitepaper) meeting this June. This presentation will include patient data from each dose cohort of bezuclastinib, including levels of serum tryptase, a validated biomarker of mast cell activity. We also continue to enroll patients in the SUMMIT and PEAK trials with bezuclastinib. In addition, our research team has rapidly advanced our pipeline with the addition of our FGFR2 and ErbB2 programs, each with clear differentiation from other programs in development and best-in-class potential. With these important advances, we believe we are well positioned to build on our momentum to bring important therapies to patients with genetically defined diseases."

Upcoming Milestones and Recent Business Highlights

On track to report initial clinical data from APEX, a global, multicenter, Phase 2 clinical trial of bezuclastinib, a potent, highly selective, minimally brain-penetrant KIT mutant inhibitor in patients with Advanced Systemic Mastocytosis (AdvSM) in the second quarter of 2022.
Data to be presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Annual Congress will include safety and tolerability from patients across each dose cohort, as well as levels of serum tryptase, a validated biomarker of mast cell activity. The hybrid conference will take place in Vienna, Austria from June 9-12, 2022.

Presented early data and outlined Cogent’s strategy to create best-in-class small molecules from the company’s growing pipeline of novel, targeted therapy programs at the American Association of Cancer Research (AACR) (Free AACR Whitepaper) annual meeting and Cogent R&D investor event.
Advancing a potent, selective FGFR2 inhibitor toward candidate selection with a product profile that includes best-in-class selectivity and potency against all known FGFR2 primary driver and secondary resistance mutations. On track for first internally developed Investigational New Drug application (IND) in the second half of 2023.
Announced development of an ErbB2 mutant selective inhibitor for patients with mutations that are not well addressed by therapies currently approved or in clinical development.

Shared additional nonclinical data demonstrating bezuclastinib’s potential as a best-in-class KIT mutant inhibitor during the 2022 AACR (Free AACR Whitepaper) annual meeting.
Presentations demonstrated bezuclastinib has a unique profile as a potent, mutant KIT inhibitor (including KIT D816V) with minimal brain penetration and no activity against related kinases including PDGFR, FLT3 and CSF1R, which have been linked to off-target toxicities including edema and pleural effusions.

Actively enrolling patients in SUMMIT, a randomized, double-blind, placebo-controlled, global, multicenter, Phase 2 clinical trial with bezuclastinib for nonadvanced systemic mastocytosis (NonAdvSM).
SUMMIT is designed to evaluate the safety and efficacy of bezuclastinib in patients with moderate to severe Indolent Systemic Mastocytosis (ISM) or Smoldering Systemic Mastocytosis (SSM).
Actively enrolling patients in PEAK, a registrational randomized, open-label, global, Phase 3 clinical trial in patients with Gastrointestinal Stromal Tumors (GIST).
PEAK is designed to evaluate the efficacy and safety of bezuclastinib in combination with sunitinib compared to sunitinib alone in patients with locally advanced, unresectable or metastatic GIST who have received prior treatment with imatinib.
First Quarter 2022 Financial Results

Cash and Cash Equivalents: As of March 31, 2022, cash and cash equivalents were $191.0 million as compared to $219.7 million as of December 31, 2021. Based on its current plans, the company expects that its existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into 2024.

R&D Expenses: Research and development expenses were $25.5 million for the first quarter of 2022 compared to $8.2 million for the first quarter of 2021. The increase was primarily due to costs associated with the on-going APEX, SUMMIT and PEAK clinical trials and costs related to expanding the Cogent Research Team, which was formed in the second quarter of 2021.

G&A Expenses: General and administrative expenses were $5.9 million for the first quarter of 2022 compared to $4.6 million for the first quarter of 2021. The increase was primarily due to the growth of the organization.

Net Loss: Net loss was $30.6 million for the first quarter of 2022 compared to a net loss of $11.7 million for the first quarter of 2021.

