KalVista Pharmaceuticals Enters Into Licensing Agreement With Kaken Pharmaceutical to Commercialize Sebetralstat for HAE in Japan




KalVista Pharmaceuticals Enters Into Licensing Agreement With Kaken Pharmaceutical to Commercialize Sebetralstat for HAE in Japan

-Up to $24 million in upfront and milestone payments, plus royalties-

-Sebetralstat has potential to become first, oral on-demand treatment of HAE in Japan, underscoring commercial opportunity-

-Kaken brings regional expertise and proven track record in commercializing innovative therapies-

CAMBRIDGE, Mass. & SALISBURY, England–(BUSINESS WIRE)–KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced that its wholly-owned subsidiary, KalVista Pharmaceuticals, Ltd., has licensed commercialization rights in Japan to Kaken Pharmaceutical, Co., Ltd. (JPX: 4521.T) for sebetralstat, an investigational, oral on-demand treatment for hereditary angioedema (HAE). KalVista will receive an upfront payment of $11 million, with an additional payment of up to $11 million upon achievement of a regulatory milestone anticipated in early 2026. Beyond these payments, the Company is also eligible for commercial milestone payments, plus royalties based on the Japan National Health Insurance (NHI) price, with the royalty rate as a percentage of sales approximately in the mid-twenties.

“We are pleased to partner with Kaken, whose expertise and demonstrated success in the region make them well-suited to work alongside our exceptional team to bring sebetralstat to the HAE community in Japan,” said Ben Palleiko, CEO of KalVista. “This collaboration is an important part of our strategy to expand the global reach of sebetralstat as we prepare for several commercial launches starting this year. Our focus remains on delivering a safe and effective oral on-demand therapy that we believe will make a meaningful difference for people living with HAE worldwide.”

As previously announced, KalVista received Orphan Drug Designation for sebetralstat from Japan’s Ministry of Health, Labour and Welfare (MHLW) and the Company has submitted a New Drug Application (NDA) for sebetralstat in Japan. If approved, sebetralstat would be the first oral on-demand treatment for HAE in the country.

About KalVista Pharmaceuticals, Inc.

KalVista Pharmaceuticals, Inc., is a global biopharmaceutical company dedicated to developing and delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. The Company’s lead investigational product is sebetralstat, a novel, oral, on-demand treatment for hereditary angioedema (HAE). Sebetralstat is under regulatory review by the U.S. FDA, with a PDUFA goal date of June 17, 2025. In addition, KalVista has completed Marketing Authorization Applications for sebetralstat to the European Medicines Agency, the Pharmaceuticals and Medical Devices Agency, and multiple other global regulatory authorities. For more information about KalVista, please visit www.kalvista.com.

About Sebetralstat

Sebetralstat is an investigational, novel oral plasma kallikrein inhibitor for the treatment of hereditary angioedema (HAE). We have filed multiple regulatory applications seeking approval of sebetralstat as the first oral, on-demand treatment for HAE in individuals aged 12 and older, and are investigating its use in children aged 2 to 11. If approved, sebetralstat has the potential to become the foundational therapy for HAE management worldwide.

About Kaken Pharmaceutical, Co., Ltd.

Kaken Pharmaceutical is a specialty pharmaceutical company in Japan with strong experience in developing and commercializing novel pharmaceuticals in the fields of orthopedics and dermatology. Kaken concentrates its R&D resources in areas such as immune system, nervous system, infectious diseases and rare diseases with unmet medical needs. Kaken, in its philosophy, strives to improve the quality of life of patients through the development and distribution of superior pharmaceuticals. For further information, visit www.kaken.co.jp/english.

Forward-Looking Statements

This press release contains “forward-looking” statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “anticipate,” “intend,” “plan,” “goal,” “seek,” “believe,” “project,” “estimate,” “expect,” “strategy,” “future,” “likely,” “may,” “should,” “will,” and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, timings or outcomes of communications with the FDA, our expectations about safety and efficacy of our product candidates and timing of clinical trials and its results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and to obtain regulatory approvals for sebetralstat and other candidates in development, the success of any efforts to commercialize sebetralstat, the ability of sebetralstat and other candidates in development to treat HAE or other diseases, and the future progress and potential success of our oral Factor XIIa program. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on form 10-K for the year ended April 30, 2024, our quarterly reports on Form 10-Q, and our other reports that we make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments, or otherwise.

