Skip to main content
Category

Press Release

Oct 25
Love0

Bayer’s Lynkuet™ (elinzanetant) approved in the U.S. for treatment of moderate to severe vasomotor symptoms due to menopause

By Advent Life Sciences, KaNDy Therapeutics, Press Release
Press Release.

 

October 24, 2025

Bayer’s Lynkuet™ (elinzanetant) approved in the U.S. for treatment of moderate to severe vasomotor symptoms due to menopause

This approval is supported by data from the Phase III OASIS clinical program evaluating Lynkuet™ (elinzanetant) for the treatment of moderate to severe vasomotor symptoms (VMS, also known as hot flashes), due to menopause1 / In OASIS 1 and 2, elinzanetant met the co-primary endpoints of reduction in number and severity of moderate to severe hot flashes day and night at weeks 4 and 12 from baseline1 / Hot flashes are a common symptom of menopause2 and one of the main reasons women seek treatment3, hot flashes may impact women differently4 and some can be disruptive5 / Elinzanetant is the first dual neurokinin (NK) targeted therapy,1 NK1 and NK3 receptor antagonist

Berlin, October 24, 2025 – Bayer announced today that the U.S. Food and Drug Administration (FDA) has approved elinzanetant as the first dual neurokinin (NK) targeted therapy1, neurokinin 1 (NK1) and neurokinin 3 (NK3) receptor antagonist, under the brand name Lynkuet™ for the treatment of moderate to severe vasomotor symptoms (VMS, also known as hot flashes) due to menopause. Inhibition of Substance P and Neurokinin B through antagonism of NK1 and NK3 receptor signaling on kisspeptin/neurokinin B/dynorphin (KNDy) neurons with elinzanetant can modulate neuronal activity in the thermoregulation associated with hot flashes.1 The FDA approval is supported by data from three Phase III clinical studies (OASIS 1, OASIS 2 and OASIS 3) that evaluated the efficacy and safety of elinzanetant for the treatment of moderate to severe VMS due to menopause.

“For more than a century, Bayer has been dedicated to pioneering advances in women’s health, and this FDA approval represents a bold step forward – our first hormone-free treatment for alleviating vasomotor symptoms of menopause,” said Christine Roth, Executive Vice President, Global Product Strategy and Commercialization and Member of the Pharmaceuticals Leadership Team at Bayer. “There is a need for more individualized approaches to menopause care, and Lynkuet™ addresses a significant gap in treatment options. The approval in the U.S. reflects our unwavering commitment to delivering science-driven solutions that meet women’s evolving healthcare needs and empower them to take charge of their health at every stage of life.”

The efficacy of elinzanetant for the treatment of moderate to severe VMS due to menopause was demonstrated in the first 12 weeks of two randomized, double-blind, placebo-controlled, multicenter clinical trials, OASIS 1 and OASIS 2, in 796 menopausal women.1 The co-primary efficacy endpoints in both trials were the mean change in frequency and severity of moderate to severe VMS from baselines to weeks 4 and 12, including day and night hot flashes.1 The safety of elinzanetant was evaluated in three randomized, double-blind, placebo-controlled, multicenter clinical trials (OASIS 1, OASIS 2 and OASIS 3) in 1420 women. In OASIS 3, 627 women received elinzanetant or placebo for up to 52 weeks to evaluate long-term safety.1

“These three studies investigated the safety and efficacy of elinzanetant for the treatment of moderate to severe hot flashes due to menopause,” said JoAnn Pinkerton, M.D., Professor and Director of Midlife Health at UVA Health and Lead Investigator on the OASIS 2 trial. “Hot flashes, particularly when severe, can have an impact on women’s daily lives and this approval provides healthcare providers with a new treatment option that can be used first-line for moderate to severe hot flashes due to menopause.”

Hot flashes are a common symptom of menopause2 that may impact women differently4. Hot flashes can be disruptive5 and are a common reason for which women in menopause seek treatment3.

“It’s important that women know they have choices for treating moderate to severe hot flashes due to menopause, and today’s approval further expands a woman’s options for treating these symptoms,” said Claire Gill, President and Founder of the National Menopause Foundation.

Elinzanetant is expected to be available in the U.S. beginning in November 2025.