On May 10, 2022 Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism pioneering therapies for genetically defined diseases, reported that the company is scheduled to present at the RBC Capital Markets Global Healthcare Conference on Tuesday, May 17, 2022, at 10:30 a.m. ET (Press release, Agios Pharmaceuticals, MAY 10, 2022, View Source [SID1234614095]).

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A live webcast of the presentation can be accessed under "Events & Presentations" in the Investors section of the company’s website at www.agios.com. A replay of the webcast will be archived on the Agios website for at least two weeks following the presentation.

On May 10, 2022 Bio-Techne Corporation (NASDAQ:TECH) reported that Exosome Diagnostics, a Bio-Techne brand, will have a major scientific presence at the annual American Urological Association Conference (AUA) taking place May 13-16 in New Orleans, LA (Press release, Bio-Techne, MAY 10, 2022, View Source [SID1234614094]). Exosome Diagnostics will have two poster presentations, including a podium presentation, six in-booth scientific presentations, and a live symposium with American icon, National Baseball Hall of Famer, and former Baltimore Orioles player "Iron Man" Cal Ripken, Jr. with leading urologist, Dr. Ronald Tutrone of Medical Director of Clinical Research at Chesapeake Urology in Baltimore, Maryland.

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Scientific presentations include:

A Combined Biomarker/mpMRI Approach Provides Enhanced Clinical Information Prior to Prostate Biopsy by Dr. Alexander Kretschmer et al. on Friday, May 13, 2022, 1:00 pm – 3:00 pm, Room 245, Ernest N. Morial Convention Center

Development of Exosome-based Plasma RNA Biomarkers for High-Risk Prostate Cancer by Dr. Sandra Gaston, et al. on Saturday, May 14, 2022, 10:30 am – 11:45 am, Room 222, Ernest N. Morial Convention Center

Validation of a urinary exosome mRNA signature for the diagnosis of human kidney transplant rejection by Dr. Rania El Fakih on Sunday, May 15, 2022, 7:00 am – 8:15 am, Room 228, Ernest N. Morial Convention Center

Exosome’s Chief Scientific Officer, Dr. Johan Skog, will lead the in-booth scientific presentation series with recently published results of the association of the ExoDx Prostate (EPI) test with post-prostatectomy outcome. Additional presentations include results of the ExoDx Prostate test clinical utility study as well as a 2.5 year follow-up and interim analysis of subjects who participated in the study and will conclude with the combined performance of the ExoDx Prostate test and MRI in the early detection of prostate cancer.

Cal Ripken, Jr. returns to partner with Exosome Diagnostics and Bio-Techne as an ambassador and advocate of the ExoDx Prostate test, which became part of Cal’s own prostate cancer journey. During his live appearances at the AUA, Cal will use his high-profile platform and strong reputation among men in his age group to raise awareness of the importance of early detection of prostate cancer.

"We are so pleased to have a robust scientific presence at the AUA, and excited to continue our campaign with an American baseball legend, Cal Ripken, Jr.," stated Kim Kelderman, President of Diagnostics and Genomics Segment of Bio-Techne. "Cal is a great example of the powerful role our ExoDx Prostate test plays in the prostate cancer journey. We look forward to continuing to provide this important tool to the millions of men and their families who are facing difficult choices in the management of this disease."

One in nine American men will be diagnosed with prostate cancer during his lifetime. Prostate cancer (PCa) is the second leading cause of cancer death among American men and is the most commonly diagnosed cancer. The American Cancer Society estimates that 268,000 men will be diagnosed with prostate cancer in 2022 and projects that ~34,000 men will die from the disease. Currently, nearly 3.6 million American men are living with the disease – only slightly less than the population of Los Angeles.

The EPI test is a urine-based genomic test that helps inform the prostate biopsy decision. This liquid biopsy test has a positive coverage decision from Medicare and is included in the National Comprehensive Cancer Network (NCCN) guidelines for early detection of prostate cancer in men for both initial and repeat biopsy. It is performed by Exosome Diagnostics in its CLIA, ISO, NY certified and CAP-accredited laboratory located in Waltham, Massachusetts. The EPI test is a risk assessment tool that assists physicians and their patients with determining if a prostate biopsy is needed when presented with an ambiguous PSA test result, thereby reducing complications from unnecessary and invasive procedures. The ExoDx Prostate is the leading test for at-home collection, enabling thousands of patients unable to travel to their healthcare professional for a routine office visit to assess prostate cancer risk.

