On November 6, 2024 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 third quarter of 2024 and provided a corporate update (Press release, Aclaris Therapeutics, NOV 6, 2024, View Source [SID1234647796]).

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"The third quarter of 2024 marked an important milestone for Aclaris with the dosing of our first patient in the Phase 2a trial of ATI-2138 for moderate to severe atopic dermatitis," said Dr. Neal Walker, Interim President & CEO and Chair of the Board of Directors of Aclaris. "This milestone, combined with our robust financial position, underscores our commitment to executing a capital-efficient development strategy."

Research and Development Highlights:

● ITK Inhibitor Programs
● ATI-2138, an investigational oral covalent ITK/JAK3 inhibitor
o Atopic Dermatitis (ATI-2138-AD-201): This Phase 2a open-label trial to investigate the safety, tolerability, pharmacokinetics, efficacy, and pharmacodynamics of ATI-2138 in patients with moderate to severe atopic dermatitis (AD) is ongoing. Aclaris continues to expect top-line data in the first half of 2025.
● ITK Selective Compound
o Aclaris is progressing a second generation ITK selective inhibitor to development candidate selection for autoimmune indications.
● Lepzacitinib (ATI-1777), an investigational topical "soft" JAK 1/3 inhibitor
o In January 2024, Aclaris reported positive top-line results from its Phase 2b trial of lepzacitinib in AD.
o Aclaris is currently seeking a global development and commercialization partner for this program (excluding Greater China). As previously announced, in 2022 Aclaris granted Pediatrix Therapeutics exclusive rights to develop and commercialize lepzacitinib in Greater China.
● Zunsemetinib (ATI-450), an investigational oral small molecule MK2 inhibitor
o Aclaris plans to support Washington University in St. Louis in its investigator-initiated Phase 1b/2 trials of zunsemetinib as a potential treatment for pancreatic cancer and metastatic breast cancer. Aclaris expects these trials to be primarily funded by grants awarded to Washington University.

Financial Highlights:

Liquidity and Capital Resources

As of September 30, 2024, Aclaris had aggregate cash, cash equivalents and marketable securities of $173.4 million compared to $181.9 million as of December 31, 2023.

Aclaris anticipates that its cash, cash equivalents and marketable securities as of September 30, 2024 will be sufficient to fund its operations into 2028, without giving effect to any potential business development transactions, financing activities or the outcome of its strategic review.

Financial Results

Third Quarter 2024

● Net loss was $7.6 million for the third quarter of 2024 compared to $29.3 million for the third quarter of 2023.
● Total revenue was $4.3 million for the third quarter of 2024 compared to $9.3 million for the third quarter of 2023. The decrease was primarily driven by higher milestones earned during the prior year period compared to the current year period.
● Research and development (R&D) expenses were $6.0 million for the quarter ended September 30, 2024 compared to $23.9 million for the prior year period.
o The $17.9 million decrease was primarily the result of:
◾ Zunsemetinib development expenses associated with clinical trials in 2023, and drug candidate manufacturing costs;
◾ Costs associated with lepzacitinib preclinical development activities and a Phase 2b clinical trial for AD which was completed in January 2024;
◾ ATI-2138 development expenses, including costs associated with a Phase 1 multiple ascending dose (MAD) trial which was completed in September 2023 and other preclinical activities, which were partially offset by clinical development expenses associated with a Phase 2a clinical trial which commenced in August 2024; and
◾ Lower compensation-related expenses due to a decrease in headcount and higher forfeiture credits.
● General and administrative (G&A) expenses were $5.7 million for the quarter ended September 30, 2024 compared to $7.1 million for the corresponding prior year period. The decrease was primarily due to a reduction in compensation-related expenses due to lower headcount and higher forfeiture credits.
● Licensing expenses were $1.8 million for the quarter ended September 30, 2024 compared to $7.3 million for the corresponding prior year period. The decrease was primarily due to higher milestones earned during the prior year period compared to the current year period.
● Revaluation of contingent consideration resulted in a $0.8 million loss for the quarter ended September 30, 2024 compared to a loss of $1.7 million for the prior year period.
Year-to-date 2024

