Skip to main content
Category

Press Release

Apr 16
Love0

Glycomine Announces $115 Million Series C Financing to Advance Lead Drug Candidate, GLM101, into a Phase 2b Clinical Trial for PMM2-CDG

By Glycomine, Press Release, Private Companies
Press Release.

 

Glycomine Announces $115 Million Series C Financing to Advance Lead Drug Candidate, GLM101, into a Phase 2b Clinical Trial for PMM2-CDG

 

  • GLM101 is the first disease-modifying therapeutic in development to treat PMM2-CDG, the most common congenital disorder of glycosylation
  • Data from the ongoing Phase 2 open-label study of GLM101 provide clinical proof of concept, showing improvement in ataxia, a key burden of disease in PMM2-CDG
  • Funding will support advancement of GLM101 into a randomized, placebo-controlled Phase 2b safety and efficacy study

SAN CARLOS, Calif., April 16, 2025 – Glycomine, Inc., a biotechnology company focused on developing transformative new therapies for orphan diseases, today announced a $115 million Series C financing to advance its lead candidate, GLM101, into a Phase 2b clinical trial. The financing was led by CTI Life Sciences Fund, funds managed by abrdn Inc., and Advent Life Sciences, alongside continued investment from existing investors, Novo Holdings, Sanofi Ventures, Abingworth, RiverVest Venture Partners, Sanderling Ventures, Chiesi Ventures, Remiges Ventures, and Asahi Kasei Ventures.

“We are excited to partner with our new investors who have strong track records in rare diseases and for the continued support from our existing investors,” said Steve Axon, Glycomine’s CEO. “This financing will enable us to advance GLM101 into a randomized, placebo-controlled trial later this year—an important step toward bringing the first disease-modifying therapeutic to patients with PMM2-CDG.”

GLM101, a first-in-class mannose-1-phosphate replacement therapy, is in development for phosphomannomutase-2 congenital disorder of glycosylation (PMM2-CDG), a rare and life-threatening genetic disorder with no approved treatments. Glycomine has enrolled more than 20 patients across Europe and the U.S. in its ongoing Phase 2 study and recently initiated dosing in pediatric patients.

Data from Glycomine’s ongoing Phase 2 open-label study have demonstrated promising improvements in ataxia, a hallmark debilitating manifestation of PMM2-CDG. Among nine adult and adolescent patients, treatment with GLM101 led to an average 11.9-point improvement on the ICARS (International Cooperative Ataxia Rating Scale) over 24 weeks.

“We are impressed by the therapeutic approach and strong progress of the Glycomine team,” said Youssef Bennani, Ph.D., Managing Partner with CTI Ventures. “We are excited to be part of such a strong investor syndicate and look forward to the potential of making a positive impact in the lives of patients with PMM2-CDG.”

Dominic Schmidt, Ph.D., General Partner with Advent Life Sciences added, “We are highly encouraged by the clinical signal observed in the ongoing Phase 2 study. Most notably, the data show strong potential for clinically meaningful improvement in ataxia, a key driver of disease burden for PMM2-CDG patients.”

In connection with the financing, Drs. Bennani and Schmidt have been appointed to Glycomine’s Board of Directors.

 About PMM2-CDG

 Phosphomannomutase 2-congenital disorder of glycosylation (PMM2-CDG), previously known as CDG-1a, is the most prevalent congenital disease of glycosylation. PMM2-CDG is caused by a genetic mutation in phosphomannomutase 2 (PMM2), which results in the protein having reduced activity. PMM2 is an enzyme that converts mannose-6-phosphate to mannose-1-phosphate, which is required to insert the mannose sugar building block into developing glycans that are crucial for proper protein structure and function. The deficiency of mannose-1-phosphate disrupts the process of N-glycosylation and causes a wide array of clinical symptoms and, in many cases, can be life-threatening.

 About Glycomine, Inc.

Glycomine is a clinical-stage biotechnology company that is advancing treatments for serious rare diseases for which no other therapeutic options exist. The Company’s lead investigational drug candidate GLM101 is a mannose-1-phosphate replacement therapy in development to treat PMM2-CDG. GLM101 is designed to deliver mannose-1-phosphate into cells and thereby bypass disease-causing PMM2 mutations to restore pathway function. GLM101 has received Orphan Drug Designation in the U.S. and E.U. and Rare Pediatric Disease Designation and Fast Track Designation in the U.S. The company is based in San Carlos, California, and supported by leading international life sciences investors. For more info visit www.glycomine.com.

 Corporate Contact: Peter McWilliams, Ph.D., info@glycomine.com

Media Contact: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com

 

Mar 24
Love0

Positive Data from Phase 1 Trial of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) Presented at the 40th Annual European Association of Urology Congress

By Aura Biosciences, Press Release, Private Companies
Press Release.

 

Positive Data from Phase 1 Trial of Bel-sar in Patients with Non-Muscle-Invasive Bladder Cancer (NMIBC) Presented at the 40th Annual European Association of Urology Congress
March 24, 2025

Clinical Complete Responses Observed with Single Low-Dose of Bel-sar in Patients with Intermediate and High-Risk NMIBC with Robust Cell-Mediated Immunity and Urothelial Field Effect

Favorable Safety Profile; Only Grade 1 Drug-Related Adverse Events Reported in Less Than 10% of Patients

Data Supports Potential for a Paradigm Shifting Approach in the Front-Line Treatment of patients with NMIBC

Aura Hosting Virtual Urologic Oncology Investor Event with Key Opinion Leaders Today at 4:30 pm Eastern Time

BOSTON, March 24, 2025 (GLOBE NEWSWIRE) — Aura Biosciences, Inc. (NASDAQ: AURA), today announced positive data from a completed Phase 1 window of opportunity trial of bel-sar (AU-011) in patients with NMIBC. These data were presented by Dr. Seth Lerner as a late breaker presentation at the 40th Annual European Association of Urology (EAU) Congress, held in Madrid, Spain.

