On March 26, 2024 The Swedish biotech company Neogap Therapeutics reported to have received SEK 5 million from Eurostars in a collaboration project with the French biotech company Okomera (Press release, Neogap Therapeutics, MAR 26, 2024, View Source,c3951209 [SID1234641451]). The funding aims to enhance the development of an analysis method for T-cell activity against the patient’s tumour.

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Neogap is developing pTTL, personalised Tumour Trained Lymphocytes, a tailored cell therapy that utilises modified proteins, known as neoantigens, to activate the immune system specifically against cancer cells. The therapy is currently being assessed in phase I/II clinical trials for the treatment of colorectal cancer.

As part of the Eurostars-funded project, Neogap collaborates with the French biotech company Okomera, which has developed an innovative method to analyse cell interactions. The method allows for the analysis of how T-cells kill tumour cells in what is known as a killing assay, requiring only a minimal quantity of the cell product. This analysis is set to deepen the understanding of the interactions between T-cells and tumour cells, potentially enabling predictions about the efficacy of Neogap’s cell therapy products. The goal is to develop a system that can easily and reliably measure the capability of Neogap’s T-cell product, pTTL, in eradicating cancer cells.

"We are very grateful for Eurostars’ backing, which not only recognises the scientific potential of our company but also reinforces our role as an innovator in analysis methods for next-generation cell therapies. The funding enables further research that is essential for advancing our cell therapy product to later clinical phases," says Samuel Svensson, CEO of Neogap Therapeutics.

"Through our collaboration with Neogap, we’re afforded the opportunity to integrate Okomera’s technology into developing analytical methods for their pioneering cancer therapies. We eagerly anticipate the advancements this partnership will bring to the field of precision medicine," says Sidarth Radjou, CEO of Okomera.

On March 26, 2024 MEI Pharma, Inc. (Nasdaq: MEIP), a clinical-stage pharmaceutical company evaluating novel drug candidates to address known resistance mechanisms to standard-of-care cancer therapies, reported initiation of enrollment in a 12-patient expansion cohort in the ongoing Phase 1 study evaluating voruciclib, an investigational selective oral cyclin-dependent kinase 9 ("CDK9") inhibitor, in combination with venetoclax (Venclexta), a B-cell lymphoma 2 ("BCL2") inhibitor, in relapsed and refractory ("R/R") acute myeloid leukemia ("AML") patients (Press release, MEI Pharma, MAR 26, 2024, View Source [SID1234641471]). The Safety Review Committee recommended initiating the expansion cohort after observing anti-leukemic activity in multiple heavily pretreated patients in the dose escalation cohorts, including responses, anticipated decreases in myeloid leukemia cell differentiation protein ("Mcl-1") in available patient samples, no overlapping toxicity or dose limiting toxicities, and favorable safety results to date.

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"MCL-1 overexpression has been associated with a poor prognosis and development of resistance to BCL-2 inhibition by venetoclax in patients with AML and CLL. Voruciclib is a potent, oral CDK9 inhibitor that indirectly also suppresses MCL-1. We are participating in the ongoing multicenter phase 1 study, where preliminary results are demonstrating good treatment tolerance and safety to date," said Yesid Alvarado-Valero, M.D., Associate Professor, Department of Leukemia, University of Texas MD Anderson Cancer Center and study chair of the combination therapy stage of the Phase 1 study. "When Voruciclib is used in combination with venetoclax, the combination appears to have no added toxicity, in addition there is evidence of synergistic, early clinical activity, with disease responses, in a group of heavily pretreated acute myeloid leukemia patients."

"Increasingly, venetoclax is being used as a standard treatment in patients with AML, but resistance to salvage therapy after venetoclax use is common and yields limited benefit upon relapse; only about 10% of patients respond to salvage therapy after venetoclax failure, representing a significant need for patients with AML," said Richard Ghalie, M.D., chief medical officer of MEI Pharma. "We see the voruciclib data to date demonstrating anti-leukemic activity as promising, particularly alongside the consistent reductions of Mcl-1 that provide evidence we are eliciting the anticipated biological response in patients, and we are excited to share additional updates as appropriate in the second half of 2024."

Dr. Ghalie continued: "As we enroll the expansion cohort evaluating the potential of voruciclib in combination with venetoclax among a larger group of patients, I would like to thank and recognize the continued engagement of our investigators, and the participation of the patients enrolling in this study."