Contacts

Ryan Baker

Head, Investor Relations

(617) 771-5001

ryan.baker@kalvista.com

Molly Cameron

Director, Corporate Communications

(857) 356-0164

molly.cameron@kalvista.com

Monroe Capital Supports Olympus Partners’ Acquisition of PAI Pharma




Monroe Capital Supports Olympus Partners’ Acquisition of PAI Pharma

CHICAGO–(BUSINESS WIRE)–Monroe Capital LLC (“Monroe”) announced it acted as joint lead arranger on the funding of a senior credit facility to support the acquisition of PAI Pharma (“PAI”) by Olympus Partners.

Founded in 1968 and based in Greenville, SC, PAI is a leading US oral generic liquids manufacturer of ready-to-dose cups and oral liquids bottles. PAI offers the broadest portfolio of generic oral liquid medicines in the industry, supporting retail chains, independent pharmacies, hospitals, long-term care facilities and government agencies.

About Monroe Capital

Monroe Capital LLC (“Monroe”) is a premier asset management firm specializing in private credit markets across various strategies, including direct lending, technology finance, venture debt, alternative credit solutions, structured credit, real estate and equity. Since 2004, the firm has been successfully providing capital solutions to clients in the U.S. and Canada. Monroe prides itself on being a value-added and user-friendly partner to business owners, management, and both private equity and independent sponsors. Monroe’s platform offers a wide variety of investment products for both institutional and high net worth investors with a focus on generating high quality “alpha” returns irrespective of business or economic cycles. The firm is headquartered in Chicago and has 11 locations throughout the United States, Asia and Australia.

Monroe has been recognized by both its peers and investors with various awards including Private Debt Investor as the 2024 Lower Mid-Market Lender of the Year, Americas and 2023 Lower Mid-Market Lender of the Decade; Inc.’s 2024 Founder-Friendly Investors List; Global M&A Network as the 2023 Lower Mid-Markets Lender of the Year, U.S.A.; DealCatalyst as the 2022 Best CLO Manager of the Year; Korean Economic Daily as the 2022 Best Performance in Private Debt – Mid Cap; Creditflux as the 2021 Best U.S. Direct Lending Fund; and Pension Bridge as the 2020 Private Credit Strategy of the Year. For more information and important disclaimers, please visit www.monroecap.com.

Contacts

For more information, please contact:

Zia Uddin

Monroe Capital LLC

312-523-2374

zuddin@monroecap.com

Daniel Abramson

BackBay Communications

857-305-8441

daniel.abramson@backbaycommunications.com

Leyden Labs Publishes New Data Confirming Potential of Intranasal Antibody Approach




Leyden Labs Publishes New Data Confirming Potential of Intranasal Antibody Approach

• In vivo influenza protection was achieved at a significantly lower antibody dose with intranasal compared to systemic administration
• In contrast to what is observed with systemic administration, Leyden Labs’ lead influenza antibody CR9114 has a unique mechanism of protection when administered intranasally
• The unmatched breadth of CR9114 is further supported by the discovery of binding to influenza C and D alongside A and B viruses – an essential attribute for durability of protection against new, emerging strains

LEIDEN, Netherlands–(BUSINESS WIRE)–Leyden Laboratories B.V. (the “Company” or “Leyden Labs”) today announced the publication of new findings that showcase the advantages of intranasal administration of monoclonal antibodies over systemic administration for protection against airborne viruses.

The studies reported in this paper demonstrate the uniqueness of intranasal administration of broadly protective antibodies as a novel, non-vaccine approach to protect against respiratory viral infection. These new insights pave the way towards intranasal antibody delivery to provide protection against influenza and other airborne viruses.