The trial included 15 patients with NMIBC, with the primary endpoints of evaluating the safety and feasibility of local administration of bel-sar alone (n=5) and bel-sar with light activation (n=10). The trial also evaluated tumor necrosis and shrinkage as an indication of biological activity as well as immune mediated changes in the tumor microenvironment (TME). All patients that received bel-sar without light activation (n=5) had intermediate-risk NMIBC, and no patients had a clinical complete response or tumor shrinkage after treatment. In NMIBC patients that received bel-sar with light activation (n=10), 5 patients had intermediate-risk disease and 5 patients had high-risk disease. In the group of patients with intermediate-risk NMIBC (n=5), 4 out of 5 patients demonstrated a clinical complete response of the target lesion with no tumor cells detected in histopathological evaluation and necrosis was observed in 3 out of 5 patients. In the group of patients with high-risk disease (n=5), 1 out of 5 patients demonstrated a clinical complete response and 3 out of 5 patients demonstrated visual tumor shrinkage on cystoscopy. Furthermore, in the light activated cohorts, 4 out of 7 patients (57%) with multiple tumors demonstrated a clinical complete response in at least one non-target tumor with infiltration of effector CD8+ and CD4+ T-cells. Initial multiplex immunofluorescence staining of biopsies after bel-sar treatment in 3 patients revealed significant infiltration of cytotoxic effector cells, as well as de novo formation of tertiary lymphoid structures in both target and non-target lesions. These results support a robust anti-tumor immune response consistent with the observed clinical complete response and urothelial field effect.

“The clinical complete responses and the immune responses seen with a single low-dose of bel-sar in such a short time frame are highly encouraging. This reinforces our belief that this novel mechanism of action could be the key to generating long term durable responses,” said Sabine Brookman-May, MD, FEBU, Senior Vice President, Therapeutic Area Head Urologic Oncology of Aura Biosciences. “We look forward to our virtual urologic oncology investor event, where we will outline the Phase 1b/2 trial to further evaluate bel-sar’s clinical activity and future development plans.”

“The positive data presented at EAU are compelling and suggest that bel-sar has the potential to introduce a new front-line focal treatment approach instead of or ahead of TURBT in patients across different risk categories,” said Seth Lerner, MD, Scott Department of Urology, Dan L. Duncan Comprehensive Cancer Center, Baylor College of Medicine. “With its favorable safety profile and the ability to be administered without general anesthesia, this novel modality could expand treatment options for patients and may represent a shift in how we approach NMIBC management.”

Trial Design: The completed Phase 1 window of opportunity trial (NCT05483868) is a two-part, open-label clinical trial, designed to assess the safety and feasibility of bel-sar in NMIBC. The study treatment was administered 7 to 12 days before the scheduled transurethral resection of bladder tumor (TURBT), the standard of care procedure. The participants were followed for safety monitoring over a 56-day period. The trial evaluated bel-sar’s biological activity with histopathological evaluation of tissue samples collected at the time of prior scheduled TURBT (regardless of tumor response) with evaluation of focal necrosis and immune changes in the TME as secondary endpoints, as well as visual assessment of tumor shrinkage. The Phase 1 window of opportunity trial is now complete, with Part 1 (n=5) patients receiving a single bel-sar dose without light activation and Part 2 (n=10) patients with a confirmed tumor at time of treatment, receiving either 100ug or 200ug of bel-sar as a single dose. Of these 10 patients, 5 had intermediate-risk disease and 5 had high-risk disease at the time of treatment. Eight of these 10 patients had a history of recurrent bladder cancer and had undergone multiple TURBTs and adjuvant treatments such as Bacillus Calmette-Guerin (BCG), mitomycin, gemcitabine, cetrelimab, and tamoxifen prior to trial enrollment.

Safety Data: Bel-sar was well-tolerated, with drug-related Grade 1 events reported in less than 10% of patients. No Grade 2 or higher drug-related adverse events and no serious adverse events were reported. No significant differences between the light-activated and non-light activated cohorts were observed.

Biological Activity: The data in the 10 patients that received bel-sar with light activation showed clinical activity detectable as soon as 7 days after a single low dose of bel-sar with light activation. This was demonstrated by histopathological evidence of clinical complete response, necrosis, immune activation or visual tumor shrinkage observed on cystoscopy. In contrast, no clinical activity was seen in the 5 patients receiving bel-sar with no light activation. For this analysis, “clinical complete response” was defined as the absence of tumor cells on histopathologic evaluation. Four out of 5 patients with intermediate-risk disease exhibited a clinical complete response of the target lesion with no tumor cells detected in histopathological evaluation post-treatment. In addition, visual tumor shrinkage was observed in several non-target tumors on cystoscopy. One of the patients with high-risk disease (based on BCG failure) had a clinical complete response, and 3 out of 5 patients with high-risk disease demonstrated visual tumor shrinkage on cystoscopy. Immune activation was noted in all patients (n=10) in both target and non-target bladder tumors. Four out of 7 patients with multiple tumors (57%) demonstrated a clinical complete response in at least one non-target tumor with infiltration of effector CD8+ and CD4+ T-cells. This data provides evidence of a bladder urothelial field effect with a single low-dose of bel-sar with light activation, potentially indicating a broader immune response and immune surveillance in the bladder beyond the target tumor in these patients.

Immune Profiling: To evaluate the local immune response after the treatment with bel-sar in the TME, multiplex immunofluorescence staining for key immune cell types was performed on tumor biopsies. Initial post-treatment results from 3 patients showed significant infiltration per unit area of cytotoxic effector cells demonstrating early activation of both innate and adaptive immunity (Natural Killer cells, CD4+ and CD8+ T cells). In addition, de novo formation of mature tertiary lymphoid structures (TLS) post-treatment was observed in 2 of these 3 participants. Early TLS were also observed in distant, non-target lesions, suggestive of an immune mediated urothelial field effect. These early observations showing induction of effector immunity and the development of local active immunosurveillance, highlight key features of bel-sar’s dual mechanism of action and the potential to translate into durable treatment responses.

Virtual Urologic Oncology Investor Event

Aura will host a virtual urologic oncology investor event today, at 4:30 pm Eastern Time, featuring Neal Shore, MD, FACS (Carolina Urologic Research Center), Gary Steinberg, MD, FACS (Rush University) and Jennifer A. Linehan, MD (Saint John’s Cancer Institute), who will join company management to discuss the data from the Phase 1 trial.

At the event, Aura will also provide an update on the bladder cancer development program, including the Phase 1b/2 trial and future development plans. A live question and answer session will follow the discussion. To register for the event, click here.

The live webcast of Aura’s virtual urologic oncology investor event will be available on the “Investors & Media” page under the “Events & Presentations” section of Aura’s website at https://ir.aurabiosciences.com/events-and-presentations, where a replay of the webcast will be archived for 90 days following the presentation date.

About Aura Biosciences

Aura Biosciences is a clinical-stage biotechnology company focused on developing precision therapies for solid tumors that aim to preserve organ function. Our lead candidate, bel-sar (AU-011), is currently in late-stage development for primary choroidal melanoma and in early-stage development in other ocular oncology indications and bladder cancer. Aura Biosciences is headquartered in Boston, MA. Our mission is to grow as an innovative global oncology company that positively transforms the lives of patients.

