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Jun 22
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Pheno Therapeutics granted FDA IND clearance for lead multiple sclerosis therapeutic candidate PTD802

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

 

PRESS RELEASE

22 June 2026

Pheno Therapeutics granted FDA IND clearance for lead multiple sclerosis therapeutic candidate PTD802

  • US FDA clearance enables first-in-human study of PTD802, a selective GPR17 antagonist
  • Small molecule therapeutic designed to promote remyelination in neurological diseases such as multiple sclerosis
  • Milestone supports Pheno Therapeutics’ clinical development strategy

Edinburgh, UK, 22 June 2026: Pheno Therapeutics Limited. (Pheno), a clinical stage biotechnology company focused on the discovery and development of small molecule therapeutics for the treatment of neurological diseases, today announced that the US Food and Drug Administration (FDA) has cleared the  Investigational New Drug (IND) application for PTD802, the Company’s lead therapeutic candidate. The clearance allows Pheno to proceed with a first-in-human clinical trial of PTD802 in the US.

The announcement follows the Company’s clinical trial authorisation (CTA) from the UK’s MHRA in January 20251. The first-in-human study would evaluate the safety and tolerability of PTD802 in healthy volunteers. PTD802 is a novel small molecule therapeutic designed to promote remyelination, with an initial focus on multiple sclerosis (MS), a neurological disease with high unmet medical need.

Developed under an exclusive worldwide licence from UCB, PTD802 is a selective GPR17 (G protein-coupled receptor 17) antagonist, and is the first GPR17 programme to receive IND clearance, delivering a new class of neuroprotective therapeutics. This development also supports Pheno Therapeutics’ IP position in the US.

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

Fraser Murray, PhD, CEO, Pheno Therapeutics, said: “FDA IND clearance is an important milestone for our PTD802 programme, and a step further toward our ultimate goal of providing an effective treatment for neurological diseases associated with demyelination. As the first company to gain approval to begin clinical trials for a selective GPR17 antagonist, we are proud to be leading the way, and believe this approach has the potential to offer real patient benefit, in MS and beyond.”

 

[1]: Pheno Therapeutics granted clinical trial authorisation for lead multiple sclerosis therapeutic candidate PTD802 – Pheno Therapeutics

ENDS

 

Contact: 

Zyme Communications

Katie Odgaard

Katie.odgaard@zymecommunications.com

 

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About Pheno Therapeutics

Pheno Therapeutics is a biotechnology company, backed by leading biotech investors Advent Life Sciences and LifeArc, focused on the discovery and development of small molecule therapeutics that boost the function of human oligodendrocytes and their natural ability to promote remyelination, for the treatment of neurological diseases with high unmet medical need such as multiple sclerosis (MS).

Pheno combines world leading expertise in myelin biology, MS trials, patient selection and clinical cohorts. MS is a devastating chronic disease with significant individual and societal impact that often manifests in young adults and is associated with a wide range of neurological symptoms which can progress to total physical and cognitive disability. MS demyelination occurs when the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibres in the central nervous system, leading to multifocal demyelination, axonal injury and neurodegeneration.

Pheno Therapeutics is seeking to promote remyelination and reverse the critical demyelination aspect of MS.

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

 

Jun 18
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F2G and Shionogi Announce Positive Topline Results from Global Phase 3 OASIS Study Evaluating Oral Olorofim Versus AmBisome® Followed by Standard of Care in Patients with Invasive Aspergillosis

By F2G, Press Release, Private Companies
Press Release.

