On November 6, 2024 Aligos Therapeutics, Inc. (Nasdaq: ALGS, "Aligos"), a clinical stage biopharmaceutical company focused on improving patient outcomes through best-in-class therapies for liver and viral diseases, reported recent business progress and financial results for the third quarter 2024 (Press release, Aligos Therapeutics, NOV 6, 2024, View Source [SID1234647798]).

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"This quarter we reached a key milestone when we announced the positive topline HERALD data in MASH subjects," stated Lawrence Blatt, Ph.D., MBA, Chairman, President, and Chief Executive Officer of Aligos Therapeutics. "With placebo-adjusted median relative reductions in liver fat of up to 46.2%, we continue to believe ALG-055009 has best-in-class potential. We are completing Phase 2b enabling studies and evaluating a variety of options to fund continued development, including potential partnering where discussions are underway. In addition, we are progressing ALG-000184 for CHB towards a Phase 2 study next year. Lastly, we expect to begin externally funded clinical studies for ALG-097558 later this year in COVID subjects. 2024 has been an exciting year for the company, and we believe we are laying the groundwork for important future successes in 2025 and beyond."

Recent Business Progress

Aligos Portfolio of Drug Candidates

ALG-000184: Potential first-/best-in-class small molecule CAM-E for CHB

Dosing continues in this ongoing Phase 1a/1b study, with subjects expected to dose for up to 96 weeks. Additional interim data readouts are planned to be presented this year at the American Association for the Study of Liver Disease’s (AASLD) The Liver Meeting (TLM) 2024
Received positive feedback from the FDA and the National Medical Products Administration in China to move forward with sustained HBV DNA suppression as the primary efficacy endpoint for future studies designed to support the potential registration of ALG-000184 for the treatment of hepatitis B infection
Announced a clinical collaboration with Xiamen Amoytop Biotech Co., Ltd.
Amoytop agreed to sponsor and perform a Phase 1b exploratory clinical study evaluating the efficacy and safety of ALG-000184 in combination with PEGBING (mipeginterferon alfa-2b) in chronic hepatitis B (CHB) patients in China
Phase 2 enabling activities are underway, including drug supply manufacturing
ALG-055009: Potential best-in-class small molecule THR-β agonist for MASH

Topline HERALD data were presented in September 2024, demonstrating that ALG-055009 dose groups met the primary endpoint with statistically significant reductions in liver fat at Week 12 as measured by MRI-PDFF
Doses of 0.5 mg to 0.9 mg ALG-055009 demonstrated statistically significant reductions in liver fat at Week 12, with placebo-adjusted median relative reductions up to 46.2% as measured by MRI-PDFF. Up to 70% of subjects achieved ≥30% relative reduction in liver fat compared to baseline
ALG-055009 demonstrated a favorable tolerability profile with no clinical hyper/hypothyroidism. Incidence of gastrointestinal-related treatment emergent adverse events were similar in ALG-055009 dose groups compared to placebo. Specifically, a non-dose-related, lower incidence of diarrhea was observed in ALG-055009 dose groups compared to placebo
Treatment with ALG-055009 resulted in significant reductions in atherogenic lipids, including LDL-C, lipoprotein (a) (LpA), and apolipoprotein B (ApoB). In addition, dose dependent increases in sex hormone binding globulin (SHBG), a marker of THR-β target engagement in the liver, were observed
Additional data readouts are planned to be presented this year at AASLD’s The Liver Meeting (TLM) 2024
ALG-097558: Potential best-in-class small molecule pan-coronavirus protease inhibitor

Three additional clinical studies are expected to begin in 2024
AGILE University of Liverpool, a UK-government supported platform trial (with MRC and Wellcome Trust funding), has agreed to sponsor and perform a study in high-risk COVID patients evaluating ALG-097558 as monotherapy or in combination with remdesivir
The NIAID has agreed to sponsor clinical studies evaluating pharmacokinetic (PK) differences in special populations (renal/hepatic impairment subjects)
Phase 2 enabling activities, including nonclinical and clinical studies, are underway with financial support from the NIH
Financial Results for the Third Quarter 2024

Cash, cash equivalents and investments totaled $74.9 million as of September 30, 2024, compared with $135.7 million as of December 31, 2023. We continue to believe our cash balance provides sufficient cash to fund planned operations through the end of 2025.