About elinzanetant
Elinzanetant is the first dual neurokinin (NK) targeted therapy,1 neurokinin 1 (NK1) and neurokinin 3 (NK3) receptor antagonist, approved in the U.S. for the treatment of moderate to severe VMS due to menopause.1

The compound is being developed globally for the treatment of moderate to severe vasomotor symptoms (VMS; also known as hot flashes) associated with menopause or endocrine therapy (ET) for breast cancer, administered orally once daily. Increasing evidence indicates that hypothalamic neurons called kisspeptin, neurokinin B, and dynorphin (KNDy) neurons, expressing both NK-1 and NK-3 receptors and their ligands Substance P and NKB, play a role in thermoregulation. Declining estrogenic activity due to natural menopause or endocrine therapy leads to hyperactivity of KNDy neurons and dysregulation of the thermoregulatory center, resulting in VMS. NK-1 receptors may also have a role in the cooling response through sweating and peripheral vasodilatation as well as on sleep disturbance.

Elinzanetant has been approved under the brand name Lynkuet™ in the U.S. as well as in Australia, Canada, the UK, and Switzerland. It is pending approval in the European Union and under review in other markets around the world.

About Menopause
By 2030, the global population of women experiencing menopause is projected to increase to 1.2 billion, with 47 million women entering this phase each year. Menopause is a phase in women’s lives, related to the progressive decline of ovarian function. It usually occurs in women during their 40s or early 50s. Menopause symptoms can also be a consequence of surgical (such as bilateral oophorectomy) or medical treatment (such as endocrine therapy for breast cancer). The most frequently reported and disruptive symptoms during the menopausal transition are VMS, sleep disturbances and mood changes.6-8Addressing these symptoms is key to maintaining functional ability and quality of life in menopause which is highly relevant from both a healthcare and socio-economic perspective.

About Women’s Healthcare at Bayer
Women’s Health is in Bayer’s DNA. As a global leader in women’s healthcare Bayer has a long-standing commitment to delivering science for a better life by advancing a portfolio of innovative treatments. Bayer offers a wide range of effective short- and long-acting birth control methods as well as therapies for menopause management and gynecological diseases. Bayer is also focusing on innovative options to address the unmet medical needs of women worldwide and to broadening treatment choices such as in menopause. Additionally, Bayer intends to provide 100 million women per year in low-and-middle income countries by 2030 with access to family planning by funding multi-stakeholder aid programs for capacity building and by ensuring the supply of affordable modern contraceptives. This is part of the comprehensive sustainability measures and commitments from 2020 onwards and in line with the Sustainable Development Goals of the United Nations.

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros. R&D expenses amounted to 6.2 billion euros. For more information, go to www.bayer.com.

Find more information at https://pharma.bayer.com/
Follow us on Facebook: http://www.facebook.com/bayer
Follow us on LinkedIn: Bayer | Pharmaceuticals

Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

References:

1LYNKUET® (elinzanetant) [Prescribing Information]. Whippany, NJ: Bayer HealthCare Pharmaceuticals, Inc.; October 2025
2Thurston, R. C., & Joffe, H. (2011). Vasomotor symptoms and menopause: Findings from the Study of Women’s Health across the Nation. Obstetrics and Gynecology Clinics of North America, 38(3), 489–501. https://doi.org/10.1016/j.ogc.2011.05.006.
3Kronenberg F. Menopausal hot flashes: a review of physiology and biosociocultural perspective on methods of assessment. J Nutr. 2010;140(7):1380S-5S.
4Shepherd JA, Shiozawa A, Schild AL, Singh D, Mancuso SA. Retrospective text and qualitative analyses of patient experience and management of vasomotor symptoms due to menopause: voices from the PatientsLikeMe community. Menopause. 2024 Sep 1;31(9):789-795. doi: 10.1097/GME.0000000000002391. Epub 2024 Jul 8. PMID: 38980735; PMCID: PMC11469626.
5US Food and Drug Administration. Estrogen and estrogen/progestin drug products to treat vasomotor symptoms and vulvar and vaginal atrophy. FDA. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/estrogen-and-estrogenprogestin-drug-products-treat-vasomotor-symptoms-and-vulvar-and-vaginal-atrophy. Accessed October 23, 2025.
6Thurston & Joffe, Obstet Gynecol Clin North Am. 2011 September ; 38(3): 489–501.
7Thurston, Climacteric 2018 April; 21(2): 96-100 6. Thurston RC et al, Sleep. 2019;42(9)
8.Utian, W.H. Health Qual Life Outcomes 3, 47 (2005)

Oct 18
Love0

Boston Scientific Announces Agreement to Acquire Nalu Medical, Inc.