Phase 1 Study Details

The Phase 1 study is a multiple stage, open-label, 3+3 dose escalation and expansion study evaluating voruciclib, an oral CDK9 inhibitor, as a monotherapy and in combination with venetoclax, a BCL2 inhibitor. The first stage of the study evaluated the dose and schedule of voruciclib as a single-agent in patients with AML or B-cell malignances after failure of standard therapies. This stage is complete.

The second stage of the study, evaluating voruciclib in combination with standard dose venetoclax in patients with R/R AML, has completed enrollment in the dose escalation cohorts evaluating seven voruciclib dose levels from 50 mg every other day to 300 mg daily for two weeks in a four-week cycle. The study is currently enrolling a 12-patient expansion cohort evaluating voruciclib administered at 300 mg daily for two weeks in a four-week cycle in combination with venetoclax. Considering the tolerability results for the combination to date, another arm of the study will evaluate escalating doses of voruciclib administered over three weeks in a four-week cycle in combination with venetoclax to increase dose intensity and potentially optimize patient response.

A total of 29 patients with R/R AML, median age 67 years (range 34-89), enrolled in the dose escalation stage of the study evaluating voruciclib in combination with venetoclax. These patients were generally heavily pretreated; the median number of prior therapies was 3 (range 1-7), and 15 (52%) patients had ≥3 prior lines. Almost all patients (28/29) were treated with venetoclax in an earlier line of therapy. Additionally, 21 (72%) patients were noted as being in an adverse 2017 ELN Risk Category due to adverse cytogenetics and molecular mutations.

The primary objectives of the study are to determine the safety and biologic effective dose of voruciclib monotherapy or voruciclib in combination with venetoclax. Secondary objectives of the study include assessing the preliminary efficacy, pharmacokinetics, pharmacodynamics, and biomarkers of voruciclib monotherapy or voruciclib in combination with venetoclax.

Voruciclib Plus Venetoclax Combination: Initial Safety and Tolerability Data

Voruciclib at doses up to 300 mg administered on 14 consecutive days in a 28-day cycle in combination with standard dose venetoclax was well tolerated with no dose limiting toxicities observed. The maximum tolerated dose of voruciclib administered on this schedule with venetoclax has not been established. There were no discontinuations due to drug-related adverse events. No evidence of overlapping toxicity has been observed to date. The most common (≥5% of patients) grade 3 adverse events were myelosuppression associated with AML. Only 1 patient was observed as having a non-hematologic grade 3 drug-related adverse event (diarrhea).

Voruciclib Plus Venetoclax Combination: Initial Efficacy Data

In the 20 patients administered voruciclib at a dose of 100 mg or more, three patients achieved a response, including two patients that achieved a complete response with incomplete hematologic recovery (CRi) and one patient that achieved a morphologic leukemia-free state (MLFS), in each case having received venetoclax in an earlier line of treatment. Responses lasted 7 months in one patient, 5 months and ongoing in the second patient, and the third patient was referred to stem cell transplant. Further, an additional 14 patients had stable disease which lasted more than 90 days in 5 patients.

In the patients administered voruciclib at a dose of 100 mg or more, initial results from correlative biomarker assay studies of available samples from patients treated with the combination demonstrate the anticipated decrease of Mcl-1. Further, the available assays from the dose escalation cohorts demonstrated dose proportional decreases in Mcl-1. Reductions in Mcl-1 are consistent with the known mechanism of action of CDK9, which regulates Mcl-1.

About Voruciclib

Voruciclib is an investigational orally administered cyclin-dependent kinase 9 ("CDK9") inhibitor with potential to treat both hematological malignancies and solid tumors. It is in clinical development for acute myeloid leukemia and B-cell malignancies. Applications in solid tumors are also being considered.

The CDK family of proteins are important cell cycle regulators responsible for the control of cell proliferation, differentiation, apoptosis, and DNA repair. CDK9, one of several members of the CDK family of proteins, functions as a gene transcription controller and is also involved in regulating protein degradation. Specifically, CDK9 is a promising target to treat a range of cancers because of its role in controlling two other proteins often dysregulated in cancerous cells: myeloid leukemia cell differentiation protein ("Mcl-1") and the MYC proto-oncogene protein ("MYC").

Mcl-1 is a member of the family of anti-apoptotic proteins which, when elevated, may prevent the cell from undergoing cell death. Inhibition of CDK9 blocks the production of Mcl-1, which is an established resistance mechanism to the B-cell lymphoma 2 ("BCL2") inhibitor venetoclax (marketed as Venclexta).

