On August 7, 2023 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 second quarter of 2023 and provided a corporate update (Press release, Aclaris Therapeutics, AUG 7, 2023, View Source [SID1234633858]).

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"During the second quarter of this year, we continued to make positive strides across our clinical development pipeline, most notably through the completion of enrollment in our Phase 2b trial of zunsemetinib in patients with rheumatoid arthritis, which positions that trial to be our next Phase 2 data read-out," stated Doug Manion, M.D., Chief Executive Officer of Aclaris. "The next several quarters are lining up well with data read-outs for each of our clinical stage development therapeutics, all created by our KINect drug discovery platform."

Research and Development Highlights:

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

Rheumatoid Arthritis (ATI-450-RA-202): This Phase 2b placebo-controlled dose ranging trial to investigate the efficacy, safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) 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) completed enrollment in June 2023. Aclaris continues to expect topline data in the fourth quarter of 2023.

Psoriatic Arthritis (ATI-450-PsA-201): This Phase 2a placebo-controlled trial to investigate the efficacy, safety, tolerability, PK and PD of zunsemetinib (50 mg twice daily) in subjects with moderate to severe psoriatic arthritis (PsA) is ongoing. Aclaris continues to expect topline data in the first half of 2024.
ATI-1777, an investigational topical "soft" Janus kinase (JAK) 1/3 inhibitor:
Currently being developed as a potential treatment for mild to severe atopic dermatitis (AD)

Atopic Dermatitis (ATI-1777-AD-202): This Phase 2b vehicle-controlled trial to determine the efficacy, safety, tolerability, and PK of multiple doses and application regimens of ATI-1777 in subjects with mild to severe AD is ongoing. Aclaris continues to expect topline data in the second half of 2023.
ATI-2138, an investigational oral covalent ITK/JAK3 inhibitor:
Currently being developed as a potential treatment for ulcerative colitis; Aclaris is also exploring additional indications for other T cell-mediated autoimmune diseases

Healthy Volunteers (ATI-2138-PKPD-102): This two-week Phase 1 MAD (multiple ascending dose) trial to investigate the safety, tolerability, PK and PD of ATI-2138 in healthy volunteers has been completed. Based on a preliminary analysis of the PK, PD and safety, Aclaris believes the data support the progression of ATI-2138 into Phase 2 clinical development in ulcerative colitis. Aclaris expects to report the data in September 2023.
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

This is the second MK2 inhibitor generated from Aclaris’ proprietary KINect drug discovery platform and is designed to have a long plasma half-life.

Aclaris is supporting Washington University in a first-in-human investigator-initiated Phase 1a trial of ATI-2231 in patients with advanced solid tumor malignancies. Aclaris expects clinical development activities to be initiated in the second half of 2023.
Financial Highlights:

Liquidity and Capital Resources

As of June 30, 2023, Aclaris had aggregate cash, cash equivalents and marketable securities of $210.8 million compared to $229.8 million as of December 31, 2022. Aggregate cash, cash equivalents and marketable securities as of June 30, 2023 included $26.7 million of net proceeds from the sale of 3.4 million shares under its ATM facility in April 2023.

Aclaris continues to anticipate that its cash, cash equivalents and marketable securities as of June 30, 2023 will be sufficient to fund its operations through the end of 2025, without giving effect to any potential business development transactions or additional financing activities.

Financial Results

Second Quarter 2023

Net loss was $29.6 million for the second quarter of 2023 compared to $20.5 million for the second quarter of 2022.

Total revenue was $1.9 million for the second quarter of 2023 compared to $1.5 million for the second quarter of 2022. The increase was driven by higher licensing revenue primarily from royalties earned on out-licensed intellectual property.

Research and development (R&D) expenses were $25.3 million for the quarter ended June 30, 2023 compared to $18.8 million for the prior year period.
The $6.5 million increase was primarily the result of higher:
Zunsemetinib development expenses related to drug candidate manufacturing and costs associated with clinical activities for a Phase 2b trial for RA.
ATI-2138 development expenses, including costs associated with a Phase 1 MAD trial and other preclinical activities.
Compensation-related expenses due to an increase in headcount.

General and administrative (G&A) expenses were $8.3 million for the quarter ended June 30, 2023 compared to $6.1 million for the prior year period. The increase was primarily due to bad debt expense recorded from Aclaris’ determination that collection of amounts due from EPI Health are uncertain as a result of their filing for Chapter 11 bankruptcy protection. Compensation-related expenses also increased due to an increase in headcount.

Licensing expenses were $0.6 million for the quarter ended June 30, 2023 resulting from separate third-party contractual obligations related to the non-exclusive patent license agreement with Lilly. There were no licensing expenses for the quarter ended June 30, 2022.

Revaluation of contingent consideration resulted in a $1.5 million gain for the quarter ended June 30, 2023 compared to a gain of $3.4 million for the prior year period.
Year-to-date 2023

Net loss was $57.7 million for the six months ended June 30, 2023 compared to $39.3 million for the six months ended June 30, 2022.

Total revenue was $4.4 million for the six months ended June 30, 2023 compared to $3.0 million for the six months ended June 30, 2022.