On May 10, 2022 CTI BioPharma Corp. (Nasdaq: CTIC) reported one poster presentation from the Company’s pacritinib program at the 2022 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, being held in Chicago and virtually, June 3-7, 2021 (Press release, CTI BioPharma, MAY 10, 2022, View Source [SID1234614093]).

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The details of the poster presentation are as follows:

Abstract Title: Risk-adjusted safety analysis of pacritinib (PAC) in patients (pts) with myelofibrosis (MF)
Abstract Number: 7058
Session Name: Hematologic Malignancies—Leukemia, Myelodysplastic Syndromes, and Allotransplant
Session Date: Saturday, June 4, 2022
Presentation Time: 8:00 – 11:00 a.m. CDT (11:00 a.m. – 2:00 p.m. ET)
Presenter: Dr. Naveen Pemmaraju

About Pacritinib
Pacritinib is an oral kinase inhibitor with activity against wild type Janus Associated Kinase 2 (JAK2), mutant JAK2V617F form and FMS-like tyrosine kinase 3 (FLT3), which contribute to signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. Myelofibrosis is often associated with dysregulated JAK2 signaling. Pacritinib has higher inhibitory activity for JAK2 over other family members, JAK3 and TYK2. At clinically relevant concentrations, pacritinib does not inhibit JAK1. Pacritinib exhibits inhibitory activity against additional cellular kinases (such as CSF1R and IRAK1), the clinical relevance of which is unknown.

On May 10, 2022 Aclaris Therapeutics, Inc. (NASDAQ: ACRS), a clinical-stage biopharmaceutical company focused on developing novel drug candidates for immuno-inflammatory diseases, reported its financial results for the first quarter of 2022 and provided a corporate update (Press release, Aclaris Therapeutics, MAY 10, 2022, View Source [SID1234614092]).

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"We have continued to progress our clinical programs, including activating multiple clinical sites in our Phase 2b trial of ATI-1777 in subjects with moderate to severe atopic dermatitis," said Dr. Neal Walker, President and CEO of Aclaris. "We look forward to advancing all of our clinical and preclinical programs."

Research and Development Highlights:

Clinical Programs

Zunsemetinib, an investigational oral small molecule MK2 inhibitor:
Currently being developed as a potential treatment for immuno-inflammatory diseases

ATI-450-RA-202: This Phase 2b dose ranging trial to investigate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of multiple doses (20 mg and 50 mg twice daily) of zunsemetinib in combination with methotrexate in subjects with moderate to severe rheumatoid arthritis (RA) is ongoing.
Aclaris expects topline data in 2023.

ATI-450-HS-201: This Phase 2a trial to investigate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of zunsemetinib (50 mg twice daily) in subjects with moderate to severe hidradenitis suppurativa (HS) is ongoing.
Aclaris expects topline data in the first half of 2023.

ATI-450-PsA-201: Aclaris expects to activate clinical sites in the coming weeks in this Phase 2a trial of zunsemetinib (50 mg twice daily) in subjects with moderate to severe psoriatic arthritis.
ATI-1777, an investigational topical "soft" Janus kinase (JAK) 1/3 inhibitor:
Currently being developing as a potential treatment for moderate to severe atopic dermatitis (AD)

ATI-1777-AD-202: Aclaris activated multiple clinical sites in May 2022 in this Phase 2b trial to determine the efficacy, safety, tolerability and pharmacokinetics of ATI-1777 in subjects with moderate to severe AD. In this trial, Aclaris will explore multiple concentrations of twice daily treatment with ATI-1777 and a single concentration of once daily treatment with ATI-1777, in patients 12 years and older.
Aclaris expects topline data in the first half of 2023.
ATI-2138, an investigational oral ITK/TXK/JAK3 (ITJ) inhibitor:
Currently being developed as a potential treatment for T cell-mediated autoimmune diseases

ATI-2138-PKPD-101: This Phase 1 single ascending dose (SAD) trial to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of ATI-2138 in healthy subjects is ongoing.
Aclaris expects topline data in 2022.