MYC regulates cell proliferation and growth. Upregulation of MYC is implicated in many human cancers and is frequently associated with poor prognosis and unfavorable patient survival. CDK9, in addition to being a transcription factor for MYC, also decreases phosphorylation of MYC protein that is implicated in stabilizing MYC in KRAS mutant cancers. Targeting MYC directly has historically been difficult, but CDK9 is a promising approach to target this oncogene.

On March 26, 2024 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated oral targeted agents to treat hematologic malignancies, reported financial results for the three months and year ended December 31, 2023, and provided a corporate update (Press release, Aptose Biosciences, MAR 26, 2024, View Source [SID1234641436]).

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"The data we have generated from tuspetinib thus far – as a single agent and in combination therapy with venetoclax in relapsed and refractory AML – have demonstrated a distinctly favorable safety profile and broad activity for tuspetinib across mutational subtypes. This profile also extends to FLT3 wildtype AML, which represents the majority of AML patients, and in which few agents have shown such broad activity," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "These data have propelled us to initiate a clinical study of tuspetinib in a triplet combination with venetoclax and azacitidine in frontline therapy for newly diagnosed AML, including both FLT3 wild type and FLT3 mutated subtypes."

Key Corporate Highlights

Aptose Completes Public Offering – On January 30, 2024, Aptose closed a public offering of 5,649,122 common shares of the Company and warrants at a combined offering price of US $1.71 per share. This included 736,842 Common Shares and warrants pursuant to a full exercise by the underwriter of its over-allotment option. Total gross proceeds from the public offering were approximately $9.7 million before deducting underwriting costs, placement agent commissions and other offering-related expenses.

Private Placement – On January 31, 2024, Aptose closed a US $4 million private placement of common shares with strategic partner Hanmi Pharmaceutical. Under the terms of the strategic investment, Hanmi purchased each common share at a price of US $1.90, representing an 11% premium over the price of the common shares issued as part of the public offering. The Company also issued Hanmi warrants to purchase common shares at an exercise price of US $1.71 per warrant share. Total gross proceeds from the private placement were approximately $4 million, excluding underwriting discounts, placement agent commissions and other offering-related expenses.

Tuspetinib Advancing to Triplet Therapy Pilot Study – Tuspetinib (TUS), a once daily oral agent with a unique kinase targeting pattern, is being developed for the treatment of patients with acute myeloid leukemia (AML). More than 170 patients to date received TUS alone or in combination with the BCL-2 inhibitor venetoclax (VEN) during the Phase 1/2 clinical program in the very ill relapsed or refractory (R/R) AML patient population. At the single agent 80 mg recommended Phase 2 dose, TUS achieved a favorable safety profile and a CR/CRh rate of 36% among patients who were naive to VEN. The safety profile of TUS remained favorable when TUS was combined with VEN in R/R AML patients, and responses were achieved in both patients naive to VEN and those who failed prior therapy with VEN. TUS avoids many typical toxicities observed with other agents and achieves broad activity across AML patients with a diversity of adverse genetics. Tuspetinib is now being advanced to a triplet combination therapy of tuspetinib, venetoclax and a hypomethylating agent (TUS/VEN/HMA) for the frontline treatment of newly diagnosed AML patients ineligible for induction chemotherapy.

Luxeptinib G3 Evaluation Completed – During 2023 and early 2024, clinical evaluation of the new generation 3 (G3) formulation of luxeptinib (LUX) was completed. The G3 formulation was tested in a single dose bioavailability study in 20 patients, including both B-cell cancer and AML patients, and across 5 dose levels (10mg to 200mg). The G3 formulation then was evaluated in R/R AML patients with continuous dosing using two different dose levels (50mg BID and 200mg BID) in a total of 11 patients. Data show the G3 formulation dosed at 200mg twice daily can achieve 2-3uM steady state plasma levels, with approximately 10-fold better absorption, and interestingly even better tolerability, than the original G1 formulation. Thus, the G3 formulation achieved the desired plasma exposure benchmark and can serve as the formulation of choice for future studies with LUX. Aptose is exploring alternative development paths and collaborations to advance LUX as a single agent or in combination with VEN to treat defined R/R patient populations of high unmet need.
Multiple Planned Value-creating Milestones Ahead