 

F2G and Shionogi Announce Positive Topline Results from Global Phase 3 OASIS Study Evaluating Oral Olorofim Versus AmBisome® Followed by Standard of Care in Patients with Invasive Aspergillosis

  • The study achieved the primary endpoint of non-inferiority, with a rate of all-cause mortality (ACM) at Day 42 for olorofim of 23.8% and for AmBisome® followed by standard of care of 24.3%
  • No new safety findings were observed
  • The rate of drug-related treatment-emergent adverse events (TEAEs) was 35.8% for olorofim and 63.9% for AmBisome® followed by standard of care
  • The study enrolled patients with invasive aspergillosis whose infection was either refractory to or unsuitable for azole therapy
  • If approved, olorofim will be the first novel mechanism agent for the treatment of invasive aspergillosis in over 20 years 1,2

MANCHESTER, United Kingdom, OSAKA, Japan – June 18, 2026 – F2G Ltd and Shionogi & Co., Ltd (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D) (“Shionogi”) today announced positive topline results from the global Phase 3 OASIS study (NCT05101187), comparing the investigational oral antifungal drug olorofim versus AmBisome® (liposomal amphotericin B for injection) followed by standard of care (SOC) in patients with invasive aspergillosis whose infection is either refractory to or unsuitable for azole therapy.

The study met its primary endpoint of non-inferiority, with a rate of all-cause mortality (ACM) at Day 42 for olorofim of 23.8% vs. 24.3% for AmBisome® followed by SOC (difference of -0.5% with 95% confidence interval of -13.1 to 10.8%). No new safety findings were observed for olorofim; the rate of drug-related treatment-emergent adverse events (TEAEs) was 35.8% for olorofim and 63.9% for AmBisome® followed by standard of care, with the difference mainly driven by the higher rate of renal events in the AmBisome arm.

These results expand on the previous Phase 2b study data that led to olorofim’s Breakthrough Therapy Designations from the U.S. Food and Drug Administration (FDA), reinforcing olorofim’s potential as a treatment for patients with invasive aspergillosis1,3. F2G and Shionogi plan to present pivotal results from the study at a future medical congress. These data will be submitted to regulatory authorities in the U.S. by F2G and in Europe and Asia by Shionogi.

“Invasive fungal infections remain difficult to treat and can be life-threatening especially in immunocompromised patients,” said Johan Maertens, MD, PhD, Professor of Hematology at University Hospitals Leuven, Belgium, the study’s Principal Investigator. “The OASIS topline results add to the growing body of evidence supporting olorofim’s therapeutic potential in a hard-to-treat population with limited antifungal options. We’re hopeful this could offer a meaningful alternative for clinicians to treat challenging infections caused by Aspergillus.”

Francesco Maria Lavino, Chief Executive Officer of F2G, commented, “We are encouraged by the topline results from the Phase 3 OASIS study, which represent an important milestone for F2G and for our collaboration with Shionogi. These findings demonstrate the potential for olorofim to serve as a new option for patients with difficult to treat invasive fungal infections, including invasive aspergillosis. We are grateful to the patients, investigators, our partner Shionogi and to the F2G team for their dedication to this pivotal study.”

Dr John Keller, Director of the Board, Senior Vice President, R&D, Shionogi & Co., Ltd., added, “This is a promising new development in antifungal medicine – an area where patients have been under-served for more than 20 years. In current clinical practice, safety and tolerability considerations, particularly effects on renal function, can pose significant challenges for treatment selection and continuation. Against this background, the results of the OASIS study suggest that olorofim has the potential to offer a new treatment option for patients with invasive aspergillosis.” Dr Keller continued: “As a leader in infectious disease R&D, our partnership with F2G for a new class of antifungal treatment supports our commitment to advancing innovation across all anti-infective medicine for some of the most challenging disease areas. We look forward to sharing the full results in due course.”

Invasive aspergillosis is a life-threatening fungal infection that primarily affects immunocompromised patients and is associated with substantial morbidity and mortality[4]. Treatment options are limited for patients who cannot be treated with available azole antifungal therapies1.

F2G and Shionogi are collaborating to develop and commercialize olorofim and bring this novel antifungal therapy to patients with invasive fungal infections. F2G has commercial responsibility for olorofim in North America and non-Shionogi territories, and Shionogi has commercial responsibility for olorofim in Europe and Asia.