For more information, visit aurabiosciences.com. Follow us on X (formerly Twitter) @AuraBiosciences and visit us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions can be used to identify forward-looking statements. These forward-looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including bladder cancer; statements regarding Aura’s plans and expectations for its ongoing and future clinical trials of bel-sar in bladder cancer; statements regarding Aura’s beliefs and expectations for bel-sar’s ability to provide durable responses in bladder cancer patients; statements regarding the timing and content of Aura’s virtual urologic oncology investor event; statements regarding Aura’s expectations for an improved quality of life of patients after treatment with bel-sar and changes to the treatment paradigm for patients; and statements regarding the timing of the announcement of bladder cancer program updates, including the Phase 1b/2 trial and future development plans.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s preclinical and clinical trials may not be predictive of future results in connection with future clinical trials; the risk that early or interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; the risk that governmental authorities may disagree with Aura’s clinical trial designs, even where Aura has obtained agreement with governmental authorities on the design of such trials, such as the Phase 3 special protocol assessment agreement with the U.S. Food and Drug Administration; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; Aura’s ongoing and planned preclinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the United States Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

Investor and Media Contact:

Alex Dasalla
Head of Investor Relations and Corporate Communications
IR@aurabiosciences.com

Mar 07
Love0

Artax Biopharma presents new preclinical data on Nck modulators at 2025 AAD Annual Meeting

By Artax Biopharma, Press Release, Private Companies
Press Release.

 

Artax Biopharma presents new preclinical data on Nck modulators at 2025 AAD Annual Meeting

 

Nck modulator reduced evidence of inflammation in an animal model of dermal irritation

Compelling preclinical data support continued development of Nck modulators in atopic dermatitis

 

Cambridge, MA, March 7, 2025 – Artax Biopharma, Inc., a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, today announced the presentation of new preclinical data in atopic dermatitis models with Nck modulators, in an ePoster with oral presentation at the 2025 American Academy of Dermatology Annual Meeting, taking place March 7-11, in Orlando, Florida.

“These preclinical results highlight the potential breadth and depth of Nck modulation, a completely novel mechanism which we believe has the potential to achieve true immunomodulation for a broad range of autoimmune conditions,” said Dr. Rob Armstrong, Chief Executive Officer of Artax Biopharma. “On the heels of our recently announced positive Phase 2a data in patients living with psoriasis, we look forward to continuing to evaluate the potential of Nck modulation to impact TCR signaling activation in response to self-antigens and offer an alternative to broad immunosuppression for patients with serious autoimmune diseases.”

ePoster presentation

“TCR-Nck Modulators: Pioneering Oral Modulation of T Cell Receptor Activation Holding the Promise of Treating Dermatologic Diseases,” Christopher L. VanDeusen, PhD; Shannon Dwyer; D. Scott Batty Jr, MD; Aldo Borroto, PhD; Andrés P Gagete, PhD; Robert W. Armstrong, PhD; Balbino Alarcón, PhD​​

Dysregulated TCR signaling leads to T-cell-mediated ​autoimmune diseases. Nck modulators specifically block Nck recruitment upon TCR activation, correcting erroneous activation. Nck plays a fundamental role in increased TCR signaling triggered by self-antigens,​ which is a key upstream event in autoimmune diseases​. Nck modulators act by modulating T cell activation, rather than broadly suppressing it. Key findings include:

  • Dose-dependent decrease in dust mite induced cytokine production from peripheral blood mononuclear cells (PBMCs) isolated from an atopic dermatitis patient.
  • Treatment of healthy PBMCs with AX-158 resulted in a dose-dependent reduction in cytokine release across all T helper cell subtypes including IL-17, IL-4 and IL-13.
  • Oral Nck modulator AX-194 significantly reduced ​the appearance of inflammation in an imiquimod model of dermal irritation, as demonstrated by reduction in the total number of hematopoietic
    cells and inflammatory macrophages in the skin.

Based on this preclinical evidence, ​the Artax team is planning further studies in additional autoimmune diseases to explore the potential of Nck modulators to correct erroneous T cell activation in dermal autoimmune disorders.

About Artax Biopharma

Artax Biopharma is a clinical-stage biotechnology company transforming the treatment of T Cell-driven autoimmune diseases. Artax’s first-in-class oral small molecules aim to deliver immune system modulation without immunosuppression, potentially unlocking new treatment options as both monotherapy and in combination with other treatments. Artax Biopharma is based in the Boston area and raised funding from investors including Advent Life Sciences, Sound Bioventures, The Termeer Family Office, the Fuhrer Family Office and Columbus Venture Partners. For more info, see www.artaxbiopharma.com or follow us on LinkedIn.

 

About Nck modulation

We believe there is significant potential for Nck modulation to revolutionize treatment of T Cell-driven diseases. Immunomodulation maintains healthy control of the immune system, while addressing the underlying source of T Cell-driven diseases. Central to a well-functioning immune system is the T Cell Receptor (TCR). When TCR signaling becomes dysregulated, it causes T Cell-driven conditions, including autoimmune diseases. We believe the immunomodulation mechanism offered by our investigational agents holds broad potential to revolutionize how these T Cell-driven autoimmune diseases are addressed, while not impairing the ability of a patient’s immune system to function properly.

 

AX-158, our lead Nck modulator, has shown strong, broad cytokine modulation as well as modulation of mixed lymphocyte reactions. Good data on therapeutic efficacy with AX-158 were observed in murine models of self-antigen activation (EAE), with a prolonged pharmacodynamic effect in EAE, suggesting durable immune modulation. AX-158 showed no immunosuppression in models of strong antigen stimulation. Studies with AX-158 showed substantial preclinical evidence of activity in the Th2, Th17, Th1/Th0 pathways, suggesting that applications could be quite broad across the autoimmune space.

 

For future clinical study inquiries: contact@artaxbiopharma.com.

                                                                #  #  # 

 

Contacts: 

Maria Nichol, DPhil, EPA, CPA 

Chief Business Officer 

mnichol@artaxbiopharma.com 

 

Media: 

Madelin Hawtin

LifeSci Communications

mhawtin@lifescicomms.com

Feb 19
Love0

Epitopea Announces License and Research Collaboration Agreement with MSD to Identify CryptigenTM Tumor-Specific Antigens

By Epitopea, Press Release, Private Companies
Press Release.

 

Montreal, Quebec and Cambridge, UK February 19, 2025: Epitopea, a transatlantic cancer immunotherapeutics company developing accessible, off-the-shelf RNA-based immunotherapies, announces a license and research collaboration agreement with MSD (tradename of Merck & Co., Inc., Rahway, N.J., USA) to identify CryptigenTM tumor-specific antigens (TSAs) in an undisclosed solid tumor. CryptigenTM TSAs are shared, non-mutated, aberrantly expressed antigens that are derived from what were thought to be non-coding regions of the genome or “junk DNA”.