TUS/VEN doublet synopsis in R/R AML: EHA (Free EHA Whitepaper) 2024
TUS/VEN/HMA planned initiation of pilot triplet study in 1L AML: Summer 2024
Triplet pilot dose escalation planned with early CR/MRD/safety data in 1L AML: ASH (Free ASH Whitepaper) 2024
Triplet pilot completed with CR/MRD data and dose selection: EHA (Free EHA Whitepaper) 2025
Triplet initiation of Ph2/Ph3 pivotal program: 2H 2025
FINANCIAL RESULTS OF OPERATIONS

Balance Sheet Data
(unaudited)
($ in thousands)

December 31, December 31,
2023 2022
Cash, cash equivalents and short-term investments $ 9,252 $ 46,959
Working capital (3,375 ) 37,235
Total assets 12,989 51,027
Non-current liabilities 621 1,002
Deficit (515,537 ) (464,330 )
Total shareholders’ equity (2,901 ) 37,741

Cash and cash equivalents, January 31, 2024 (unaudited) was $18.6 million, after gross proceeds from January 2024 financings (unaudited) of $13.7 million.
Total cash and cash equivalents and investments as of December 31, 2023, were $9.3 million, a decrease of $37.7 million as compared to $47.0 million at December 31, 2022. Based on current operations, the Company expects that cash on hand and available capital provide the Company with sufficient resources to fund planned Company operations including research and development through August of 2024.
Working capital is a non-GAAP measure and represents cash, cash equivalents, investments, prepaid expenses and other current assets less current liabilities.
Common shares issued and outstanding as at March 26, 2024 were 15,717,701.

Statements of Operations Data
(unaudited)

Year ended December 31,
(in thousands except per Common Share data) 2023
2022

Revenues $ - $ -
R&D, related party 3,492 3,556
Research and development expenses 33,273 24,532
General and administrative expenses 15,591 14,514
Net finance income 1,149 779
Net loss $ (51,207 ) $ (41,823 )
Basic and diluted loss per Common Share $ (7.58 ) $ (6.80 )
Weighted average number of common shares outstanding used in the calculation of basic loss per share 6,755 6,151

Net loss for the year ended December 31, 2023 increased by $9.4 million to $51.2 million, as compared to $41.8 million for the comparable period in 2022.

Research and Development Expenses

The research and development expenses for years ended December 31, 2023, and 2022 are as follows:

Twelve months ended
December 31,
(in thousands) 2023 2022

Program costs – Tuspetinib $ 24,925 $ 10,083
Program costs – Luxeptinib 3,510 8,426
Program costs – APTO-253 40 141
Personnel related expenses 6,878 7,181
Stock-based compensation 1,373 2,218
Depreciation of equipment 39 39
Total $ 36,765 $ 28,088

R&D expenses increased by $8.7 million to $36.8 million for the year ended December 31, 2023, as compared with $28.1 million for the comparative period in 2022. Changes to the components of our R&D expenses presented in the table above are primarily related to the following activities:

Program costs for tuspetinib increased by $14.8 million. The higher program costs for tuspetinib in 2023 represent the enrollment of patients in our APTIVATE clinical trial, our healthy volunteer trial, manufacturing activities to support clinical development, purchase of clinical trial materials, and related expenses.
Program costs for luxeptinib decreased by approximately $4.9 million, primarily due to lower clinical trial costs and lower manufacturing costs as a result of the current formulation requiring less API than the prior formulation.
Program costs for APTO-253 decreased by approximately $101 thousand due to the Company’s decision on December 20, 2021, to discontinue further development of APTO-253.
Personnel-related expenses decreased by $0.3 million due to lower headcount in 2023.
Stock-based compensation decreased by approximately $845 thousand in the year ended December 31, 2023, compared with the year ended December 31, 2022, primarily due to stock options granted with lower grant date fair values in the current period.
General and Administrative Expenses

General and administrative expenses consist primarily of salaries, benefits, and travel, including stock-based compensation for our executive, finance, business development, human resources, and support functions. Other general and administrative expenses and professional fees include auditing, and legal services, investor relations and other consultants, insurance. and facility related expenses.

We expect that our general and administrative expenses will increase for the foreseeable future as we incur additional costs associated with being a publicly traded company and to support our pipeline of activities. We also expect our intellectual property related legal expenses to increase as our intellectual property portfolio expands.