● Net loss was $35.5 million for the nine months ended September 30, 2024 compared to $87.0 million for the nine months ended September 30, 2023.
● Total revenue was $9.5 million for the nine months ended September 30, 2024 compared to $13.7 million for the nine months ended September 30, 2023. The decrease was primarily driven by higher milestones earned during the prior year period compared to the current year period.
● R&D expenses were $24.6 million for the nine months ended September 30, 2024 compared to $71.7 million for the corresponding prior year period.
o The $47.1 million decrease was primarily the result of:
◾ Zunsemetinib development expenses associated with clinical trials in 2023, and drug candidate manufacturing costs;
◾ Costs associated with lepzacitinib preclinical development activities and a Phase 2b clinical trial for AD which was completed in January 2024;
◾ ATI-2138 development expenses, including costs associated with a Phase 1 MAD trial which was completed in September 2023 and other preclinical activities, which were partially offset by clinical development expenses associated with a Phase 2a clinical trial which commenced in August 2024; and
◾ Lower compensation-related expenses due to a decrease in headcount and higher forfeiture credits.
● G&A expenses were $17.2 million for the nine months ended September 30, 2024 compared to $24.2 million for the prior year period. The decrease was primarily due to a reduction in compensation-related expenses due to lower headcount and higher forfeiture credits and the recognition of bad debt expense recorded in the prior year period from Aclaris’ determination that collection of amounts due from EPI Health are uncertain as a result of their filing for Chapter 11 bankruptcy protection.
● Licensing expenses were $4.1 million for the nine months ended September 30, 2024 compared to $9.0 million for the prior year period. The decrease was primarily due to higher milestones earned during the prior year period compared to the current year period.
● Revaluation of contingent consideration resulted in a $3.8 million loss for the nine months ended September 30, 2024 compared to a gain of $0.6 million for the corresponding prior year period.

On November 5, 2024 Avid Bioservices, Inc. (NASDAQ: CDMO) ("Avid" or the "Company"), a dedicated biologics contract development and manufacturing organization ("CDMO") working to improve patient lives by providing high quality development and manufacturing services to biotechnology and pharmaceutical companies, GHO Capital Partners LLP ("GHO") and Ampersand Capital Partners ("Ampersand") reported they have entered into a definitive merger agreement for Avid to be acquired by funds managed by GHO and Ampersand in an all-cash transaction valued at approximately $1.1 billion (Press release, Avid Bioservices, NOV 6, 2024, View Source [SID1234647786]).

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Under the terms of the merger agreement, GHO and Ampersand would acquire all the outstanding shares held by Avid’s stockholders for $12.50 per share in cash. The per share purchase price represents a 13.8% premium to Avid’s closing share price of $10.98 on November 6, 2024, the last full trading day prior to the transaction announcement, and a 21.9% premium to the Company’s 20-day volume-weighted average share price for the period ended November 6, 2024. This transaction equates to an enterprise value of approximately $1.1 billion, a 6.3x multiple to consensus FY2025E revenue.

"Since our founding, Avid Bioservices’ business has grown by evolving to meet our customers’ broad range of development and manufacturing needs. After years of investment and expansion, now is the right time to move forward as a private company with new owners that will support our next phase," stated Nick Green, president and CEO of Avid Bioservices. "In evaluating this transaction, our Board considered a range of alternatives and determined that it provides our stockholders significant, immediate and certain cash value for their shares. Partnering with GHO Capital and Ampersand Capital Partners allows us to build on our strong foundation by accessing their significant knowledge base, network and capital to position the business for the future with our customers."