Key Findings:

• Intranasal antibody administration is effective at significantly lower dosages compared to systemic administration. Central to the strategy of Leyden Labs is to deliver broadly protective antibodies directly at the gate of viral entry, namely, to the respiratory mucosa. With these in vivo data, it is demonstrated that intranasal administration of CR9114 is efficacious at significant lower dosages than required for systemic administration. CR9114 is the monoclonal antibody component of the Company’s PanFlu candidate. CR9114 was shown to have a more potent (low dose) protective effect when administered intranasally vs intravenously in mice. Typically, there is a trade-off between potency and breadth in antibody activity: broadly-neutralizing antibodies often are less potent in vitro. This publication however shows that intranasal administration resolves this trade-off in vivo: by administering CR9114 intranasally, low-dose protection is effective.

• Intranasal antibody administration enables protection that is not dependent on Fc-mediated effector functions. CR9114 protection against influenza was shown to be Fc-independent with intranasal administration in vivo. Although Fc-mediated effector functions play a large role in the mechanism of action of systemically administered antibodies, they are not required for efficacy following intranasal administration. Intranasal CR9114 can provide protection through its front-end (Fab) alone, both against strains that are neutralized in vitro and strains that are not neutralized in vitro. Importantly, this observation further highlights the opportunity to redefine criteria for preclinical evaluation of the protective potential of an antibody based on the route of administration.

Clarissa Koch, PhD, Chief Research Officer of Leyden Labs commented, “We are excited to share this great work from our team and collaborators as we continue to expand our understanding about the nuances of administration of protective antibodies directly to the respiratory mucosa. We expect to publish additional data later this year regarding intranasal antibody dynamics as well. Under our Mucosal Protection Platform, findings such as these help in important ways to inform the ongoing development of PanFlu, as well as to shape additional pipeline programs by our team.”

Nigel Temperton, Professor of Molecular Virology and Director of Viral Pseudotype Unit at the Medway School of Pharmacy, University of Kent and University of Greenwich UK, who collaborated with Leyden Labs on these studies, said, “These findings have the potential to meaningfully impact the field of viral prophylaxis using antibodies. Although many valuable efforts have been made over time to optimize antibody composition for prophylactic use, contribution of the route of administration may have been underappreciated.”

Not only does this paper report discoveries about the uniqueness of intranasal administration, it also demonstrates that the breadth of activity of CR9114 is even greater than previously recognized. In addition to its known affinity for influenza A and B subtypes, it has now been shown that CR9114 also binds influenza C and D. Although influenza C and D are less at the forefront of current healthcare concerns – they mostly circulate in cattle and other animals – this finding reinforces the unique breadth of activity of CR9114 achieved by targeting a highly conserved domain. Since it is known that breadth is important for the effectiveness and durability of antibody prophylaxis, particularly when seeking to protect against new, emerging strains, this too is a significant discovery.

The article published in Scientific Reports is entitled, “Intranasal administration of a panreactive influenza antibody reveals Fc-independent mode of protection.”

About Leyden Laboratories B.V.

Leyden Labs is working to free people from the threat of respiratory viruses. Leyden Labs is leveraging its Mucosal Protection Platform to develop a portfolio of candidates aimed at providing protection against influenza, coronaviruses, and other respiratory viruses through a new class of broadly protective nasal sprays.

Systemically administered antibodies or vaccines primarily generate systemic protection. Airborne viruses, including influenza and coronaviruses, enter the body through the nose and mouth. Thus, what we need is protection right at the gate, at the respiratory mucosa, to more effectively prevent initial infection and subsequent illness.

Leyden Labs is pursuing this strategy by developing nasal sprays that administer broadly protective antibodies directly to the respiratory mucosa. These antibodies aim to protect against full viral families, so they keep working even when a virus mutates and evolves. In addition, this intranasal strategy is beneficial for people with weakened immune systems because it does not require a fully functional immune response.

To learn more, visit www.leydenlabs.com.