By Advent Life Sciences, Nalu Medical, Press Release
Press Release.

 

Acquisition to expand the neuromodulation offerings for people living with chronic pain

MARLBOROUGH, Mass., Oct. 17, 2025 /PRNewswire/ — Boston Scientific Corporation (NYSE: BSX) today announced it has entered into a definitive agreement to acquire Nalu Medical, Inc., a privately held medical technology company focused on developing and commercializing innovative and minimally invasive solutions for patients with chronic pain.

Boston Scientific has been a strategic investor in Nalu Medical since 2017. The transaction consists of an upfront cash payment of approximately $533 million for the remaining equity not owned by Boston Scientific.*

The Nalu Neurostimulation System is designed to deliver targeted relief for adults living with severe, intractable chronic pain of peripheral nerve origin, including areas such as the shoulder, lower back and knee, through peripheral nerve stimulation (PNS). The therapy uses mild electrical impulses to interrupt pain signals before they reach the brain. The system features a miniaturized, battery-free implantable pulse generator, powered wirelessly by a small, externally worn therapy disc and controlled via a smartphone app.

Nalu Medical received U.S. Food and Drug Administration 510(k) clearance for the Nalu system in 2019. In the COMFORT and COMFORT 2 randomized controlled trials, evaluating the safety and efficacy of PNS, the system demonstrated significant and sustained pain relief for patients. In COMFORT, 87% of participants reported more than a 50% reduction in pain at 12 months,i while in COMFORT 2, 79% of patients reached an average pain relief of 64% at six months.ii Real-world data from more than 2,000 individuals reinforced these findings, with 94% of patients achieving clinically meaningful improvement across a broad range of chronic peripheral nerve pain conditions.iii

“Peripheral nerve stimulation is an exciting field with a significant unmet patient need,” said Jim Cassidy, president, Neuromodulation, Boston Scientific. “Adding the highly differentiated Nalu Medical technology complements our existing therapies—including spinal cord stimulation, basivertebral nerve ablation and radiofrequency ablation—enabling us to deliver advanced pain relief options to a wider variety of patient populations.”

Boston Scientific expects to complete the transaction in the first half of 2026, subject to customary closing conditions. Nalu is expected to generate sales in excess of $60 million in 2025 and to deliver year-over-year growth in excess of 25% in 2026. On an adjusted basis, the transaction is expected to be immaterial to adjusted earnings per share (EPS) in 2026, slightly accretive in 2027, and increasingly accretive thereafter. On a GAAP basis, the transaction is expected to be more dilutive due to amortization expense and acquisition-related charges.

*On a 100% basis before consideration of Boston Scientific’s current equity ownership in Nalu Medical, Inc. and other closing adjustments, the transaction price consists of an upfront cash payment of $600 million.  

About Boston Scientific

Boston Scientific transforms lives through innovative medical technologies that improve the health of patients around the world. As a global medical technology leader for more than 45 years, we advance science for life by providing a broad range of high-performance solutions that address unmet patient needs and reduce the cost of healthcare. Our portfolio of devices and therapies helps physicians diagnose and treat complex cardiovascular, respiratory, digestive, oncological, neurological and urological diseases and conditions. Learn more at www.bostonscientific.com and follow us on LinkedIn.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Forward-looking statements may be identified by words like “anticipate,” “expect,” “project,” “believe,” “plan,” “estimate,” “may,” “intend” and similar words. These forward-looking statements are based on our beliefs, assumptions and estimates using information available to us at the time and are not intended to be guarantees of future events or performance. These forward-looking statements include, among other things, statements regarding statements regarding our business plans, the financial and business impact of the transaction and the anticipated benefits of the transaction, the closing of the transaction and the timing thereof, anticipated sales by Nalu, and product performance and impact. If our underlying assumptions turn out to be incorrect, or if certain risks or uncertainties materialize, actual results could vary materially from the expectations and projections expressed or implied by our forward-looking statements. These factors, in some cases, have affected and in the future (together with other factors) could affect our ability to implement our business strategy and may cause actual results to differ materially from those contemplated by the statements expressed in this press release. As a result, readers are cautioned not to place undue reliance on any of our forward-looking statements.