R&D expenses were $47.9 million for the six months ended June 30, 2023 compared to $33.1 million for the prior year period.
The $14.8 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 PsA.
ATI-2138 development expenses, including costs associated with a Phase 1 MAD trial and other preclinical activities.
Compensation-related expenses due to an increase in headcount.

G&A expenses were $17.1 million for the six months ended June 30, 2023 compared to $12.2 million for the prior year period.
The $4.9 million increase was primarily the result of higher compensation-related costs, including stock-based compensation, due to increased headcount and the impact of equity awards granted during the six months ended June 30, 2023. Bad debt expense recorded from Aclaris’ determination that collection of amounts due from EPI Health are uncertain as a result of their filing for Chapter 11 bankruptcy protection also contributed to the increase.
Revaluation of contingent consideration resulted in a $2.3 million gain for the six months ended June 30, 2023 compared to a gain of $4.6 million for the prior year period.

On August 6, 2023 Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancer, chronic hepatitis B (CHB), and age-related diseases, reported that lisaftoclax (APG-2575), a novel Bcl-2 inhibitor and one of the company’s core assets, has been cleared by the US Food and Drug Administration (FDA) to enter a global registrational Phase III study for treatment of patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who were BTKi previously treated (Press release, Ascentage Pharma, AUG 6, 2023, View Source [SID1234633837]). This clearance marks a major step-forward in the global development of lisaftoclax and another important milestone following the approval by the Center for Drug Evaluation (CDE) in China for the registrational Phase II study of lisaftoclax (APG-2575) in patients with relapsed/refractory CLL/SLL (R/R CLL/SLL) in December 2021, as it could potentially pave the way for lisaftoclax to become the second Bcl-2 inhibitor approved anywhere globally.

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This global, multi-center, randomized-controlled, registrational Phase III study (APG2575CG301) is designed to assess the efficacy and safety of lisaftoclax (APG-2575) plus a Bruton’s tyrosine kinase inhibitor (BTKi) in patients with CLL/SLL who were BTKi previously treated. The study is set to commence in the second half of 2023.

CLL/SLL is the most common form of leukemia in adults, accounting for one-quarter of all leukemia cases in the Western World, with over 100,000 new diagnoses globally every year1. Despite significant initial responses to current first-line treatments such as immunotherapies, chemotherapies, and BTKis, relapse and drug resistance remain a major clinical challenge. At present, patients with CLL/SLL still have a huge unmet medical need for new treatment options.

Lisaftoclax (APG-2575) is a novel, orally administered small-molecule Bcl-2 selective inhibitor developed by Ascentage Pharma to treat a range of malignancies by selectively blocking the antiapoptotic protein Bcl-2, thus restoring the normal apoptosis process in cancer cells. With strong global best-in-class potential, lisaftoclax (APG-2575) is the first Bcl-2 inhibitor in China and the second anywhere globally that has demonstrated compelling clinical activity and entered a pivotal study.

Currently, Ascentage Pharma initiated a total of 19 clinical studies of lisaftoclax (APG-2575) globally, having treated more than 600 patients with the drug thus far, including more than 300 patients with CLL/SLL. In prior studies, lisaftoclax, both as a monotherapy and in combinations, showed clear therapeutic potential as a safe, effective, and easy-to-use therapy for patients with CLL/SLL. According to the initial results from a global Phase II study, lisaftoclax (APG-2575) combined with the next-generation BTKi acalabrutinib achieved encouraging objective response rates (ORRs) in patients with CLL/SLL, including an ORR of 100% (16/16) in treatment-naïve patients and 98% (56/57) in relapsed/refractory patients. In terms of safety, combination regimens demonstrated a safety profile that was on par with lisaftoclax (APG-2575) monotherapy and an extremely low incidence of tumor lysis syndrome (TLS). Furthermore, the study of lisaftoclax (APG-2575) was initiated with a daily dose ramp-up schedule that was friendly to patients and allowed them to quickly reach target doses2.

"With progress in the research and development of targeted therapies, we have seen considerable improvement to the survival of patients with CLL/SLL. However, there remain major clinical challenges and urgent unmet medical needs for novel therapies that are safe and effective," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "Lisaftoclax, a key drug candidate of our apoptosis-targeted pipeline with global best-in-class potential, has shown promising efficacy and favorable safety in earlier studies. We are very encouraged by the FDA’s clearance for the global registrational Phase III study as it marks a major milestone in the development of lisaftoclax. Fulfilling the mission of addressing unmet clinical needs in China and around the world, we will press ahead with the global registrational Phase III study of lisaftoclax(APG-2575) to allow patients around the world to benefit from this novel therapeutic as soon as possible."

On August 7, 2023 Clarity Pharmaceuticals (ASX: CU6) ("Clarity", "the Company"), a clinical stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for children and adults with cancer, reported an exclusive license from Memorial Sloan Kettering Cancer Center (MSK) (Press release, Clarity Pharmaceuticals, AUG 6, 2023, View Source [SID1234633834]). The license is to intellectual property that covers cutting-edge technology that enables antibody "pre-targeting" for the diagnosis and treatment of cancer.