The general and administrative expenses for the years ended December 31, 2023 and 2022 are as follows:

Year ended December 31,
(in thousands) 2023 2022
General and administrative, excluding items below: $ 13,262 $ 11,444
Stock-based compensation 2,280 2,989
Depreciation of equipment 49 81
Total $ 15,591 $ 14,514

General and administrative expenses increased by approximately $1.1 million $15.6 million for the year ended December 31, 2023, as compared with $14.5 million for the comparative period in 2022. The increase was primarily as a result of higher salaries expenses, higher professional fees, and higher travel expenses, partly offset by a decrease in stock-based compensation costs.

Stock-based compensation decreased by $709 thousand mostly as a result of options having a lower grant date fair value as compared with the options granted in the comparative period.

Conference Call & Webcast:
Date: Tuesday, March 26, 2024
Time: 5:00 PM ET
Audio Webcast Only: link
Q&A Participant Registration Link*: link
(https://register.vevent.com/register/BIe69f08fc83634a6aa38bf7081d82c6a2)

*Analysts interested in participating in the question-and-answer session will pre-register for the event from the participant registration link above to receive the dial-in numbers and a unique PIN, which are required to access the conference call. They also will have the option to take advantage of a Call Me button and the system will automatically dial out to connect to the Q&A session.

The audio webcast also can be accessed through a link on the Investor Relations section of Aptose’s website here. A replay of the webcast will be available on the company’s website for 30 days.

The press release, the financial statements and the management’s discussion and analysis for the year ended December 31, 2023 will be available on SEDAR at www.sedar.com and EDGAR at www.sec.gov/edgar.shtml.

On March 26, 2024 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS’’ or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported topline data from the Phase 2a study of SLS009 and provides an update on Phase 3 REGAL Study of GPS in AML (Press release, Sellas Life Sciences, MAR 26, 2024, View Source [SID1234641452]). The Company will host a webinar to discuss the data and the REGAL update today at 8:15 am ET.

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"Completion of enrollment in the Phase 3 REGAL trial represents an important milestone in our goal to deliver GPS to AML patients," said Angelos Stergiou, MD, ScD h.c., President and Chief Executive Officer of SELLAS. "We are extremely grateful to the patients, their families, and investigators who have helped us achieve this significant milestone. Additionally, we are pleased to share that the Steering Committee has reviewed the study as of the March 1, 2024, cutoff date. As of this evaluation, 123 patients were enrolled with 66 of them discontinuing the treatment. In the trial, patients are recorded as having stopped the study treatment in cases of death for any reason, relapse, intolerable toxicity, or treatment completion. Regarding the GPS arm, we are pleased to report that we have not observed any intolerable toxicities in any patient population across all our clinical studies thus far, although toxicities are commonly observed with therapies used in the control arm. Therefore, almost all patients who are off treatment may have most likely either relapsed or passed away. The most frequent cause of death in this patient population is relapse. As the study sponsors, we lack specific information on the outcomes of these 66 patients, hindering our ability to confirm whether the required number of events for interim analysis – 60 – has been reached. The determination of such outcomes, the primary endpoint of the trial, lies within the purview of the IDMC, which is now scheduled to meet by the end of April."

The REGAL Steering Committee met on March 22, 2024, to discuss the study and believes the high number of patients who completed participation in the study signals that the interim analysis requiring 60 events may be imminent. The Committee also expressed its satisfaction with SELLAS’ overall clinical study conduct and complimented SELLAS for addressing such a debilitating and high unmet medical need as no drugs are approved in the AML CR2 maintenance setting.

Dr. Stergiou continued: "We are extremely excited to share positive topline data from the Phase 2a trial of SLS009 in AML patients resistant to venetoclax combination therapies. In the selected optimal dose regimen of 30 mg BIW a 50% response rate was achieved, far surpassing the targeted 20% rate. Notably, we identified promising biomarkers and observed a 100% response rate at the optimal dose level and a 57% response rate across all the levels tested in patients with those biomarkers. The SLS009 aza-ven treatment was well-tolerated and evoked anti-leukemic effects in 67% of patients across all levels dosed. The first patient who achieved a complete response continues on the study and remains leukemia-free 9 months post-enrollment. These compelling results from the Phase 2a reinforce our belief that SLS009 represents a potential breakthrough for relapsed and/or refractory AML patients, addressing one of the most urgent unmet medical needs."