"We are excited to announce this recommended cash acquisition of Avid," said Alan MacKay and Mike Mortimer, Managing Partners of GHO. "As experienced CDMO industry investors, GHO brings deep expertise and experience to support Avid’s management team going forward. Our mission at GHO is to make healthcare better, faster, and more accessible and at the heart of this is enabling efficient, high-quality manufacturing of innovative treatments. Avid exemplifies this perfectly – the Company operates in high-growth markets, producing complex biologics for leading pharmaceutical and biotech innovators at both the clinical and commercial stages. Avid’s recent investments, both in capacity and its exemplary team, position it strongly for future growth. We look forward to working with the Avid team to unlock the Company’s full potential through our established playbook of expanded offerings, talent investment and greater geographic reach."

"Avid has long been a trusted provider of biopharmaceutical development and manufacturing services, and we have tremendous respect for its team’s expertise, its broad spectrum of customized services and its strong regulatory track record. We look forward to leveraging our deep industry experience, focused strategy, and collaborative approach to drive growth," said, David Anderson, General Partner of Ampersand.

Transaction Details

The transaction, which was unanimously approved by the Avid Board of Directors, is currently expected to close in the first quarter of 2025, subject to customary closing conditions, including approval by Avid’s stockholders and receipt of required regulatory approvals. The transaction is not subject to a financing condition. The companies will continue to operate independently until the proposed transaction is finalized.

Upon completion of the transaction, Avid common stock will no longer be listed on any public stock exchange. The Company will continue to operate under the Avid name and brand.

Advisors

Moelis & Company LLC is serving as exclusive financial advisor to Avid, and Cooley LLP is serving as legal counsel to Avid. William Blair & Company, LLC is serving as exclusive financial advisor and Ropes & Gray LLP is serving as legal counsel to GHO and Ampersand.

On November 5, 2024 Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, reported the presentation of data from its MP0533 and MP0621 programs at the upcoming Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in San Diego, running December 7–10, 2024 (Press release, Molecular Partners, NOV 5, 2024, View Source [SID1234655799]).

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The poster presentation details are as follows:
Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Poster II
Publication Number: 2881
Title: MP0533 (CD33 x CD123 x CD70 x CD3), a Tetra-Specific CD3-Engaging Darpin for the Treatment of Patients with Relapsed/Refractory AML or MDS/AML: Results of an Ongoing Phase 1/2a Study
Session Location: San Diego Convention Center, Halls G-H
Presentation Date & Time: Sunday, December 8, 2024, 6:00–8:00 pm PT

Session Name: 701. Experimental Transplantation: Basic and Translational: Poster III
Publication Number: 4775
Title: MP0621 (cKit x CD16a x CD47), a Multi-Specific Switch-Darpin with Conditional Blockade of CD47 Targeting Hematopoietic Stem Cells: Preclinical Evaluation of a Next-Generation Conditioning Agent for Stem Cell Transplantation
Session Location: San Diego Convention Center, Halls G-H
Presentation Date & Time: Monday, December 9, 2024, 6:00–8:00 pm PT

The full abstracts will be available on the ASH (Free ASH Whitepaper) website from 9:00 am ET on November 5, 2024.

About MP0533 (CD33 x CD123 x CD70 x CD3)

MP0533 is a novel tetraspecific T cell engaging DARPin which simultaneously targets the three tumor-associated antigens (TAAs) CD33, CD123 and CD70, as well as CD3 on T cells. The mechanism of action of MP0533 is designed to preferentially kill AML cells that express any combination of these three TAAs while sparing healthy cells, which express only one or none of these targets. The immune activation against the malignant cells is achieved through CD3-mediated T cell-engagement.

The poster to be presented at ASH (Free ASH Whitepaper) 2024 will provide a clinical update of the ongoing first-in-human dose-escalation phase 1/2a study of MP0533 in patients with relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS)/AML. MP0533 showed an acceptable safety profile in the first 7 dose cohorts, with the majority of adverse events reported being infusion-related reactions and cytokine release syndrome.

Based on this observed tolerability profile and initial antitumor and pharmacodynamic activity data, Molecular Partners is amending the protocol to further optimize the dosing schedule and improve the exposure profile of MP0533.