About the Phase 3 OASIS Study
The Phase 3 OASIS (Olorofim Aspergillus Infection Study) trial (NCT05101187) was a global, randomized study that evaluated the efficacy and safety of olorofim versus AmBisome® followed by standard of care in adult patients with invasive aspergillosis whose infection is either refractory to or unsuitable for azole therapy. OASIS was designed as a non-inferiority study using a non-inferiority margin of 20% comparing outcomes in 225 subjects randomized 2:1 to olorofim or AmBisome-based standard of care. Invasive aspergillosis is a life-threatening fungal infection with limited treatment options due to rising drug resistance and toxicity concerns1,2,4.  The study’s primary endpoint was all-cause mortality at Day 42, with additional measures of clinical response, safety, and quality of life.

About Olorofim
Olorofim (formerly, F901318) is F2G’s leading candidate from the orotomide class and has been studied in a recently completed global Phase 3 trial (“OASIS”, NCT05101187). Olorofim is a first-in-class antifungal with a novel mechanism of action, oral dosing, and activity against a wide range of Aspergillus species, including strains that are resistant to currently approved agents1. If approved based on the Phase 3 OASIS data, olorofim will be the first novel mechanism agent for invasive aspergillosis in more than 20 years1,2.

In the U.S., olorofim has received orphan drug status from the FDA for the treatment of invasive aspergillosis, scedosporiosis, invasive scopulariopsis, and coccidioidomycosis[5]. Olorofim has been granted Qualified Infectious Disease Product (QIDP) designation for several invasive fungal infections, including invasive aspergillosis and coccidioidomycosis1.

Additionally, olorofim has also received two Breakthrough Therapy designations (BTD) from the FDA1. The first BTD was for the treatment of invasive mold infections in patients with limited or no treatment options, including aspergillosis refractory or intolerant to currently available therapy, and infections due to Lomentospora prolificans, Scedosporium and Scopulariopsis species1. The second BTD was for the treatment of central nervous system (CNS) coccidioidomycosis refractory or otherwise unable to be treated with standard of care therapy1.

In Europe, olorofim has been granted orphan designation from the European Medicines Agency for the treatment of invasive aspergillosis, scedosporiosis, and invasive scopulariopsis1.

Olorofim is an investigational therapy and has not been approved by any regulatory authorities.

About F2G
F2G is a clinical-stage biopharmaceutical company with operations in the UK, US, and Austria focused on the discovery and development of novel therapies to treat potentially life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides, which selectively target a key enzyme in the pyrimidine biosynthesis pathway, a novel mechanism of action that is distinct from currently marketed antifungal agents. This mechanism provides the orotomides with fungicidal activity against a broad range of rare and resistant fungal mould infections. For more information, please visit: www.f2g.com

About Shionogi & Co., Ltd.
Shionogi & Co., Ltd. is a 148-year-old global, research-driven pharmaceutical company headquartered in Osaka, Japan, that is dedicated to bringing benefits to patients based on its corporate philosophy of “supplying the best possible medicine to protect the health and wellbeing of the patients we serve.” The company currently markets products in several therapeutic areas including anti-infectives, pain, CNS disorders. Shionogi’s research and development currently targets two therapeutic areas: infectious diseases and diseases with unmet medical needs in pain/CNS, including Alzheimer’s disease, oncology, rare diseases, and sleep apnea. For more information on Shionogi & Co., Ltd., please visit https://www.shionogi.com/global/en.

Shionogi Forward-Looking Statements 
This announcement contains forward-looking statements. These statements are based on expectations in light of the information currently available, assumptions that are subject to risks and uncertainties which could cause actual results to differ materially from these statements. Risks and uncertainties include general domestic and international economic conditions such as general industry and market conditions, and changes of interest rate and currency exchange rate. These risks and uncertainties particularly apply with respect to product-related forward-looking statements. Product risks and uncertainties include, but are not limited to, completion and discontinuation of clinical trials; obtaining regulatory approvals; claims and concerns about product safety and efficacy; technological advances; adverse outcome of important litigation; domestic and foreign healthcare reforms and changes of laws and regulations. Also, for existing products, there are manufacturing and marketing risks, which include, but are not limited to, inability to build production capacity to meet demand, lack of availability of raw materials and entry of competitive products. The company disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events or otherwise.