Under the terms of the agreement, Epitopea will deploy its proprietary CryptoMapTM platform to identify and provide novel, immunogenic CryptigenTM TSAs for a prespecified tumor type. MSD will have the exclusive right to develop and commercialize therapeutics derived from the collaboration. In return Epitopea will receive an undisclosed upfront payment and is eligible for milestone payments with the potential to total up to $300 million per product.

“Epitopea has been at the forefront of identifying CryptigenTM TSAs, whose intratumor shared nature across patients has made them ideal targets for the development of off-the-shelf immunotherapies,” commented Alan C. Rigby, Epitopea’s CEO. “At Epitopea we continue to accelerate the development of our preclinical pipeline as we transition to a clinical-stage company on the ‘heels’ of our recent, oversubscribed, pre-Series A financing. We believe that this strategic collaborative relationship with MSD, a leader in immunotherapy therapeutic development, provides us with an additional opportunity to validate the potential impact of these differentiated tumor specific antigens. We are thrilled to collaborate with MSD as our teams collectively look to impact the lives of patients with cancer by helping to improve outcomes.”

“Despite the remarkable progress made in cancer treatment over the past decade, more therapeutic options are needed,” said George Addona, senior vice president, discovery, preclinical development and translational medicine, Merck Research Laboratories. “We continue to explore new ways to build upon our strong foundation in immuno-oncology and look forward to collaborating with the Epitopea team.”

“Having supported the company since its formation, we are thrilled that Epitopea’s next-generation antigen discovery platform will be leveraged in this license and research collaboration agreement with MSD, a leading biopharmaceutical company in the immuno-oncology space. This partnership provides strong validation for Epitopea’s unique capabilities and the potential to help develop breakthrough immunotherapies for cancer patients with the highest unmet need,” added Michael Anstey, Partner at Cambridge Innovation Capital.

About Epitopea

Epitopea is a transatlantic cancer immunotherapeutics company developing accessible off-the-shelf RNA-based immunotherapies for use in hard-to-treat cancers by targeting a new class of untapped tumor-specific antigens, which are known as CryptigenTM TSAs, that are broadly shared across multiple patients with the same tumor type.

The company has created an extensive library of novel CryptigenTM TSAs, discovered by its proprietary CryptoMapTM platform that leverages immunopeptidomics, genomics, and a bioinformatics pipeline, allowing the identification of aberrantly expressed, tumour-specific antigens that are hidden within cancer’s ’junk’ DNA. These hidden CryptigenTM TSAs were first discovered through research led by Drs. Claude Perreault and Pierre Thibault at the Institute for Research in Immunology and Cancer at the Université de Montréal.

Epitopea is backed by a leading transatlantic life science investor syndicate that includes Advent Life Sciences, CTI Life Sciences, Cambridge Innovation Capital, Le Fonds de Solidarité FTQ, Investissement Québec, adMare BioInnovations, Jonathan Milner, IRICoR, the Harrington Discovery Institute, and Novateur Ventures.

To date the company has raised financing of more than USD $45 million. Epitopea was founded in 2021 with sister companies based in Cambridge, UK and in Montreal, Canada. For additional information, please visit www.epitopea.com and follow us on LinkedIn.

For enquires please contact:
Epitopea
Dr. Alan C. Rigby – CEO
Alan.Rigby@epitopea.com

Scius Communications
Katja Stout
+44 7789 435990
katja@sciuscommunications.com

Daniel Gooch
+44 7747 875479
Daniel@sciuscommunications.com

Jan 22
Love0

Artax Biopharma achieves clinical validation of first-in-class, oral,  Nck modulator AX-158 for autoimmune disease in phase 2a study

By Artax Biopharma, Press Release, Private Companies
Press Release.

 

Artax Biopharma achieves clinical validation of first-in-class, oral,  Nck modulator AX-158 for autoimmune disease in phase 2a study

  • Novel breakthrough: first molecule to modulate the fundamental mechanism of T cell activation underlying autoimmune pathology
  • Statistically significant improvements in T cell and psoriasis-related biomarkers in line with PASI improvements in Phase 2a in patients with psoriasis
  • Phase 2a confirms well-tolerated safety profile, with no infections or discontinuations

Cambridge, MA, January 22, 2025 – Artax Biopharma, Inc., a clinical-stage biotechnology company focused on transforming the treatment of autoimmune diseases, today announces achievement of clinical validation through its Phase 2a clinical trial in psoriasis with fully proprietary oral agent AX-158. These results confirm the strong Phase 1 and preclinical data package, underscoring the potential breadth and depth of Nck modulation, a completely novel mechanism in autoimmune disease.

AX-158 was evaluated in a Phase 2a trial in people with mild to moderate plaque psoriasis in multiple centers in the UK. Participants were randomized 2:1 to receive a single daily dose of 10mg AX-158 or placebo. A total of 30 participants were treated for 28 days and followed for an additional 30 days for safety. The main objectives of the study were safety and proof of mechanism.

The Phase 2a study confirmed the well-tolerated safety profile seen in the Phase 1 studies, with no infections reported and no severe adverse events. The few adverse events reported were mild or moderate in nature and resolved without intervention, and there were no trial discontinuations.

An extensive psoriasis biomarker analysis was designed, conducted, and analyzed by Dr. James Krueger, Head of Laboratory for Investigative Dermatology at the Rockefeller University and member of the Scientific Advisory Board at Artax Biopharma. His analysis demonstrates statistically significant and consistent effects on both disease and pathway-related genes in participants treated with AX-158.  Dr. Kruger commented that “For a novel mechanism of action, it is exciting to see how it can begin to impact disease processes, and I look forward to seeing how the response expands over a longer treatment period in future trials.”

CEO Rob Armstrong added: “Today’s results validate this truly orthogonal, novel mechanism of action in the autoimmune space. We were thoroughly delighted to see that Nck modulation was able to impact relevant biomarkers as well as clinical measurements, particularly in the moderate patient population. We were pleased to observe that PASI 75 responses also progressed in line with biomarker data in this study. We would like to thank the investigative clinicians, their teams, and all participating patients for their valuable contribution to advancing this science. There is so much work left to do in the autoimmune space. With AX-158 and our portfolio of Nck modulators, we see great potential to address the treatment gap for patients.”

The company intends to report the results at an upcoming medical conference.