Risks and uncertainties that may cause such differences include, among other things: economic conditions, including the impact of foreign currency fluctuations; future U.S. and global political, competitive, reimbursement and regulatory conditions, including changing trade and tariff policies; geopolitical events; manufacturing, distribution and supply chain disruptions and cost increases; disruptions caused by cybersecurity events; disruptions caused by public health emergencies or extreme weather or other climate change-related events; labor shortages and increases in labor costs; variations in outcomes of ongoing and future clinical trials and market studies; new product introductions; expected procedural volumes; the closing and integration of acquisitions, including our ability to achieve the anticipated benefits of the proposed transaction and successfully integrate Nalu’s operations; business disruptions (including disruptions in relationships with employees, customers and suppliers) following the announcement and/or closing of the proposed transaction; demographic trends; intellectual property; litigation; financial market conditions; the execution and effect of our business strategy, including our cost-savings and growth initiatives; future business decisions made by us and our competitors; the conditions to the completion of the proposed transaction, including the receipt of any required regulatory approvals and clearances, may not be satisfied at all or in a timely manner; and the closing of the proposed transaction may not occur or may be delayed. These and any new risks and uncertainties, which may arise from time to time, are difficult to predict accurately and many of them are beyond our control. For a further list and description of these and other important risks and uncertainties that may affect our future operations, see Part I, Item 1A – Risk Factors in our most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, which we may update in Part II, Item 1A – Risk Factors in Quarterly Reports on Form 10-Q we have filed or will file hereafter. We disclaim any intention or obligation to publicly update or revise any forward-looking statements to reflect any change in our expectations or in events, conditions, or circumstances on which those expectations may be based, or that may affect the likelihood that actual results will differ from those contained in the forward-looking statements, except as required by law. This cautionary statement is applicable to all forward-looking statements contained in this press release.

Note: Amounts reported in millions within this press release are computed based on the amounts in thousands. As a result, the sum of the components reported in millions may not equal the total amount reported in millions due to rounding. Certain columns and rows within tables may not add due to the use of rounded numbers. Percentages presented are calculated from the underlying unrounded amounts.

CONTACTS:
Jessica Sachariason
Media Relations
+1 (415) 720-2310
jessica.sachariason@bsci.com

Lauren Tengler
Investor Relations
+1 (508) 683-4479
BSXInvestorRelations@bsci.com

i Hatheway, J., Hersel, A., Engle, M., Gutierrez, G., Khemlani, V., Kapural, L., Moore, G., Ajakwe, R., Trainor, D., Hah, J., Staats, P., Makous, J., Heit, G., Kottalgi, S., & Desai, M. J. (2024). Clinical study of a micro-implantable pulse generator for the treatment of peripheral neuropathic pain: 12-month results from the COMFORT-randomized controlled trial. Regional Anesthesia & Pain Medicine. Advance online publication. https://doi.org/10.1136/rapm-2024-106099
ii Engle M, Gutierrez G, Hersel A, Netzel C, Khemlani V, Kapural L, Cubillo E, Hatheway J, Moore G, Valimahomed A, Khan K, Shuayto M, Majjhoo A, Sayed D, Latif U, Trainor D, Ajakwe R, Staats P, Makous J, Martin P*, Kottalgi S, Desai MJ; COMFORT 2 Study Group. A Confirmatory Randomized Controlled Trial Evaluating a Micro-Implantable Pulse Generator for the Treatment of Peripheral Neuropathic Pain: 3- and 6-Month Results from the COMFORT 2 Study. Chronic Pain & Management. 2025; 9: 171. DOI: 10.29011/2576-957X.1000171
iii Hatheway, J. A., Ratino, T., Swain, A. R., Ratino, T., Latif, U., Arulkumar, S., & Desai, M. J. (2025). Long-term pain relief delivered by micro-implantable pulse generator: Findings from a large-scale, real-world data peripheral nerve stimulation patient registry. Chronic Pain & Management, 9, 169. https://doi.org/10.29011/2576-957X.100069

 

SOURCE Boston Scientific Corporation

Sep 03
Love0

Glycomine Initiates Dosing in a Global, Randomized, Placebo-Controlled Phase 2b Study of GLM101 for the Treatment of PMM2-CDG

By Advent Life Sciences, Press Release
Press Release.