If the Phase 1 SAD trial is successful, Aclaris currently plans to initiate a two-week Phase 1 multiple ascending dose trial of ATI-2138 in subjects with psoriasis in 2022. Aclaris is also currently exploring alternative indications to the planned indication that are relevant to the mechanism of action.
Preclinical Programs

ATI-2231, an investigational oral MK2 inhibitor compound:
Currently being explored as a potential treatment for pancreatic cancer and metastatic breast cancer as well as in preventing bone loss in patients with metastatic breast cancer

Second MK2 inhibitor generated from Aclaris’ proprietary KINect drug discovery platform and designed to have a long half-life.

IND-enabling studies are underway, and Aclaris expects to submit an IND by the end of 2022.
Discovery Programs

Currently developing oral gut-biased JAK inhibitors with limited systemic exposure as potential treatments for inflammatory bowel disease.
Central nervous system (CNS) kinase inhibitor targets:
Currently engaged in research to identify brain penetrant kinase inhibitor candidates and assess their impact on neuronal pro-inflammatory cytokine production, microglia growth and survival, and neurodegeneration.
Other Highlights

Aclaris continues to expand its senior R&D team and recently appointed Ian Anderson, Ph.D., as Executive Vice President, Translational Research & Development, and Rob Ortmann, M.D., as Vice President, Clinical Development. Dr. Anderson brings more than 30 years of immunology research experience in drug development, from discovery through Phase 2. He previously held senior scientific leadership roles at Flame Biosciences, Janssen Pharmaceutical, MedImmune and Cambridge Antibody Technology. Dr. Ortmann is a board-certified rheumatologist with more than 10 years of clinical research experience in autoimmune-related therapeutic areas. He previously held clinical development positions at Horizon Therapeutics and Eli Lilly and Company.

Financial Highlights:

Liquidity and Capital Resources

As of March 31, 2022, Aclaris had aggregate cash, cash equivalents and marketable securities of $204 million compared to $226 million as of December 31, 2021. Additionally, in April 2022, Aclaris sold approximately 4.8 million shares under its ATM facility for aggregate net proceeds of $73 million.

Aclaris now anticipates that its cash, cash equivalents and marketable securities as of March 31, 2022 in combination with the $73 million in net proceeds from the April 2022 ATM sale will be sufficient to fund its operations through the end of 2025, without giving effect to any additional potential business development transactions or financing activities.

Financial Results

First Quarter 2022

Net loss was $18.8 million for the first quarter of 2022 compared to $28.8 million for the first quarter of 2021.
Total revenue was $1.5 million for the first quarter of 2022 compared to $1.8 million for the first quarter of 2021.
Research and development (R&D) expenses were $14.3 million for the quarter ended March 31, 2022 compared to $7.8 million for the prior year period.
The $6.5 million increase was primarily the result of:
Higher zunsemetinib development expenses, including costs associated with clinical activities for a Phase 2b trial for RA and a Phase 2a trial for HS.
Higher ATI-1777 development expenses related to drug candidate manufacturing and other preclinical activities and start-up costs associated with a Phase 2b clinical trial.
Higher preclinical development activities related to ATI-2231.

General and administrative (G&A) expenses were $6.1 million for the quarter ended March 31, 2022 compared to $4.8 million for the prior year period.
The $1.3 million increase was primarily the result of higher compensation-related costs, including stock-based compensation, due to increased headcount and the impact of new equity awards granted during the first quarter of 2022.
Revaluation of contingent consideration resulted in a $1.2 million credit for the quarter ended March 31, 2022 compared to a contingent consideration charge of $16.4 million for the prior year period.