Shionogi Media contacts:
SHIONOGI Website Inquiry Form: https://www.shionogi.com/global/en/contact.html
Shionogi Europe Press Office: pressoffice@shionogi.eu

F2G Media Contact:
Luke Shiplo
LifeSci Communications
F2GMedia@lifescicomms.com

AmBisome® is a registered trademark of Gilead Sciences, Inc. All other trademarks are the property of their respective owners.

References:

1 Vanbiervliet, Y., Van Nieuwenhuyse, T., Aerts, R. et al. Review of the novel antifungal drug olorofim (F901318). BMC Infect Dis 24, 1256 (2024). https://doi.org/10.1186/s12879-024-10143-3
2 Oliver et al. (2016). F901318 represents a novel class of antifungal drug that inhibits dihydroorotate dehydrogenase. Proceedings of the National Academy of Sciences of the United States of America, 113(45), 12809–12814. https://doi.org/10.1073/pnas.1608304113
3 Maertens JA, Thompson GR 3rd, Spec A, et al. Olorofim for the treatment of invasive fungal diseases in patients with few or no therapeutic options: a single-arm, open-label, phase 2b study. Lancet Infect Dis. 2025;25(11):1177-1188. doi:10.1016/S1473-3099(25)00224-5)
4 Centers for Disease Control and Prevention. Data and statistics on aspergillosis. https://www.cdc.gov/aspergillosis/statistics/index.html (Accessed June 2026)
5 US Food & Drug Administration. Orphan drug designations and approvals for olorofim. Available at: https://www.accessdata.fda.gov/scripts/opdlisting/oopd/listResult.cfm (Accessed June 2026)

Jun 08
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Amphista Therapeutics announces FDA clearance of IND application for AMX-883, its lead Targeted Glue™ degrader for acute myeloid leukaemia

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

 

Amphista Therapeutics announces FDA clearance of IND application for AMX-883, its lead Targeted Glue™ degrader for acute myeloid leukaemia

June 8, 2026

  • Amphista to initiate its first clinical trial for AMX-883, a potent, orally bioavailable DCAF16-dependent degrader of BRD9, for acute myeloid leukaemia (AML) in H2 2026
  • The Phase 1 monotherapy dose-escalation and optimisation trial will enrol patients with relapsed or refractory AML and high-risk myelodysplastic syndrome
  • As a broad-acting, pro-differentiation agent, AMX-883 acts independently of karyotype status and has the potential to benefit a wider population of AML patients than current targeted therapies

Cambridge, UK, 8 June 2026  – Amphista Therapeutics (“the Company” or “Amphista”), a leader in the discovery and development of next generation targeted protein degradation (TPD) medicines, today announces that the U.S. Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for AMX-883, an orally available non-cereblon degrader of BRD9, for the treatment of acute myeloid leukaemia (AML). AMX-883 is expected to enter the clinic in H2 2026.

IND clearance allows Amphista to start its Phase 1 monotherapy dose-escalation and optimisation clinical trial of AMX-883 in patients with relapsed or refractory AML and high-risk myelodysplastic syndrome (MDS), a related bone marrow disorder that often progresses to AML. After establishing the monotherapy profile, the Company intends to explore AMX-883 in combination with venetoclax and azacitidine in early lines of therapy, where treatment resistance continues to pose a major clinical challenge.

Louise Modis, Chief Executive Officer at Amphista, said: “FDA clearance of our IND for AMX-883, our lead Targeted Glue™, for acute myeloid leukaemia is a significant milestone as we transition into a clinical-stage company. AMX-883 is the only BRD9 degrader currently being developed and the compelling preclinical findings submitted to the FDA support its potential as a first-line treatment option in the earlier disease setting in one of the most aggressive blood cancers. We look forward to commencing the clinical trial in H2 2026.”

Patrick Kelly, Chief Medical Officer at Amphista, added: “AML remains one of the most devastating blood cancers, with a 5-year survival rate of just 33%, and resistance to standard-of-care treatments like venetoclax a critical challenge. FDA clearance of our IND for AMX-883 supports the advancement of a differentiated therapeutic approach, with the potential to establish an important new treatment pathway for patients in urgent need of innovative therapies.”