 

Glycomine Initiates Dosing in a Global, Randomized, Placebo-Controlled Phase 2b Study of GLM101 for the Treatment of PMM2-CDG

September 3, 2025

SAN CARLOS, Calif., September 3, 2025 — Glycomine, Inc. announced today that the first participant has been dosed in the global Phase 2b study, called POLAR. This randomized placebo-controlled multi-center clinical trial is designed to assess the safety and efficacy of GLM101, an investigational mannose-1-phosphate substrate replacement therapy for the treatment of phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG), a rare genetic disorder leading to serious neurological and multi-systemic impairments.

“We’ve been encouraged by the improvements in ataxia seen in our Phase 2 open-label study, and we are excited to bring GLM101 forward into this next phase of clinical development to further assess its safety and efficacy,” said Dr. Marino, Glycomine’s CMO.

POLAR (ClinicalTrials.gov Identifier: NCT06892288) plans to enroll approximately 50 adults and children ≥4 years of age living with PMM2-CDG across a planned 16 sites and 10 countries. The trial will consist of two phases. Part A is a 24-week randomized, double-blind, placebo-controlled treatment period designed to evaluate the primary efficacy of GLM101 compared to placebo. Participants will be randomized 1:1 to receive weekly intravenous infusions of 30 mg/kg GLM101 or placebo for 24 weeks before transitioning into Part B of the study. Part B is a 24-week open-label extension phase in which all participants will receive weekly IV infusions of GLM101.

The primary outcome assessment, evaluated after Part A of the study, is improvement in ataxia as measured by the International Cooperative Ataxia Rating Scale (ICARS), a clinical tool to assess the severity of ataxia. Ataxia is a hallmark of PMM2-CDG and has been reported to be a key driver of disease burden. Additional secondary and exploratory endpoints include clinical assessments of gross motor function, cognition, strength, and patient and clinician-reported global impressions of change.

The POLAR study design has been informed by results of an ongoing open-label Phase 2 study in pediatric and adult patients with PMM2-CDG, which has demonstrated encouraging clinical improvements in ataxia as measured by the ICARS and a favorable safety and tolerability profile.

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 with no other therapeutic options. 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.

Contacts

Corporate Contact: Peter McWilliams, Ph.D., info@glycomine.com
Media Contact: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com

Sep 01
Love0

Cyted Health Secures $44 Million in Series B Financing to Accelerate US Expansion

By Advent Life Sciences, Press Release
Press Release.

 

Cyted Health, a leading gastrointestinal (GI) molecular diagnostics company, today announces a first close of $44 million in its Series B financing round to accelerate its ongoing US expansion.

The round was led by EQT Life Sciences, investing through its EQT Health Economics strategy, and co-led by Advent Life Sciences and British Business Bank with continued support from existing investors Morningside and BGF.

The financing will be used to accelerate the commercial expansion of Cyted’s diagnostics platform in the US, consolidate existing commercial success across the UK, and expand its portfolio of advanced diagnostic tests. Cyted’s existing platform consists of EndoSign®, an FDA 510(k)-cleared device enabling minimally invasive collection of esophageal cells, and advanced biomarker molecular testing for the detection of esophageal conditions.

Cyted’s technology has already demonstrated significant success across the UK’s National HealthService, completing over 35,000 tests and building a robust portfolio of peer-reviewed publications proving unmatched patient acceptability and real-world clinical impact. Esophageal cancer is considered one of the deadliest cancers globally, with a survival rate of only 20%.

“This Series B financing marks a defining moment for Cyted as we continue to deepen our commitment to detecting esophageal diseases earlier,”said Marcel Gehrung, CEO and Co-founder of Cyted Health. “This investment will help us consolidate our leading position in the market by expanding our US presence and adding new life-saving innovations to our advanced diagnostics portfolio.”

Bruno Holthof, Partner at EQT Life Sciences, commented: “Cyted is strongly positioned to redefine the standard of care in upper GI diagnostics worldwide.Its minimally invasive diagnostics platform is the standout innovation to capture this significant market opportunity, and we’re delighted to add the company to our portfolio.”

“Cyted has demonstrated a rare combination of clinical innovation, patient-centered delivery, and commercial execution with an impressive foundation in the UK. We’re proud to support its next phase of growth,” said Kaasim Mahmood, General Partner at Advent Life Sciences.