About MP0621 (cKit x CD16a x CD47)

MP0621 is a Switch-DARPin candidate designed to induce killing of hematopoietic stem cells (HSCs) as a next-generation conditioning regimen for HSC transplantation (HSCT). The Switch-DARPin platform provides a logic-gated "on/off" function (the "Switch") to multispecific DARPin candidates leading to target activation only in the presence of defined antigens. In MP0621, the Switch-DARPin binds to either cellular cKit or to the anti-CD47 DARPin binder. Upon MP0621 binding to cKit on HSCs, the Switch-DARPin will unmask the anti-CD47 DARPin, which in turn will bind CD47 and block the "don’t-eat-me" signal, leveraging the power of CD47 inhibition without its associated toxicity to healthy cells.

The poster to be presented at ASH (Free ASH Whitepaper) 2024 builds on the data presented earlier this year at the European Haematology Association 2024 Congress and provides further preclinical in vivo proof-of-mechanism data, demonstrating that MP0621 could be an efficient next-generation conditioning regimen for autologous HSCT.

At present non-human primate data do not indicate that MP0621 would serve as a treatment for AML, as was previously hypothesized, in addition to HSCT. As Molecular Partners’ portfolio strategy prioritizes therapeutic candidates for oncology, MP0621 is being evaluated for partnering.

About DARPin Therapeutics
DARPin (Designed Ankyrin Repeat Protein) therapeutics are a new class of custom-built protein drugs based on natural binding proteins that open new dimensions of multi-functionality and multi-target specificity in drug design. The flexible architecture, intrinsic potential for high affinity and specificity, small size and high stability of DARPins offer benefits to drug design over other currently available protein-based therapeutics. DARPin candidates can be radically simple, with a single DARPin unit acting as the delivery vector to a specific target; or multispecific, with the possibility of engaging more than five targets, and combining multiple and conditional functionalities in a unique DARPin drug candidate. The DARPin platform is designed to be a rapid and cost-effective drug discovery engine, producing drug candidates with optimized properties and high production yields. DARPin therapeutics have been clinically validated across several therapeutic areas and developed through to the registrational stage.

On November 5, 2024 Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical stage biopharmaceutical company developing targeted protein modulation drugs designed to treat patients with cancer and inflammatory diseases, reported that data will be presented from its two Bruton’s tyrosine kinase (BTK) degrader programs, NX-5948 and NX-2127, in two oral presentations and one poster at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition being held December 7-10, 2024, in San Diego, CA (Press release, Nurix Therapeutics, NOV 5, 2024, View Source [SID1234649114]).

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Oral Presentation Details:

Title: Efficacy and Safety of the Bruton’s Tyrosine Kinase (BTK) Degrader NX-5948 in Patients with Relapsed/Refractory (R/R) Chronic Lymphocytic Leukemia (CLL): Updated Results from an Ongoing Phase 1a/b Study
Authors: Nirav N. Shah, Zulfa Omer, Graham Collins, Francesco Forconi, Alexey Danilov, John Byrd, Dima El Sharkawi, Emma Searle, Alvaro Alencar, Shuo Ma, Sarah Injac, Talha Munir
Session Name: 642. Chronic Lymphocytic Leukemia: Clinical and Epidemiological: Treating Refractory Disease-Novel Agents and Quality-of-Life
Session Date and Time: Monday, December 9, 2024, 2:45 p.m. – 4:15 p.m. PT
Presentation Time: 3:00 p.m. PT
Room: Marriott Marquis San Diego Marina, Marriott Grand Ballroom 5-6

Title: NX-2127 and NX-5948, Two Clinical Stage Cereblon-Recruiting BTK Degraders, Facilitate T Cell Functionality in Chronic Lymphocytic Leukemia
Authors: Tiana Huynh, Sonia Rodriguez-Rodriguez, Carly Roleder, Sarah Whelan, May Tan, Ernestine Lee, Paul Munson, and Alexey Danilov
Session Name: 641. Chronic Lymphocytic Leukemia: Basic and Translational: Therapeutic Vulnerabilities, Signaling, and Microenvironment
Session Date and Time: Saturday, December 7, 2024, 9:30 a.m. – 11:00 a.m. PT
Presentation Time: 10:30 a.m. PT
Room: Marriott Marquis San Diego Marina, Marriott Grand Ballroom 5-6