Given the broad effect of Nck modulation across all T helper cell populations (Th17, Th1/0 and Th2) in pre-clinical and animal models, the team is planning further studies in additional autoimmune diseases such as atopic dermatitis. Clinical results from these studies are anticipated by the end of 2026.

About Artax Biopharma, Nck modulation, and AX-158 

Artax Biopharma is a clinical-stage biotechnology company transforming the treatment of T Cell-driven autoimmune diseases. Artax’s first-in-class oral small molecules aim to deliver immune system modulation without immunosuppression, potentially unlocking new treatment options as both monotherapy and in combination with other treatments.

We believe there is significant potential for Nck modulation to revolutionize treatment of T Cell-driven diseases. Immunomodulation maintains healthy control of the immune system, while addressing the underlying source of T Cell-driven diseases. Central to a well-functioning immune system is the T Cell Receptor (TCR). When TCR signaling becomes dysregulated, it causes T Cell-driven conditions, including autoimmune diseases. We believe the immunomodulation mechanism offered by our investigational agents holds broad potential to revolutionize how these T Cell-driven autoimmune diseases are addressed, while not impairing the ability of a patient’s immune system to function properly.

AX-158, our lead Nck modulator, has shown strong, broad cytokine modulation as well as modulation of mixed lymphocyte reactions. Good data on therapeutic efficacy with AX-158 were observed in murine models of self-antigen activation (EAE), with a prolonged pharmacodynamic effect in EAE, suggesting durable immune modulation.

AX-158 showed no immunosuppression in models of strong antigen stimulation. Studies with AX-158 showed substantial preclinical evidence of activity in the Th2, Th17, Th1/Th0 pathways, suggesting that applications could be quite broad across the autoimmune space.

Artax Biopharma is based in the Boston area and raised funding from investors including Advent Life Sciences, Sound Bioventures, The Termeer Family Office, the Fuhrer Family Office and Columbus Venture Partners. For more info, see www.artaxbiopharma.com or follow us on LinkedIn.

For future clinical study inquiries: contact@artaxbiopharma.com

Jan 14
Love0

Pheno Therapeutics granted clinical trial authorisation for lead multiple sclerosis therapeutic candidate PTD802

By Pheno Therapeutics, Press Release, Private Companies
Press Release.

 

Pheno Therapeutics granted clinical trial authorisation for lead multiple sclerosis therapeutic candidate PTD802

  • UK MHRA grants CTA for first-in-human trial of a selective GPR17 antagonist
  • Small molecule therapeutic designed for treatment of neurological diseases such as multiple sclerosis

Edinburgh, UK, 14 January 2025: Pheno Therapeutics Limited., a biotechnology company focused on the discovery and development of small molecule therapeutics for the treatment of neurological diseases, announced today it has received clinical trial authorisation (CTA) from the UK’s MHRA (Medicines and Healthcare products Regulatory Agency) for its lead candidate, PTD802.

A selective GPR17 (G protein-coupled receptor 17) antagonist, PTD802 is a novel small molecule therapeutic designed to promote remyelination. Developed under an exclusive worldwide licence agreement with UCB, the programme is targeted toward treatment of neurological diseases with high unmet medical need, with an initial focus on multiple sclerosis (MS).

Demyelination in MS occurs when the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibres in the central nervous system, leading to multifocal demyelination, axonal injury, and neurodegeneration. MS is a chronic disease caused by demyelination, often associated with a wide range of neurological symptoms, which despite the ability of existing drugs to control the inflammatory component of the disease, can progress to total physical and cognitive disability.

Professor Siddharthan Chandran, Co-Founder of Pheno Therapeutics, added: “Current treatments for MS focus mainly on the immune aspects of the disease, reducing severity and frequency of relapses. There is an urgent and unmet need for effective therapeutics that limit disability progression in MS, with remyelination offering a promising neuroprotective treatment. Whilst GPR17 antagonists have potential utility beyond MS, PTD802 is a hugely promising first-in-class oral remyelination agent which we believe will be the next step in devising combinatorial approaches to preventing MS progression.”

“We are delighted to have received approval from the MHRA to progress our PTD802 programme to a Phase 1 trial, a major milestone, marking our transition to a clinical stage organisation. As the first company to carry out dosing of a selective GPR17 antagonist in healthy humans we are leading the way in the race to develop GPR17-targeting remyelination therapeutics,” said Fraser Murray, PhD, Chief Executive Officer of Pheno Therapeutics. “With this first-in-human programme we are moving closer to our goal of delivering transformational drugs for the treatment of neurological diseases associated with demyelination.”

 

Contact:

Zyme Communications

Katie Odgaard

Katie.odgaard@zymecommunications.com

 

To opt-out from receiving press releases from Zyme Communications please e-mail info@zymecommunications.com. To view our privacy policy, please click here.

 

About Pheno Therapeutics

Pheno Therapeutics is a biotechnology company, backed by leading biotech investors Advent Life Sciences and LifeArc, focused on the discovery and development of small molecule therapeutics that boost the function of human oligodendrocytes and their natural ability to promote remyelination, for the treatment of neurological diseases with high unmet medical need such as multiple sclerosis (MS).  Pheno combines world leading expertise in myelin biology, MS trials, patient selection and clinical cohorts. MS is a devastating chronic disease with significant individual and societal impact that often manifests in young adults and is associated with a wide range of neurological symptoms which can progress to total physical and cognitive disability.  Pheno Therapeutics is seeking to promote remyelination and reverse the critical demyelination aspect of MS. MS demyelination occurs when the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibres in the central nervous system, leading to multifocal demyelination, axonal injury and neurodegeneration.

For more information on Pheno Therapeutics, please visit https://www.phenotherapeutics.com.

Jan 13
Love0

Rappta Therapeutics enters into a global license agreement with SpringWorks Therapeutics for a pre-clinical first-in-class molecular glue targeting PP2A 

By Press Release, Private Companies, Rappta Therapeutics
Press Release.

 

Rappta Therapeutics enters into a global license agreement with SpringWorks Therapeutics for a pre-clinical first-in-class molecular glue targeting PP2A 

  • Goal to accelerate development and commercialization of the lead asset RPT04402 for the treatment of a subset of uterine cancers
  • Financials include a $13 million upfront payment and potential for significant further milestones

13 January 2025, Helsinki, Finland: Rappta Therapeutics (“Rappta”), focused on developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A), today announces an exclusive global license agreement with SpringWorks Therapeutics (“SpringWorks”) for RPT04402, a first-in-class molecular glue of specific Protein Phosphatase 2A (PP2A) complexes.