Ends

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases, including cancer and neurodegenerative disorders, through the discovery of advanced, next-generation targeted protein degradation (TPD) medicines. Using its proprietary Eclipsys® platform, Amphista develops unique bifunctional Targeted Glue™ therapeutics with a differentiated mechanism and leading drug-like properties. Its portfolio has the potential to deliver first- and/or best-in-class therapeutics with performance characteristics beyond the limitations of CRBN- and VHL-based agents. Amphista was co-founded by Advent Life Sciences and is additionally funded by a premier group of investors including Forbion, Gilde Healthcare, Novartis Venture Fund, SV’s Dementia Discovery Fund and Eli Lilly. For more information, please visit: www.amphista.com

Amphista, Eclipsys, Targeted Glue, Targeted Glues and the Amphista logo are all trademarks or registered trademarks of Amphista Therapeutics Limited.

About BRD9 and Acute Myeloid Leukaemia

Acute myeloid leukaemia (AML) is one of the most aggressive blood cancers. Despite the availability of anti-proliferative treatments, survival remains poor, with a 5-year survival rate of just 33%, and AML is estimated to cause around 130,000 deaths globally each year. The disease is characterised by a differentiation block that prevents normal myeloid cell maturation and leads to a build-up of immature cells. Therapies that remove this block and allow AML blasts to mature—including ATRA, FLT-3 inhibitors and, more recently, menin inhibitors—have shown clinical benefit in certain AML subtypes. However, there remains a pressing need for broader-acting therapies that can benefit patients regardless of genetic profile.

BRD9 is a subunit of the non-canonical BAF complex and plays an important structural and functional role in regulating chromatin structure and maintaining genomic stability in AML. Degradation of BRD9 removes the differentiation block and drives the differentiation and death of AML blasts.

About AMX-883

AMX-883 is an orally bioavailable, potent and rapid degrader of BRD9. It uses Amphista’s proprietary Targeted Glue™ technology to drive BRD9 degradation through recruitment of DCAF16, a mechanism distinct from cereblon- or VHL-based PROTACs. AMX-883 achieves near complete degradation of BRD9 within two hours of treatment, while retaining exquisite selectivity over all other bromodomain containing proteins, and beyond, as shown by global proteomics.

Amphista’s preclinical data package supports the advancement of AMX-883 into clinical development as a karyotype-independent, pro-differentiation agent, with the first trial planned for H2 2026. In patient-derived tumour models, AMX-883-induced BRD9 degradation is sufficient to inhibit tumour growth in vivo as a monotherapy, while in combination AMX-883 shows synergistic efficacy with venetoclax in vivo and prevents the emergence of venetoclax resistance in vitro, addressing a major clinical challenge in acute myeloid leukaemia.

For more information please contact:

Amphista Therapeutics
John Goodall
Email: Info@amphista.com

ICR Healthcare
Namrata Taak, Ashley Tapp, Emily Johnson
Email: Amphista@icrhealthcare.com
Tel: +44 (0)20 3709 5813

May 04
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Aura Biosciences Announces Pricing of $260 Million Public Offering of Common Stock and Pre-funded Warrants

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

 

Aura Biosciences Announces Pricing of $260 Million Public Offering of Common Stock and Pre-funded Warrants

BOSTON, May 04, 2026 (GLOBE NEWSWIRE) — Aura Biosciences, Inc. (“Aura”) (Nasdaq: AURA), a clinical-stage biotechnology company developing precision therapies for solid tumors designed to preserve organ function, today announced the pricing of an underwritten public offering consisting of (i) 39,591,000 shares of its common stock and (ii) in lieu of common stock to certain investors, pre-funded warrants to purchase an aggregate of up to 3,800,000 shares of its common stock at an exercise price of $0.00001 per pre-funded warrant. In addition, Aura has granted the underwriters a 30-day option to purchase up to an additional 6,508,650 shares of its common stock on the same terms and conditions. The offering price of each share of common stock is $6.00. The offering price of each pre-funded warrant is $5.99999.

