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Levicept Announces FDA Acceptance of IND Application for LEVI-04, a Potentially Disease-Modifying Treatment for Osteoarthritis

By Levicept, Press Release, Private Companies
Press Release.

 

Levicept Announces FDA Acceptance of IND Application for LEVI-04, a Potentially Disease-Modifying Treatment for Osteoarthritis


SANDWICH, United Kingdom, Jan. 20, 2026 (GLOBE NEWSWIRE) — Levicept Ltd, a biotechnology company focused on the development of LEVI-04, a first-in-class treatment for osteoarthritis (OA), today announces the US Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for LEVI-04. This enables the initiation of further clinical development as a potentially disease-modifying treatment for osteoarthritis.

Levicept has completed a successful 518-participant Phase II study of LEVI-04 in patients with pain and disability due to OA of the knee (ClinicalTrials.gov ID: NCT05618782). Data presented at ACR Convergence, showed the primary endpoint of the trial was met with significant analgesia and symptom control across all doses, with a favourable safety and tolerability profile.

Further data analysis shared at ACR Convergence has shown that LEVI-04 had positive dose-dependent effects on the size and presence of bone marrow lesions, compared to placebo. Reduction in bone marrow lesions was significantly correlated with improvements in OA symptom scores, suggesting LEVI-04 may have disease modification properties in addition to the analgesic properties already reported.

Levicept is now planning for clinical studies in a broader participant population and to allow further data gathering on long-term dosing.

Eliot Forster, CEO of Levicept, said: “This new IND is an important step in our further development of LEVI-04 with the potential to treat millions of OA patients in need worldwide. We have already demonstrated LEVI-04 has a unique profile in its potential to significantly reduce pain and also directly address the disease process. We look forward to the next steps in our clinical programme and further building the data package around this important molecule.”

Levicept

Eliot Forster, CEO – eliot@levicept.com

Media Enquiries

Charles Consultants

Sue Charles – Sue@charles-consultants.com +44 (0)7968 726585

Chris Gardner – Chris@CGComms.onmicrosoft.com +44 (0)7956 031077

About Levicept – www.levicept.com

Levicept Ltd is a UK-based biotechnology company developing the first in a new class of novel, safe and efficacious biological therapies, LEVI-04 [p75NTR-Fc], for the treatment of osteoarthritis and chronic pain. LEVI-04 inhibits NT-3, one of the neurotrophin family of proteins. LEVI-04 has completed a Phase II clinical trial in more than 500 patients with osteoarthritis. It is estimated that the market opportunity for drugs that treat osteoarthritis is worth in excess of $10 billion. LEVI-04 was discovered by Levicept’s founder, Simon Westbrook. Levicept’s investors include Medicxi, Advent Life Sciences, Gilde Healthcare and Pfizer Ventures.

Beacon Therapeutics Announces Closing of Oversubscribed Series C Financing for Over $75 Million

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

 

Beacon Therapeutics Announces Closing of Oversubscribed Series C Financing for Over $75 Million

 

  • Round led by new investor Life Sciences at Goldman Sachs Alternatives with participation by new investor the Retinal Degeneration Fund, and supported by existing investors
  • Funds will advance laru-zova toward commercialization for treatment of X-linked retinitis pigmentosa and accelerate development of additional pipeline candidates

 

LONDON and CAMBRIDGE, Mass., Jan. 8, 2026 – Beacon Therapeutics Holdings Limited (‘Beacon Therapeutics’ or ‘the Company’), a leading clinical-stage biotechnology company with a mission to save and restore vision in people with rare and prevalent ocular diseases, today announced that it has raised over $75 million in an oversubscribed Series C financing led by Life Sciences at Goldman Sachs Alternatives and with participation from the Retinal Degeneration Fund (RD Fund), the venture arm of Foundation Fighting Blindness. Beacon’s existing investors, Syncona Limited, Forbion, Oxford Science Enterprises, and Advent Life Sciences, also participated in the round.

“This significant fundraising, led by new blue-chip investor Life Sciences at Goldman Sachs Alternatives, validates our strategy to save and restore sight for people living with rare and prevalent ocular diseases,” said Lance Baldo, MD, Chief Executive Officer of Beacon Therapeutics. “With pivotal laru-zova data expected in the second half of 2026, these funds will accelerate our commercial preparations for this potentially life-changing product, as well as advance and expand our pipeline. We thank our new and existing investors for their confidence in our mission to deliver lasting impact in blinding ocular diseases.”