Net loss for the three months ended September 30, 2024 were $19.3 million or basic and diluted net loss per common share of $(3.07), compared to net losses of $18.0 million or basic and diluted net loss per common share of $(10.37) for the three months ended September 30, 2023.

Research and development (R&D) expenses for the three months ended September 30, 2024 were $16.8 million, compared with $15.9 million for the same period of 2023. The increase was primarily due to an increase in third party expenses for the clinical trials. Total R&D stock-based compensation expense incurred for the three months ended September 30, 2024 was $1.2 million, compared with $1.6 million for the same period of 2023.

General and administrative (G&A) expenses for the three months ended September 30, 2024 were $4.6 million, compared with $6.4 million for the same period of 2023. The decrease in G&A expenses for this comparative period is primarily due to a decrease in third party expenses including legal expenses. Total G&A stock-based compensation expense incurred for the three months ended September 30, 2024 was $0.9 million, compared with $1.6 million for the same period of 2023.

Interest and other income, net, for the three months ended September 30, 2024 was income of $1.0 million compared with income of $1.1 million for the same period of 2023.

On November 6, 2024 Alector, Inc. (Nasdaq: ALEC), a clinical-stage biotechnology company pioneering immuno-neurology, reported third quarter 2024 financial results and recent portfolio and business updates (Press release, Alector, NOV 6, 2024, View Source [SID1234647797]). As of September 30, 2024, Alector’s cash, cash equivalents, and investments totaled $457.2 million.

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"We continue to make meaningful progress advancing our innovative pipeline of product candidates and remain on track to report data in 2024 from the INVOKE-2 Phase 2 trial of AL002, the most advanced TREM2 candidate in clinical development for early Alzheimer’s disease," said Arnon Rosenthal, Ph.D., Chief Executive Officer of Alector. "We believe that increased TREM2 signaling via AL002 may recruit disease-fighting microglia to broadly counteract the progression of Alzheimer’s disease. INVOKE-2 is designed to provide meaningful insights into AL002’s potential benefits across a combination of clinical and functional endpoints, as well as imaging and fluid biomarkers. We also believe that the broad mechanism of AL002 may have the potential to deliver potent and durable therapeutic benefits, both as a standalone therapy and in combination with anti-amyloid beta antibodies."

Dr. Rosenthal continued, "For our progranulin franchise, we recently reported the patient baseline characteristics for our INFRONT-3 Phase 3 clinical trial of latozinemab in frontotemporal dementia with a progranulin gene mutation, suggesting a representative study population in this indication. We are approaching a top-line data readout for INFRONT-3 in late 2025/early 2026. Additionally, PROGRESS-AD, the Phase 2 trial of AL101/GSK4527226 in early Alzheimer’s disease, has reached more than one-third of its target enrollment. It is an exciting time at Alector, and we are well positioned to advance our proprietary pipeline of novel immuno-neurology drugs."

Sara Kenkare-Mitra, Ph.D., President and Head of Research and Development at Alector, added, "We are actively advancing our Alector Brain Carrier, ABC, a proprietary versatile blood-brain barrier technology, and we are strategically leveraging this platform across our portfolio. We believe our ABC technology may advance treatment of neurodegenerative diseases by potentially enabling improved delivery of our therapeutics to the brain."

Recent Clinical Updates

Immuno-Neurology Portfolio
TREM2 Program (AL002) Being Developed in Collaboration with AbbVie

The results of the INVOKE-2 Phase 2 clinical trial of AL002 are expected in 2024. INVOKE-2, a randomized, double-blind, placebo-controlled, dose-ranging study, is designed to assess the efficacy and safety of AL002 in slowing disease progression in individuals with early Alzheimer’s disease (AD). AL002 is a novel investigational humanized monoclonal antibody (mAb) that is designed to bind to TREM2 to increase TREM2 signaling and, thereby, is hypothesized to improve the functionality of microglia. It is the most advanced TREM2 agonist product candidate in clinical development worldwide.
98% of eligible participants who completed the planned treatment period of INVOKE-2 have elected to participate in the long-term extension (LTE) study.
In October 2024, Alector published a manuscript titled "Preclinical and first-in-human evaluation of AL002, a novel TREM2 agonistic antibody for Alzheimer’s disease" in Alzheimer’s Research & Therapy. The publication outlines preclinical and INVOKE-1 Phase 1 study results, demonstrating that AL002 engaged TREM2, and induced pharmacodynamic biomarker changes associated with microglial proliferation, survival, and phagocytic activity in a dose-dependent manner.
AbbVie has an exclusive option to globally develop and commercialize AL002. Alector will deliver a data package resulting from the INVOKE-2 study to AbbVie for their evaluation. AbbVie’s exercise of its option would prompt a $250 million payment to Alector.
Progranulin Programs (latozinemab (AL001) and AL101/GSK4527226) Being Developed in Collaboration with GSK