All of the shares and pre-funded warrants in the offering are being sold by Aura. The gross proceeds from the offering to Aura are expected to be $260.3 million, before deducting underwriting discounts and commissions and other offering expenses and excluding any exercise of the underwriters’ option to purchase additional shares. The offering is expected to close on or about May 5, 2026, subject to customary closing conditions.

Aura intends to use $205.1 million of the net proceeds from the offering, together with existing cash, cash equivalents and marketable securities, to advance its clinical programs, including in early choroidal melanoma, and for registration-enabling activities for bel-sar, as well as for general corporate purposes. Aura plans to use the remaining net proceeds from the offering to repurchase up to 6,922,870 shares from its stockholder, Matrix Capital Management Master Fund, LP (“Matrix”), which represents all shares of Aura’s common stock held by Matrix, at $5.64 per share, which is the price per share at which the underwriters will purchase shares of Aura’s common stock from Aura in the offering.

Leerink Partners, TD Cowen and Evercore ISI are acting as joint bookrunning managers for the offering. LifeSci Capital is also acting as a bookrunning manager in the offering. Citizens Capital Markets is acting as a co-manager for the offering.

A shelf registration statement relating to the shares of common stock and pre-funded warrants offered in the public offering described above was filed with the Securities and Exchange Commission (the “SEC”) on March 27, 2024 and declared effective by the SEC on April 5, 2024 and a related registration statement that was filed with the SEC on May 4, 2026 pursuant to Rule 462(b) under the Securities Act of 1933, as amended (and became automatically effective upon filing). The offering is being made only by means of a written prospectus and prospectus supplement that form a part of the registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering were filed with the SEC on May 4, 2026, and are available on the SEC’s website located at www.sec.gov. A final prospectus supplement containing additional information relating to the offering and an accompanying prospectus will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to this offering may also be obtained by contacting: Leerink Partners LLC, Syndicate Department, 53 State Street, 40th Floor, Boston, MA 02109, or by telephone at (800) 808-7525 ext. 6105, or by email at syndicate@leerink.com; TD Securities (USA) LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717 or by email at TDManualrequest@broadridge.com; or Evercore Group L.L.C., Attention: Equity Capital Markets, 55 East 52nd Street, 35th Floor, New York, NY 10055, or by telephone at (888) 474-0200, or by email at ecm.prospectus@evercore.com.

This press release shall not constitute an offer to sell or the solicitation of an offer to buy the securities being offered, nor shall there be any sale of the securities being offered in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About Aura Biosciences

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

Forward-Looking Statements

Various statements in this release concerning the use of proceeds, timing and completion of the public offering and stock repurchase on the anticipated terms or at all may constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of substantial risks and uncertainties, many of which are outside Aura’s control, that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include fluctuations in Aura’s stock price, changes in market conditions and satisfaction of customary closing conditions related to the public offering and stock repurchase, as well as those risks more fully discussed in the section entitled “Risk Factors” in the prospectus supplement and registration statement referenced above, Aura’s Annual Report on Form 10-K for the year ended December 31, 2025, filed on March 30, 2026 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. There can be no assurance that Aura will be able to complete the public offering on the anticipated terms. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Aura undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, unless required by law.

Investor and Media Contact:

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

Source: Aura Biosciences, Inc.

Mar 18
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Amphista Therapeutics appoints Louise Modis Ph.D. as CEO and Patrick Kelly M.D. as CMO

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

 

Amphista Therapeutics appoints Louise Modis Ph.D. as CEO and Patrick Kelly M.D. as CMO and provides a business update as the Company prepares to file IND application for AMX-883

                                                                                                              March 18, 2026

  • Strengthening the clinical leadership as the Company progresses to the clinic with its BRD9 Targeted Glue™, AMX-883, in acute myeloid leukemia
  • Amphista continues to advance its SMARCA and TEAD programs to key inflection points

Cambridge, UK, 18th March 2026 – Amphista Therapeutics Ltd. (the “Company” or “Amphista”), a leader in the discovery and development of next generation targeted protein degradation (TPD) medicines, today announces the appointment of Louise Modis, Ph.D., as its Chief Executive Officer (CEO) and member of the Board of Directors, and Patrick Kelly M.D. as its Chief Medical Officer (CMO). Dr. Modis succeeds Antony Mattessich who is retiring.