Summary of Topline Data from Phase 2a data of SLS009 in AML

Patients Characteristics

As of March 15, 2024 data cutoff, 21 patients were treated
All patients were diagnosed with AML refractory to or relapsed after venetoclax containing regimens
20 out of 21 (95%) enrolled patients had adverse/high-risk cytogenetics and 1 patient (5%) had intermediate cytogenetics
Median age was 70 and 19/21 (90.5%) of patients were older than 60
Safety

SLS009 in combination with aza/ven has been well-tolerated at all tested dose levels
No dose-limiting toxicities (DLT) at any of the studied dose levels and no treatment-related high-grade (≥G3) toxicities were observed
Hematologic toxicities profile was consistent with aza/ven standalone treatment

Efficacy

A total of 21 patients were enrolled in the study as of March 15, 2024: 10 in the 45 mg safety cohort, 11 in the 60 mg cohort (2 x 30 mg twice a week or 60 mg once a week)

10% response rate in the 45 mg QW safety cohort (dose level below the recommended Phase 2 dose, RP2D)
20% response rate in the 60 mg QW cohort
50% response rate in the 60 mg, 2 x 30 mg BIW cohort
Observed strong anti-leukemic activity, defined as 50% or more bone marrow blast reduction in 67% of patients across all dose levels
Median survival rate has not been reached in any of the dose levels
The first patient enrolled in the study who achieved a complete response (CR) continues on the study and remains leukemia-free 9 months after enrollment
Biomarkers

During the trial the Company identified potential biomarkers currently undergoing testing as predictive markers in the most recent portion of the study
Patients with the identified biomarkers exhibited significantly higher response rates:
100% response rate at the optimal dose level (30 mg BIW)
57% response rate across all dose levels
Furthermore, the Company has clarified the proposed biological basis and mechanism of action for SLS009 activity in patients with these biomarkers
The relevant biomarkers are present in multiple hematologic and solid cancer indications, with a substantial proportion of patients exhibiting them in additional indications, ranging up to ~50% of patients in some indications
The Phase 2a clinical trial of SLS009 is an open-label, single-arm, multi-center study designed to evaluate the safety, tolerability, and efficacy of SLS009 in combination with aza/ven at two dose levels, 45 and 60 mg. In the 60 mg dose cohort patients were randomized into either a 60 mg dose once per week or a 30 mg dose two times per week. The target response rate at the optimal dose level is 20% with a target median survival over 3 months. In addition, the study aims to identify biomarkers for the target patient population and enrichment for further trials.

On March 26, 2024 Ubiquigent Limited (Ubiquigent), a drug discovery and development company harnessing novel deubiquitinase (DUB) modulators as new therapeutics for areas of high unmet medical need, reported an agreement with Debiopharm, a biopharmaceutical company aiming to develop tomorrow’s standard-of-care treatments to cure cancer and infectious diseases (Press release, Debiopharm, MAR 26, 2024, View Source [SID1234641472]). The agreement will support the development of Debiopharm’s USP1 inhibitor programme, Debio 0432.

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Under the terms of the agreement, Ubiquigent will deploy its DUB-focused platform, combined with its deep understanding of the DUB field, to develop novel target engagement assays to support Debio-0432 as it approaches the clinic.

Currently, in late-stage preclinical development, Debio 0432 is a small molecule with best-in-class potential that could be deployed to combat multiple tumour types. Through its potent and selective inhibition of USP1, a critical player in the DNA damage repair (DDR) pathway, Debio 0432 has the potential to induce synthetic lethality in tumour types with underlying defects of DNA repair genes.

"We are delighted to enter this agreement with Debiopharm, supporting the development of its advanced USP1 inhibitor programme. Following our successful collaborations with other clinical stage companies, this latest agreement further demonstrates the capability of Ubiquigent’s platform to address all aspects of DUB drug discovery and development, encompassing target validation, hit-to-lead, candidate selection, translational research, and the development of assays to support clinical evaluation." Jason Mundin, CEO of Ubiquigent, said: "With multiple assets now reaching clinical stage, we look forward to seeing the continued progression and expansion of the DUB field over the coming years as more companies enter the space and new therapeutics enter the clinic."

Bertrand Ducrey, CEO, Debiopharm, commented: "Ubiquigent’s specialised drug discovery platform is uniquely positioned to support our USP1 inhibitor programme as it approaches the clinic, enabling the development of novel target engagement assays. Selective inhibition of USP1 to interrupt DNA damage repair pathway is an exciting approach to cancer treatment."