The pivotal, randomized, double-blind, placebo-controlled INFRONT-3 Phase 3 clinical trial of latozinemab targeting frontotemporal dementia with a progranulin gene mutation (FTD-GRN) is ongoing and on track, with enrollment completed in October 2023 and a treatment duration of 96 weeks. Latozinemab is a novel investigational human mAb that aims to increase progranulin (PGRN) levels by inhibiting sortilin and is the most advanced PGRN-elevating candidate in development for the treatment of FTD-GRN.
In September 2024, Alector presented a poster highlighting the patient baseline characteristics for INFRONT-3 at the 14th International Conference on Frontotemporal Dementias (ISFTD 2024). Notably, the baseline characteristics of symptomatic INFRONT-3 participants, including age, Clinical Dementia Rating scale plus National Alzheimer’s Disease Coordinating Center Frontotemporal Lobar Degeneration Sum of Boxes (CDR plus NACC FTLD-SB) score and neurofilament light chain (NfL) levels, were representative of the broader FTD-GRN registry population, based on available registry data. Additionally, Alector shared findings from the FTD Caregiver Survey and FTD Insights Survey, highlighting the challenges faced by caregivers of individuals living with FTD.
PROGRESS-AD, a global, randomized, double-blind, placebo-controlled Phase 2 clinical study evaluating AL101/GSK4527226 in early AD has reached more than one-third of its target enrollment of 282 participants, with dosing initiated in February 2024. AL101 is an investigational human mAb designed to block and downregulate the sortilin receptor to elevate the level of PGRN in the brain in a manner that is similar to investigational latozinemab but with different pharmacokinetic and pharmacodynamic properties.
Early Research Pipeline

Alector continues to advance its Alector Brain Carrier (ABC), a proprietary, versatile blood-brain barrier technology platform, which is being applied selectively to the company’s next-generation product candidates and research pipeline. The technology platform enables customization of affinity, valency, and format to optimize effector function and half-life in preclinical models. Alector is applying its ABC technology, combined with its expertise in immuno-neurology, to work on novel targets and develop first or best-in-class therapeutics.
Third Quarter 2024 Financial Results

Revenue. Collaboration revenue for the quarter ended September 30, 2024, was $15.3 million, compared to $9.1 million for the same period in 2023. The increase was mainly due to an increase in revenue recognized for the AL002 program.

R&D Expenses. Total research and development expenses for the quarter ended September 30, 2024, were $48.0 million, compared to $46.3 million for the quarter ended September 30, 2023. The increase was mainly driven by the increase in research and development expenses for the AL101 programs resulting from the initiation of the PROGRESS-AD Phase 2 clinical trial in 2024.

G&A Expenses. Total general and administrative expenses for the quarter ended September 30, 2024, were $15.8 million, compared to $13.4 million for the quarter ended September 30, 2023. The increase was mainly due to the impairment of the right-of-use asset and the leasehold improvements as the Company transitioned operations from its laboratory and office space in Newark to its South San Francisco headquarters.

Net Loss. For the quarter ended September 30, 2024, Alector reported a net loss of $42.2 million, or $0.43 per share, compared to a net loss of $44.5 million, or $0.53 per share, for the same period in 2023.

Cash Position. Cash, cash equivalents, and investments were $457.2 million as of September 30, 2024. Management expects that this will be sufficient to fund current operations through 2026.

2024 Guidance. The Company continues to anticipate collaboration revenue to be between $60 million and $70 million, total research and development expenses to be between $210 million and $220 million, and total general and administrative expenses to be between $60 million and $70 million.

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.