Dr. Modis joined Amphista in May 2023 as Chief Scientific Officer. She is an accomplished leader who brings a combination of deep scientific expertise and strategic focus to the role, gained from over two decades of experience advancing innovative medicines into the clinic across biotech and pharma. She has been instrumental in driving Amphista’s progress to date, having defined the Company’s R&D strategy and building its strong pipeline. Dr. Modis will continue to provide scientific leadership while guiding the Company’s strategic direction.

Dr. Kelly joins Amphista and strengthens the clinical leadership as the Company enters the clinic with AMX-883 in acute myeloid leukemia (AML). He brings 35 years of clinical research and clinical practice experience, most recently serving as CMO at Forma Therapeutics where he led the development of olutasidenib, which is now approved for the treatment of IDH1-mutated AML. Dr. Kelly will play a pivotal role in leading the clinical development team and shaping Amphista’s development strategy across the pipeline.

Joshua Brumm, Chairman of Amphista’s Board of Directors said: “The Board is delighted to announce the appointments of Louise as CEO and Patrick as CMO. We have been impressed with Louise’s vision and execution of Amphista’s science strategy, and her appointment reflects our confidence in her leadership as we transition to be a clinical-stage company. Combined with Patrick’s clinical expertise, Amphista is well positioned to successfully deliver our differentiated pipeline to patients. On behalf of the Board, I would also like to thank Antony for his contributions and wish him the best in his retirement.”

Louise Modis, Chief Executive Officer of Amphista, commented: “I’m honored to lead Amphista. As CEO, my priority is to ensure we deliver clinical excellence for AMX-883 and to accelerate our earlier programs into the clinic. With the addition of Patrick as CMO, and the strength of our leadership team, we are well positioned to execute our clinical strategy and advance our BRD9, SMARCA, and TEAD programs for patients and their families.”

Patrick Kelly, Chief Medical Officer of Amphista, commented, “I am excited to join Amphista as the Company prepares to enter the clinic with AMX-883 on the back of impressive pre-clinical data. I look forward to working with Louise and the team to deliver the AMX-883 clinical program in AML and building a robust clinical strategy across the broader pipeline.”

As a karyotype-independent, pro-differentiation agent, AMX-883 has the potential to transform the treatment paradigm for AML. Amphista expects to submit the Investigational New Drug application to the US Food and Drug Administration for AMX-883 in April and to start its clinical trial in H2 2026.

Amphista is developing potent, selective, orally bioavailable degraders of SMARCA2 and TEAD. The Company remains on track to deliver a shortlist of best-in-class compounds later this year for further profiling ahead of candidate nomination.

 

About Amphista Therapeutics

At Amphista Therapeutics, we are focused on transforming the lives of patients with severe diseases, including cancer and neurodegenerative disorders, through the discovery and development of advanced, next generation targeted protein degradation (TPD) medicines. Amphista applies its proprietary Eclipsys® platform to generate unique, sequentially bifunctional Targeted Glue™ therapeutics with a differentiated mechanism and leading drug-like properties. Our portfolio offers the potential to deliver first- and/or best-in-class therapeutics with performance characteristics beyond the limitations of CRBN and VHL-based agents. Amphista was co-founded by Advent Life Sciences and is additionally funded by a premier group of investors including Forbion, Gilde Healthcare, Novartis Venture Fund, SV Health Investors’ Dementia Discovery Fund and Eli Lilly. For more information, please visit: www.amphista.com

Amphista, Eclipsys, Targeted Glue, Targeted Glues and the Amphista logo are all trademarks or registered trademarks of Amphista Therapeutics Limited.

 

For more information please contact:

Amphista Therapeutics
John Goodall
Email: Info@amphista.com

 

ICR Healthcare
Namrata Taak, Ashley Tapp, Emily Johnson
Email: Amphista@icrhealthcare.com
Tel: +44 (0)20 3709 5813

Jan 20
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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

  | Source: Levicept

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.

Jan 08
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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

 

 

Oct 25
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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
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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
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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