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Pre-targeting is a radiopharmaceutical approach to diagnosing and treating cancer patients that harnesses the benefits of antibody targeting, amplifying uptake of radiopharmaceutical products in cancerous tissue, while reducing healthy tissue exposure to radiation that can arise due to the slow clearance of antibodies. This is achieved by tagging an antibody, designed specifically to target cancer cells, and then injecting it into the body. After several days, a chaser compound, which only attaches to the antibody tag, is injected. The chaser compound is initially radiolabelled with copper-64 to enable imaging with a Positron Emission Tomography (PET) camera which visualises the extent of cancer burden. Once the cancer is visualised, a second administration of the chaser is administered, this time radiolabelled with the therapeutic radionuclide copper-67, so that the cancer cells can be irradiated with the goal of killing the tumours.

The ability of the chaser compound to attach to the tag on the antibody occurs due to a "click chemistry" reaction. This chemistry is so groundbreaking, it was awarded a Nobel Prize in Chemistry to scientists Carolyn R. Bertozzi, Morten Meldal and K. Barry Sharpless in 2022 for Click Chemistry and Bioorthogonal Chemistry.

This technology, developed at MSK by Jason Lewis, PhD and Dr Brian Zeglis (formerly MSK, currently Hunter College, NY USA), has been licensed to Clarity under a worldwide exclusive license. Key publications on the pre-targeting research include PNAS1 and NEJM2. This IP complements a growing number of patent applications in Clarity’s portfolio covering various aspects of antibody pre-targeting: International Application No.PCT/AU2019/050322 (assigned to Clarity from the University of Melbourne); International Application No.PCT/AU2019/050324; and Australian Provisional Patent Application No. 2022903384.

A clinical trial using the MSK licensed technology is open for recruitment in patients with pancreatic cancer at MSK headed by Dr Pandit-Taskar (NCT05737615)3. The trial is titled: "PET Imaging Using 64Cu-Tz-SarAr and hu5B1-TCO in People With Pancreatic Cancer". It is a first-in-human diagnostic trial, utilising copper-64 and a sarcophagine chelator, core to Clarity’s SAR Technology. The antibody (hu5B1-TCO) being used targets pancreatic cancer.

Clarity’s Executive Chairman, Dr Alan Taylor, commented, "Pre-targeting presents an exciting and groundbreaking avenue to pursue our mission to develop next-generation products that improve treatment outcomes for children and adults with cancer. Pre-targeting holds promise of overcoming the safety issues of antibody-based radiopharmaceuticals, opening up a massive opportunity to use the large cache of antibodies developed over the last 20 years and applying it in the theranostic setting. Combining this approach with the "perfect pairing" of copper-64 and copper-67 represents an untapped opportunity to deliver significant payloads to cancers in a safer and more effective way as we start to turn the tide on our war with cancer.
"Pre-targeting using copper-64 in MSK’s first-in-human trial is in alignment with Clarity’s clinical and commercial development of the SAR Technology to make a pipeline of theranostic products. The trial will validate the technique, and, if positive, will pave the way for a whole new area of development, not only for Clarity, but the entire field of antibody-based therapy. We have valued our engagement with MSK on many fronts over the years and we look forward to progressing the development of MSK’s pre-targeting technology, potentially providing a new path for the accurate and precise detection and treatment of various cancers."

Dr Jason Lewis, Emily Tow Chair in Oncology and Vice Chair for Research, Department of Radiology at MSK, commented, "We have been conducting research on pre-targeting for many years now and are very excited with the impending first-in-human clinical trial by our team here at MSK. Pre-targeting is a promising method of treatment for cancer patients and the continued exploration and validation of the benefits associated with it in a clinical setting is the next stage for this field. To have Clarity involved now on the clinical and commercial development of a product is excellent and is a continuation of their support and input on the preclinical research to date. Although pre-targeting has been in humans in the past, using various techniques, the field is still in its infancy. The theranostic approach in pre-targeting with the next generation of isotopes, such as copper-64 and copper-67, provides a renewed interest in the field, with a focus on both diagnosis and therapy. This technique has the ability to widen the whole field of nuclear medicine and utilise a large array of existing knowledge on antibody therapies."

On August 4, 2023 Oncolys BioPharma reported its Non-consolidated Financial Results for the Six Months Ended June 30, 2023 (Press release, Oncolys BioPharma, AUG 4, 2023, View Source [SID1234634882]).

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On August 4, 2023, iBio, Inc. (the "Company") reported to have agreed to amend the exercise price with certain holders of the Series A Warrants and Series B Warrants that were acquired from the Company in the underwritten public offering that was completed in December 2022 (Filing, 8-K, iBioPharma, AUG 4, 2023, View Source [SID1234633874]). Under the amended warrants, the Company agreed to amend existing Series A warrants to purchase up to 3,475,916 shares of common stock and existing Series B warrants to purchase up to 2,058,000 shares of common stock that were previously issued in December 2022 to the certain investors in the public offering, with exercise prices of $1.04 per share (the "Existing Warrants"), to lower the exercise price of the Existing Warrants to $0.50 per share.

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