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Private Companies

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.

May 01
Love1

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

By AviadoBio, Press Release, Private Companies
Press Release.

 

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

Clinical Trial Studying AVB-101 for FTD-GRN

 AVB-101 Biomarker Data Readout Expected in 2026

 Clinical Milestone Shared at AFTD Annual Caregiver Conference

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

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

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

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

About ASPIRE-FTD

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

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

About AVB-101

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

About AviadoBio

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

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

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

 

# # #

Contact For Media Enquiries

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

Apr 16
Love0

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

By Glycomine, Press Release, Private Companies
Press Release.

 

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

 

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

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

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

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

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

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

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

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

 About PMM2-CDG

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

 About Glycomine, Inc.

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

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

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

 

Mar 24
Love0

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

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

 

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

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

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

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

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

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

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

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

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

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

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

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

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

Virtual Urologic Oncology Investor Event

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

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

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

About Aura Biosciences

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

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

Forward-Looking Statements

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

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

Investor and Media Contact:

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

Mar 07
Love0

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

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

 

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

 

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

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

 

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

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

ePoster presentation

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

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

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

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

About Artax Biopharma

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

 

About Nck modulation

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

 

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

 

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

                                                                #  #  # 

 

Contacts: 

Maria Nichol, DPhil, EPA, CPA 

Chief Business Officer 

mnichol@artaxbiopharma.com 

 

Media: 

Madelin Hawtin

LifeSci Communications

mhawtin@lifescicomms.com