Poster Presentation Details

Title: BTK Degradation As a Novel Therapeutic Strategy in Relapsed CNS Lymphoma: Proof of Concept Studies in Intracranial Patient-Derived, Rodent Models

Authors: Jun Ma, Michael Randall, Ming Lu, Lingjing Chen, Huimin Geng, Aishwarya Kumar, Saloni Malla, Mark Noviski, Ryan Rountree, James L. Rubenstein
Session Name: 622. Lymphomas: Translational – Non-Genetic: Poster II
Session Date and Time: Sunday, December 8, 2024, 6:00 p.m. – 8:00 p.m. PT
Location: San Diego Convention Center, Halls G-H

About NX-5948: NX-5948 is an investigational, orally bioavailable degrader of BTK that is currently being evaluated in a Phase 1a/b clinical trial in adults with relapsed or refractory B-cell malignancies. Additional information on the Phase 1a/b clinical trial can be accessed at www.clinicaltrials.gov (NCT05131022).

About NX-2127: NX-2127 is an investigational, orally bioavailable degrader of BTK and cereblon neosubstrates Ikaros (IKZF1) and Aiolos (IKZF3). NX-2127 is currently being evaluated in a Phase 1a/b clinical trial in adults with relapsed or refractory B-cell malignancies. Additional information on the ongoing clinical trial can be accessed at www.clinicaltrials.gov (NCT04830137).

On November 5, 2024 C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science, reported that two abstracts from the ongoing Phase 1/2 trial of cemsidomide have been accepted for presentation at the ASH (Free ASH Whitepaper) Annual Meeting taking place from December 7 – 10, 2024 in San Diego, California (Press release, C4 Therapeutics, NOV 5, 2024, View Source [SID1234648575]). Non-Hodgkin’s lymphoma (NHL) data will be shared as an oral presentation and multiple myeloma (MM) data will be shared as a poster presentation. The abstracts are available online on the ASH (Free ASH Whitepaper) Annual Meeting website.

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The presentations on December 8th at the ASH (Free ASH Whitepaper) Annual Meeting will include updated data compared to the abstracts.

NHL Oral Presentation Details:

Title: Initial Results of a Phase 1 First-in-Human Study of Cemsidomide (CFT7455), a Novel MonoDAC Degrader, in Patients with Non-Hodgkin’s Lymphoma
Format: Oral (10-minute presentation, followed by a 5-minute discussion)
Session: T Cell, NK Cell, or NK/T Cell Lymphomas: Clinical and Epidemiological: When Old Meets New in T Cell Lymphomas
Session Date: Sunday, December 8, 2024
Presentation Time: 10:30 AM PT (1:30 PM ET)
Location: San Diego Convention Center, Ballroom 20CD

MM Poster Presentation Details:

Title: Initial Results of a Phase 1 First-in-Human Study of Cemsidomide (CFT7455), a Novel MonoDAC Degrader, with Dexamethasone in Patients with Relapsed/Refractory Multiple Myeloma
Format: Poster presentation
Session: Multiple Myeloma: Pharmacologic Therapies: Poster II
Session Date: Sunday, December 8, 2024
Presentation Time: 6:00 PM – 8:00 PM PT (9 PM – 11 PM ET)
Location: San Diego Convention Center, Halls G-H
Publication Number: 3366

C4T Investor Webcast

C4T will host a webcast on Sunday, December 8, 2024 at 2 PM PT (5 PM ET). A live webcast of the event will be available using this link or under the "Events & Presentations" page of the Investors section on the company’s website at www.c4therapeutics.com. A replay of the webcast will be archived and available following the event.