Carmine Circelli, Director, Life Sciences at British Business Bank, added: “Cyted is an excellent example of the kind of high-impact business we aim to back. Their innovation has the potential to transform early cancer detection and create lasting health outcomes both in the UK and globally.”

Science Minister Lord Vallance said: “I am pleased to see the British Business Bank back Cyted – a company whose growth demonstrates the ability of our universities to turn world-class life sciences research into successful commercial opportunities with the potential to improve people’s lives. Life sciences form a critical part of this Government’s ambitions for both the economy and healthcare – and through our Life Sciences Sector Plan, we will continue to support the industry to even greater success.”

Founded by leading experts at the University of Cambridge, Cyted is pioneering the development of minimally invasive cell collection coupled with proprietary biomarker discovery to transform the detection of pre-cancerous, cancerous and inflammatory esophageal conditions.

Cyted’s Series B financing round follows the recent launch of Cyted’s DETECT-ME clinical validation study, which has been enrolling patients across multiple sites in the US since March 2025. This study will validate clinical performance of Cyted’s newest assays and molecular tests for the detection of esophageal conditions using EndoSign®.

Jul 08
Love0

MHRA approves elinzanetant to treat moderate to severe vasomotor symptoms (hot flushes) caused by menopause

By Advent Life Sciences, Press Release
Press Release.

 

The Medicines and Healthcare products Regulatory Agency (MHRA) has today, 8 July, become the first regulator in the world to approve elinzanetant (Lynkuet) for the treatment of moderate to severe vasomotor symptoms (hot flushes) associated with the menopause.

When oestrogen levels drop during menopause, certain brain cells become overactive and interrupt the body’s ability to control temperature, which leads to hot flushes and night sweats.   Elinzanetant is a new non-hormonal medication which works by calming these signals in the brain, helping bring the body’s temperature control back into balance.

It may also help improve sleep problems that often come with menopause.   This medicine is administered in capsule form, to be taken orally.

Julian Beach, MHRA Interim Executive Director of Healthcare Quality and Access, said:

“Hot flushes and night sweats associated with menopause can have a significant negative impact on quality of life.

“We are therefore pleased to announce our approval of elinzanetant, which has met the MHRA’s standards for safety, quality and effectiveness.

“Elinzanetant offers a non-hormonal alternative for those who may not be able to, or prefer not to, take hormone-based therapies. As with all licensed medicines, we will continue to monitor its safety closely as it becomes more widely used.”

Elinzanetant’s approval is based on results from the OASIS clinical trials, which involved over 1,400 women aged 40 to 65 across several countries. These studies showed that taking a daily 120 mg capsule of elinzanetant significantly reduced the number and intensity of hot flushes and night sweats over 26 to 52 weeks, compared to a placebo.

Like all medicines, this medicine can cause side effects in some people. A full list of side effects can be found in the Patient Information Leaflet (PIL) or the Summary of Product Characteristics (SmPC), available on the MHRA website within 7 days of approval.

Anyone who suspects they are having a side effect from this medicine should talk to their doctor, pharmacist or nurse and report it directly to the MHRA Yellow Card scheme, either through the website (https://yellowcard.mhra.gov.uk/) or by searching the Google Play or Apple App stores for MHRA Yellow Card.

Notes to editors

  • The new marketing authorisation was granted on 8 July 2025 to Bayer plc.
  • This national approval was granted after an Access Consortium new active substance work-sharing initiative (NASWSI) procedure.
  • More information can be found in the Summary of Product Characteristics and Patient Information leaflets which will be published on the MHRA Products website within 7 days of approval.
  • The Medicines and Healthcare products Regulatory Agency (MHRA) is responsible for regulating all medicines and medical devices in the UK by ensuring they work and are acceptably safe.  All our work is underpinned by robust and fact-based judgements to ensure that the benefits justify any risks.
  • The MHRA is an executive agency of the Department of Health and Social Care.
  • For media enquiries, please contact the newscentre@mhra.gov.uk, or call on 020 3080 7651.
May 01
Love1

AviadoBio Announces Completion of Second Cohort in Phase 1/2 ASPIRE-FTD Clinical Trial Studying AVB-101 for FTD-GRN

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio Announces Completion of Second Cohort in Phase 1/2 ASPIRE-FTD

Clinical Trial Studying AVB-101 for FTD-GRN

 AVB-101 Biomarker Data Readout Expected in 2026

 Clinical Milestone Shared at AFTD Annual Caregiver Conference

 LONDON — MAY 1, 2025 — AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced the completion of the second dose cohort in its Phase 1/2 ASPIRE-FTD clinical trial. ASPIRE-FTD is evaluating multiple doses of AviadoBio’s investigational gene therapy, AVB-101, in people living with frontotemporal dementia (FTD) with GRN gene mutations (FTD-GRN). The company intends to initiate dosing for a third cohort in Q3 2025 and expects to share early biomarker data in 2026. The announcement was made at the Association for Frontotemporal Degeneration (AFTD) annual caregiver conference in Denver.