Rappta’s PP2A-reactivating technologies developed using its proprietary high resolution structural data have the potential to create a new class of anti-cancer drugs for treating a broad range of human cancers. PP2A is a critical enzyme in regulating protein de-phosphorylation and its reactivation is fundamental for tumor suppression, but it has been historically hard to target.

PP2A mutations are oncogenic drivers in molecularly defined subsets of uterine cancer and represent a targetable subset of patients with a high unmet clinical need. In pre-clinical models of PP2A mutant uterine cancer, RPT0402 achieved rapid, deep and durable tumor regressions at as monotherapy.

Under the exclusive license agreement, SpringWorks Therapeutics will be responsible for global development and commercialization of RPT04402. SpringWorks has paid Rappta $13 million upfront, and Rappta is also eligible to receive further clinical, regulatory and commercial milestone payments, and tiered single-digit royalties on net sales. SpringWorks expects to file an Investigational New Drug (IND) application for RPT04402 by the end of 2025.

Sunjeet Sawhney, Chief Executive Officer of Rappta Therapeutics, commented: “Rappta is the only company to have successfully targeted PP2A, a notoriously difficult and undruggable target. Data we generated demonstrated the potential of RPT04402 for treating large, underserved patient populations. SpringWorks, a leader in the targeted oncology space, has the expertise and knowledge to accelerate the further development of our first in class asset. I would like to thank our team and investors who have supported our journey and we look forward to following the progress made in this area”

Goutham Narla, Chief Scientific Officer of Rappta Therapeutics and Professor of Internal Medicine and Human Genetics at the University of Michigan, said: “Our team at Rappta Therapeutics has been able to leverage our proprietary structural data to develop a first-in-class series of molecular glues to the previously undruggable tumor suppressor PP2A. We are excited to be able to work with SpringWorks to potentially translate this approach to the clinic with the hope that this approach will have broad applications for the treatment of human cancers.”

Rappta was founded by Goutham Narla & Mikko Mannerkoski, back in 2019 with funding from Novartis Venture Fund (“NVF”), Novo Holdings, Advent Life Sciences and a family office in Series A financing alongside other non-dilutive funding from Business Finland.

ENDS

 

For more information please contact:

Rappta Therapeutics
Sunjeet Sawhney, CEO
+447425 421967
sunjeet.sawhney@rappta-therapeutics.com

Optimum Strategic Communications
Mary Clark, Hana Malik
+44 (0) 20 3882 2119
rappta@optimumcomms.co

About Rappta Therapeutics

Rappta Therapeutics, a private biotech with operations in Finland and the US, is developing first-in-class anti-cancer drugs activating protein phosphatase 2A (PP2A). It has developed proprietary tools and a unique understanding of PP2A which allows it to therapeutically reactivate PP2A, a critical enzyme regulating protein de-phosphorylation and tumor growth, with the potential to create a new class of anti-cancer drugs. Rappta has a strong scientific, management and commercial team. Its scientific team, led by CSO and co-founder, Professor Goutham Narla, Former Division Chief of Genetic Medicine at the University of Michigan, represents world-leading expertise in PP2A. It is backed by blue-chip investors Advent Life Sciences, Novartis Venture Fund, Novo Seeds and a family office. For more information, go to https://www.rappta-therapeutics.com/.

About PP2A

Reversible phosphorylation is a fundamental mechanism controlling all cell signaling and communication and this process is regulated through the opposing actions of phosphatases (which remove phosphate groups from proteins) and kinases (which add phosphate groups to proteins). Altered cellular signaling because of protein hyperphosphorylation, results in the sustained growth of malignant cells and is a hallmark of human cancer development and progression.

Protein Phosphatase 2A (PP2A) is a serine/threonine phosphatase that functions as a tumor suppressor by negatively regulating multiple oncogenic signaling pathways responsible for driving cancer progression. PP2A is made up of three subunits, that form a complete and active enzyme when bound together. The active enzyme is comprised of a scaffolding subunit (A), serving as the structural platform for the assembly of the catalytic (C) subunit and one substrate directing regulatory (B) subunit. In cancer, the tumor-suppressive activity of PP2A is often disrupted as a result of the inability of the three subunits to bind together correctly, rendering the PP2A enzyme inactive. This inactivation of PP2A, leads to increased oncogenic signaling, driving cancer progression and growth. Therefore, the reactivation of PP2A affords a unique therapeutic strategy to restore PP2A activity and cellular homeostasis, that can be used for the treatment of cancer and a broad range of other diseases.

About SpringWorks Therapeutics

SpringWorks is a commercial-stage biopharmaceutical company applying a precision medicine approach to developing and delivering life-changing medicines for people with severe rare diseases and cancer. OGSIVEO® (nirogacestat), approved in the United States for the treatment of adult patients with progressing desmoid tumors who require systemic treatment, is the Company’s first FDA-approved therapy. SpringWorks also has a diversified targeted therapy pipeline spanning solid tumors and hematological cancers, with programs ranging from preclinical development through advanced clinical trials. In addition to its wholly owned programs, SpringWorks has also entered into multiple collaborations with innovators in industry and academia to unlock the full potential for its portfolio and create more solutions for patients in need.

For more information, visit www.springworkstx.com and follow @SpringWorksTx on X (formerly Twitter), LinkedIn, and YouTube.

Jan 09
Love0

Relief Cardiovascular Announces $12M Series A Financing to Advance First-in-Human Study of the Relief System

By Advent Life Sciences, Press Release
Press Release.

 

Relief Cardiovascular Announces $12M Series A Financing to Advance First-in-Human Study of the Relief System

Irvine, California – January 8, 2025 – Relief Cardiovascular, an inQB8 Medical Technologies portfolio company pioneering innovative solutions for the management of heart failure, today announced the closing of a $12M Series A financing. The funding round was co-led by Broadview Ventures and Advent Life Sciences, with participation from Pacific Health Investment, Heartwork Capital, and an undisclosed multinational strategic. The funds will support further product development and the initiation of the company’s first-in-human feasibility study, designed to evaluate the safety and efficacy of the Relief System – a transcatheter smart implant designed to monitor and modulate renal vein pressures, offering a novel approach to fluid management in heart failure.

As part of the financing, Shahzad Malik, MD, General Partner at Advent Life Sciences, and Maria Berkman, MD, Head of Medtech at Broadview Ventures, will join the company’s Board of Directors.

“Heart failure continues to impose a tremendous burden on patients and healthcare systems,” said Maria Berkman, MD. “The Relief System addresses a critical gap in patient care, and we are proud to partner with the team at Relief Cardiovascular to advance this novel therapy into the clinic.”