Beacon Therapeutics also appointed Colin Walsh, PhD, Managing Director, Life Sciences at Goldman Sachs Alternatives, to its Board of Directors.

“We are excited to work with Beacon Therapeutics at this critical time to support the development of laru-zova as a potential game-changing ocular gene therapy,” added Colin Walsh, PhD, Managing Director, Life Sciences at Goldman Sachs Alternatives. “Beacon has deep experience in gene therapy and its application in ocular disease. With laru-zova’s advancement through pivotal trials and the Company’s expanding pipeline, the Company is setting the roadmap for how to build successful gene therapies and bring transformative treatments to patients with X-linked retinitis pigmentosa (XLRP).”

“RP affects over 100,000 patients in the US, with 14% of those estimated to have XLRP. With no available treatment options, halting XLRP progression and restoring patients’ vision would represent a life-changing breakthrough. We are proud to support Beacon as it approaches the pivotal VISTA trial readout of laru-zova later this year,” commented Rusty Kelley, PhD, MBA, Managing Director of the RD Fund.

The new funds will be used to complete the development of lead program, laru-zova, a potential best-in-class gene therapy currently being investigated for the treatment of patients with XLRP, and progress commercialization plans. The funds will also be used to help support the development of Beacon’s pipeline candidates for geographic atrophy, as well as an inherited cone rod dystrophy, and another undisclosed asset.

44th Annual JP Morgan Healthcare Conference presentation

The Company will present at the 44th Annual JP Morgan Healthcare Conference being held January 12-15, 2026, in San Francisco, US.

The presentation is scheduled for Tuesday, January 13th at 2:30 PM PT.

About Beacon Therapeutics

Beacon Therapeutics is a clinical-stage biotechnology company dedicated to saving and restoring sight for people living with rare and prevalent ocular diseases. The Company is harnessing the transformative power of gene therapy to deliver the most meaningful outcomes for severe ocular diseases. Beacon’s pipeline currently targets devastating blinding retinal diseases such as X-linked retinitis pigmentosa (XLRP) and geographic atrophy.

Beacon Therapeutics’ investors include Advent Life Sciences, Forbion, Life Sciences at Goldman Sachs Alternatives, Oxford Science Enterprises, Retinal Degeneration Fund, Syncona Limited, and TCGX, among others. Learn more about Beacon Therapeutics at beacontx.com and follow on LinkedIn for more updates.

 

About Life Sciences at Goldman Sachs Alternatives

Goldman Sachs (NYSE: GS) is one of the leading investors in alternatives globally, with over $500 billion in assets and more than 30 years of experience. The business invests in the full spectrum of alternatives including private equity, growth equity, private credit, real estate, infrastructure, sustainability, and hedge funds. Clients access these solutions through direct strategies, customized partnerships, and open-architecture programs.

The business is driven by a focus on partnership and shared success with its clients, seeking to deliver long-term investment performance drawing on its global network and deep expertise across industries and markets.

Life Sciences at Goldman Sachs Alternatives was established in 2021 and focuses on later-stage venture investments, targeting therapeutic companies in early clinical development with multi-asset portfolios. The Life Sciences team bring decades of experience investing in the sector and leverage the expansive resources of the Goldman Sachs platform to source differentiated investments and partner with companies to enhance value creation.

The alternative investments platform is part of Goldman Sachs Asset Management, which delivers investment and advisory services across public and private markets for the world’s leading institutions, financial advisors and individuals. Goldman Sachs has approximately $3.5 trillion in assets under supervision globally as of September 30, 2025.

 

About Retinal Degeneration Fund

The Retinal Degeneration Fund (RD Fund) is a mission-driven venture fund established by the Foundation Fighting Blindness to invest in companies developing innovative therapies for inherited retinal diseases and dry age-related macular degeneration. The RD Fund was designed to bridge the funding gap between discovery research and confirmatory clinical trials. For more information, visit www.RDFund.org.

Contact:
info@beacontx.com

Media & Investors:
beacon@icrhealthcare.com

 

 

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 differentlyand some can be disruptive/ 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)

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

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

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

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®.

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.

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

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

 

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