ASPIRE-FTD is now open and actively recruiting participants at multiple sites across the U.S., Spain, Poland, Sweden and the Netherlands, with additional countries expected to join the trial in the near future. For central nervous system diseases like FTD, the blood-brain barrier presents a challenge to gene therapy delivery. AVB-101, an investigational gene therapy, is administered via a neurosurgical procedure directly to the thalamus—a key hub with widespread projections across the brain, including the frontal and temporal cortex regions most affected in FTD-GRN. This delivery method bypasses the blood-brain barrier and the pial membrane that separates the brain from the cerebrospinal fluid, targeting therapy precisely where needed while potentially reducing required dosage and limiting systemic exposure. Pre-clinical data indicate AVB-101 delivered to the thalamus can increase progranulin levels in cortical brain tissue, potentially restoring physiological function in people with FTD-GRN.

“Dosing our sixth patient with AVB-101 marks an important and encouraging step forward in our research pathway,” said David Cooper, M.D., Chief Medical Officer of AviadoBio. “In the first cohort, we did not see any clinically significant safety findings through follow-up of up to 52 weeks and did not require any immunosuppression prophylactically or reactively.”

“We’re proud to share this important milestone with the FTD community, who continue to inspire our commitment to advancing research for therapies that may slow or stop the progression of this devastating disease,” said Lisa Deschamps, CEO, AviadoBio. “People living with FTD-GRN and their families remain at the heart of everything we do. As we continue this vital work, we look forward to sharing clinical updates.”

About ASPIRE-FTD

ASPIRE-FTD is a Phase 1/2 open-label, multi-center study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, eligible patients receive a one-time administration of AVB-101 delivered as a set of MRI-guided infusions into the thalamus during a minimally invasive stereotactic neurosurgical procedure at an expert neurosurgical center in the US, or EU.

More information about the ASPIRE-FTD study can be found at www.aspire-ftd.com or https://clinicaltrials.gov/study/NCT06064890.

About AVB-101

An investigational gene therapy, AVB-101 contains a correct (non-mutated) version of the GRN gene. It is designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. AVB-101 is delivered as a one-time infusion directly into the brain via a minimally invasive neurosurgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center. AVB-101 has been granted orphan designation by the U.S. Food and Drug Administration (FDA) and European Commission. AviadoBio and Astellas Pharma Inc., have entered into an option agreement for a worldwide exclusive license for the development and commercial rights to AVB-101 in FTD-GRN and other indications.

About AviadoBio

At AviadoBio, we are relentlessly chasing cures by translating groundbreaking science and precision delivery into life-changing medicines for people living with neurological conditions. With our deep understanding of the brain and suite of proprietary gene therapy platforms and delivery technologies, AviadoBio is working to overcome the challenges of delivering the right drug to the right place. Its innovative, neuroanatomy-led approach is designed to maximize the therapeutic potential of gene therapy to halt or potentially reverse neurodegenerative diseases. AviadoBio was founded on pioneering research from King’s College London and the UK Dementia Research Institute and has a leadership team with extensive gene therapy development, delivery, and commercialization experience which uniquely positions the company for success in bringing transformative medicines to patients.

AviadoBio’s investors include New Enterprise Associates (NEA), Monograph Capital, F-Prime Capital, Johnson & Johnson Innovation – JJDC, Inc., SV Health Investor’s Dementia Discovery Fund (DDF), Advent Life Sciences, EQT Life Sciences (Dementia Fund), LifeArc Ventures, and Astellas Pharma.

For more information, please visit www.aviadobio.com and follow us on X @AviadoBio and LinkedIn at AviadoBio.

 

# # #

Contact For Media Enquiries

Farah Speer
SVP, Head of Communications and External Relations
fspeer@aviadobio.com
+1-312-543-2881

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