“Relief Cardiovascular’s innovative approach to volume management tackles the unmet needs of diuretic-resistant heart failure,” said Shahzad Malik, MD. “The Relief System’s ability to dynamically modulate renal vein pressures offers the potential to redefine treatment and improve outcomes for patients facing limited options.”

The Relief System employs a transcatheter implant to reduce renal vein pressure using an innovative “puller” mechanism, paired with integrated sensors that autonomously capture renal vein pressures and multiparametric data. The system provides physicians with actionable data for optimizing fluid management and offers tailored therapeutic device-driven intervention.

“The Relief System’s ability to integrate monitoring and therapeutic modulation of renal pressures represents a promising advance in volume management in heart failure,” said William T. Abraham, MD, Professor of Medicine at The Ohio State University Wexner Medical Center. Alex Rothman, MD, Professor of Cardiology at the University of Sheffield, added, “By enabling data-driven adjustments, the Relief System empowers more personalized and effective care for patients, particularly those with diuretic resistance.”

“This financing marks a pivotal milestone for us as we advance the Relief System into human clinical studies,” said Alex Cooper, CEO of Relief Cardiovascular. “We’re honored to have the support of world-class investors and clinicians, along with industry veterans like Michael Minogue. With Shahzad and Maria joining our board, our team is positioned to transform the management and treatment of heart failure”

About Relief Cardiovascular
Relief Cardiovascular is a privately held medical device company committed to improving outcomes for patients with heart failure. The company’s flagship technology, the Relief System, is a transcatheter smart implant designed to enhance fluid management and improve quality of life for heart failure patients. Relief Cardiovascular was founded and incubated within inQB8 Medical Technologies, LLC.

About Broadview Ventures
Broadview Ventures is a mission-driven investment firm focused on accelerating the development of transformative solutions for cardiovascular and neurovascular diseases. Broadview partners with innovators to address significant clinical challenges and improve patient outcomes globally. For more information, please visit www.broadviewventures.org

About Advent Life Sciences
Advent Life Sciences founds and invests in early- and mid-stage life sciences companies that have a first- or best-in-class approach to unmet medical needs. The investing team consists of experienced professionals, each with extensive scientific, medical and operational experience, a long-standing record of entrepreneurial and investment success in the US and Europe and is particularly focused on supporting entrepreneurs and founders to take innovative new medical entities from concept to approval. The firm invests in a range of sectors within life sciences, principally drug discovery, enabling technologies and med tech, always with an emphasis on innovative, paradigm-changing approaches. Advent Life Sciences has a presence in the UK, US and France.  For more information, please visit www.Adventls.com

About inQB8 Medical Technologies

inQB8 Medical Technologies is a privately held medical device incubator based in Massachusetts, co-founded by Dr. Arshad Quadri, MD, and J. Brent Ratz, MBA. inQB8 specializes in developing cutting-edge interventional solutions for major cardiovascular diseases, accelerating projects through prototyping, bench, and pre-clinical testing.

For inquiries, contact: info@reliefcardio.com

Dec 06
Love0

Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP)

By Beacon Therapeutics, Press Release, Private Companies
Press Release.

 

Beacon Therapeutics Announces Positive 3-Month Data from Phase 2 DAWN Trial of laru-zova (AGTC-501) in Patients with X-Linked Retinitis Pigmentosa (XLRP)

  • To date, laru-zova has been well-tolerated by all participants in the Phase 2 DAWN study.
  • Data show promising early improvements in low luminance visual acuity (LLVA), a critical measure of visual function.
  • Data build confidence in laru-zova as a potential treatment for patients with XLRP.
  • Pivotal Phase 2/3 VISTA trial for laru-zova in XLRP is currently enrolling.

London, UK, Cambridge, MA, 6 December 2024 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading ophthalmic gene therapy company with a purpose to save and restore the vision of patients with blinding retinal diseases, today announced the presentation of 3-month interim safety and efficacy results of the Phase 2 DAWN trial in patients with XLRP at the FLORetina-ICOOR Meeting 2024 in Florence, Italy.

Key presentation highlights:

  • The three-month data show that laru-zova has been well-tolerated by all participants.
  • No study agent-related treatment emergent adverse events (TEAEs) were reported, including no ocular inflammatory adverse events.
  • Data also show promising early improvements in LLVA, a critical measure of visual function.
  • The benefit-risk profile supports on-going clinical development for the treatment of patients with XLRP caused by RPGR mutations.

DAWN is a non-randomized, open-label study of laru-zova in participants with XLRP who have previously been treated with a full-length AAV-vector based gene therapy targeting the RPGR protein. The purpose of DAWN is to assess two different doses of laru-zova for efficacy, safety and tolerability in the untreated eye of participants who previously received gene therapy for XLRP.

XLRP is a severe, aggressive, inherited retinal disease that often leads to blindness by middle age, with no treatment options available. XLRP primarily affects young males with an estimated prevalence of 1 in 25,000 males in US, Europe and Australia having XLRP with RPGR mutations. Laru-zova expresses the full length RPGR protein and is therefore expected to address the entirety of photoreceptor damage caused by XLRP, including both rod and cone loss, representing a potential best-in-class treatment for progressive vision loss in patients with XLRP.

Lance Baldo, MD, chief executive officer of Beacon Therapeutics, stated, “We are encouraged by the early results from the Phase 2 DAWN study. The strong safety profile observed to date is complemented by promising early improvements in low luminance visual acuity – a meaningful and functional measure of vision in patients with XLRP. These data not only support the ongoing pivotal VISTA study, but also strengthen our commitment to this opportunity to bring hope to patients and families affected by this devastating disease.”

Beacon Therapeutics is also enrolling patients for its pivotal Phase 2/3 VISTA trial of laru-zova as it develops this potential treatment for patients with XLRP.

Presentations –
• Subretinal Gene Therapy laru-zova (laru-zova (AGTC-501)) for X-Linked Retinitis Pigmentosa (XLRP) Phase 2 Multicenter Study (DAWN): Preliminary Results

• RPGR gene therapy and the Beacon clinical trials: Beacon Therapeutics Subretinal Gene Therapy laru-zova (laru-zova (AGTC-501)) for X-Linked Retinitis Pigmentosa (XLRP)

Presenting Author – Professor Paulo Eduardo Stanga, Founder and Chief Medical Officer, The Retina Clinic London

Contact:
info@beacontx.com

Media:
beacontherapeutics@edelman.com

About Beacon Therapeutics
Beacon Therapeutics is an ophthalmic gene therapy company founded in 2023 to save and restore the vision of patients with a range of prevalent and rare retinal diseases that result in blindness.

The Company has an established scientific foundation that combines a late-stage development candidate to treat X-linked retinitis pigmentosa (XLRP) and two preclinical programs, one targeting dry age-related macular degeneration (AMD) and another targeting an inherited cone-rod dystrophy (CRD). Beacon Therapeutics also has access to a target generation technology platform that will identify, screen, and search secreted proteins in the ophthalmology space.

Lead development candidate laru-zova (laru-zova (AGTC-501)), is a gene therapy program currently being investigated for the treatment of XLRP, an inherited monogenic recessive disorder that causes progressive vision loss, primarily in boys and young men. XLRP is predominantly caused by mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. Laru-zova expresses the full length RPGR protein, thereby addressing the full complement of photoreceptor damage caused by XLRP, including both rod and cone loss.

Beacon is supported by funds from Syncona Limited, Forbion, Oxford Science Enterprises, TCGX, Advent Life Sciences and additional investors.

Find out more about Beacon Therapeutics at beacontx.com.

Dec 05
Love0

Arrakis Therapeutics Unveils Data for its Lead, Rationally Designed, RNA-Targeted Small Molecule (rSM) Drug Program Demonstrating Disease-Modifying Binding to RNA to Treat Myotonic Dystrophy

By Arrakis Therapeutics, Press Release, Private Companies
Press Release.

 

Arrakis Therapeutics Unveils Data for its Lead, Rationally Designed, RNA-Targeted Small Molecule (rSM) Drug Program Demonstrating Disease-Modifying Binding to RNA to Treat Myotonic Dystrophy

Preclinical data presented at Cell Symposia conference show a small molecule directed at a pathogenic RNA achieved reversal of myotonia in an animal model of myotonic dystrophy Type 1 (DM1)

Supports additional product development with Company’s multi-modality rSM drug platform, broadening the reach of genetic medicine and offering the potential to treat diseases at their root cause with oral medicines

Waltham, Mass., December 4, 2024 – Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced the first presentation of data for its RNA-targeted small molecule (rSM) drug program for the treatment of myotonic dystrophy type 1 (DM1), a form of muscular dystrophy. These preclinical data demonstrate Arrakis’s leading-edge capability to rationally design a disease-modifying RNA-targeted small molecule, using its pioneering and comprehensive drug discovery platform. The data are being presented today at the Cell Symposia conference Chemical Biology in Drugging the Undrugged, taking place in San Francisco, CA on December 2-4, 2024.

The preclinical data from in vivo animal studies demonstrated the reversal of myotonia with Arrakis rSMs binding the target pathogenic CUG repeat RNA element responsible for DM1. Arrakis molecules selectively bind the CUG repeat, disrupt the formation of nuclear aggregates, release sequestered splicing factors, specifically Muscleblind-Like Splicing Regulator 1 (MBNL1), and correct splicing defects responsible for myotonia. Arrakis’s proprietary RNA‐specific chemical, biological, and structural methods and RNA-directed medicinal chemistry enabled structure-based small molecule drug design targeting the trinucleotide (CUG) repeat expansion in the mRNA of DMPK (myotonic dystrophy protein kinase) that drives DM1 pathology. The methodologies applied to the DM1 drug program are being used by Arrakis to advance its pipeline of proprietary and partnered rSM drug candidates for a range of other diseases.

“While there have been serendipitous discoveries of small molecule drugs that bind to RNA, we believe this is one of the clearest examples of a rationally designed small molecule that has been purpose-built to address an RNA disease target,” said Michael Gilman, PhD, Chief Executive Officer of Arrakis Therapeutics. “With the many tools we’ve built and lessons we’ve learned along the way, we believe that our DM1 program is the first of many potential RNA-targeted small molecule drug candidates that will emerge from our platform in coming years.”

Dr. Gilman added, “From the beginning, we have believed in the power of targeting RNA and committed to deeply understand RNA sequence, structure and function. rSMs combine the specificity, validation, and impact of genetic medicines with the well understood benefits of small molecule drugs, including systemic biodistribution, oral delivery, and more streamlined and cost-efficient supply chains. With our platform now built and scaled, we look forward to advancing our pipeline and vastly expanding the reach of RNA-targeted medicines by deploying our industry’s most established drug modality, oral small molecules.”

A presentation at Cell Symposia describes results from several preclinical studies. Highlights include:

  • High-resolution X-ray structures revealing the interaction of multiple Arrakis rSMs bound to the CUG RNA repeat.
  • Arrakis rSMs selectively bind the target RNA and displace MBNL1, a splicing factor sequestered in nuclear aggregates caused by CUG RNA repeats associated with DM1.
  • Arrakis rSMs neutralize repeat aggregates and correct splicing defects in DM1 patient-derived myocytes.
  • Arrakis rSMs modulate splicing and reverse myotonia in the HSALR mouse, the gold standard murine model of muscle dysfunction in DM1.

The presentation from Cell Symposia is available here on Arrakis’s website.

“We are pleased to share data on our DM1 program. For the first time, we are showing how our in silico, wet lab, and structural biology capabilities enable Arrakis to identify druggable RNA target sites and to rationally design and optimize small molecules that selectively bind to an RNA structure to modify its function,” said Jennifer Petter, PhD, Founder and Chief Scientific Officer at Arrakis Therapeutics and presenter of the data at Cell Symposia. “While there are promising muscle-directed oligonucleotide therapies in clinical development for the treatment of DM1, our rSMs have the potential to address the full systemic manifestations of the disease and represent a new class of genetic medicine that use small molecules to directly address the genetic basis of disease by targeting pathogenic RNA.”

 

About the rSM Program to Treat Myotonic Dystrophy Type 1 (DM1)

Myotonic dystrophy type 1 (DM1) is a form of muscular dystrophy and a genetic neuromuscular disease affecting at least 1 in 8,000 people worldwide or approximately 45,000 people in the United States. It is a multi-system disease, affecting the skeletal muscle, heart, diaphragm, central nervous system, and gastro-intestinal tract. DM1 is caused by a trinucleotide (CUG) repeat expansion of the RNA encoding DMPK (myotonic dystrophy protein kinase), resulting in the formation of nuclear aggregates that bind and sequester splicing factors important for cellular function. Arrakis’s rSM selectively binds to CUG repeats, disrupting aggregate formation in cells, liberating splicing factors, and correcting splicing defects in genes that drive DM1 pathology, including myotonia.

 

About Arrakis Therapeutics

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on genetically validated targets and numerous disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Massachusetts. For more information, please visit www.arrakistx.com and engage with us on LinkedIn.

 

Media Contact:

Kathryn Morris
The Yates Network LLC
914-204-6412
kathryn@theyatesnetwork.com