About PanFlu candidate CR9114

CR9114, Leyden Labs’ lead product candidate for the PanFlu program, is a human monoclonal antibody that protects against influenza in preclinical models. Leyden Labs holds an exclusive license from Janssen Pharmaceuticals, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, to develop and commercialize CR9114.

Contacts

Investor Contact
Elizabeth Goodwin

+1 781-460-1784

investors@leydenlabs.com

Media Contact
Megan Prock McGrath

+1 978-800-7468

media@leydenlabs.com

DATROWAY® Approved in the EU for Patients with Previously Treated Metastatic HR Positive, HER2 Negative Breast Cancer




DATROWAY® Approved in the EU for Patients with Previously Treated Metastatic HR Positive, HER2 Negative Breast Cancer

• First approval in the EU for Daiichi Sankyo and AstraZeneca’s DATROWAY based on TROPION-Breast01 trial showing 37% reduction in the risk of disease progression or death versus chemotherapy
• Second DXd antibody drug conjugate approved in EU based on Daiichi Sankyo’s DXd technology

TOKYO & MUNICH–(BUSINESS WIRE)–DATROWAY^® (datopotamab deruxtecan) has been approved in the European Union (EU) for the treatment of adult patients with unresectable or metastatic hormone receptor (HR) positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received endocrine therapy and at least one line of chemotherapy in the advanced setting.

DATROWAY is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/Nasdaq: AZN).

The approval by the European Commission follows the positive opinion of the Committee for Medical Products for Human Use of the European Medicines Agency and is based on results from the TROPION-Breast01 phase 3 trial.

In TROPION-Breast01, DATROWAY significantly reduced the risk of disease progression or death by 37% compared to investigator’s choice of chemotherapy (hazard ratio [HR]=0.63; 95% confidence interval [CI]: 0.52-0.76; p<0.0001) in patients with HR positive, HER2 negative metastatic breast cancer as assessed by blinded independent central review (BICR). Median progression free survival (PFS) was 6.9 months in patients treated with DATROWAY versus 4.9 months with chemotherapy. A confirmed objective response rate (ORR) of 36% was observed in the DATROWAY arm compared to an ORR of 23% observed in the chemotherapy arm. The median duration of response (DoR) was 6.7 months (95% CI: 5.6-9.8) in the DATROWAY arm compared to 5.7 months (95% CI: 4.9-6.8) in the chemotherapy arm. The final overall survival (OS) results of the trial did not achieve statistical significance (median OS of 18.6 months in the DATROWAY arm versus 18.3 months in the chemotherapy arm [HR 1.01; 95% CI: 0.83-1.22]) and may have been affected by subsequent ADC treatment.

“With the majority of breast cancer cases historically considered HR positive, HER2 negative, additional treatment options are needed to improve outcomes for patients with metastatic disease that continues to progress following endocrine-based therapy and initial chemotherapy,” said Barbara Pistilli, MD, Head of the Breast Cancer Unit in the Medical Oncology Department of Gustave Roussy Cancer Center, Villejuif, France. “The approval of DATROWAY in the EU will provide these patients with a new treatment option that can help slow the progression of this disease.”

“Treating metastatic HR positive, HER2 negative breast cancer presents challenges, particularly treatment resistance and disease progression that occur following endocrine-based therapy and initial chemotherapy,” said Ken Keller, Global Head of Oncology Business, and President and CEO, Daiichi Sankyo, Inc. “DATROWAY represents the second antibody drug conjugate approved for breast cancer based on Daiichi Sankyo’s DXd technology and the third medicine to be approved in the EU from our oncology pipeline, underscoring our commitment to creating new medicines for patients with cancer.”

“Though the HR positive breast cancer treatment landscape has evolved in the last several years, disease progression on front-line therapies remains a common and complex challenge for patients with metastatic disease,” said Dave Fredrickson, Executive Vice President, Oncology Hematology Business Unit, AstraZeneca. “With today’s approval of DATROWAY, patients in the EU with HR positive, HER2 negative breast cancer now have a new and needed alternative to conventional chemotherapy.”

Grade 3 or higher adverse events from a pooled safety analysis of two clinical studies, including 443 patients who received DATROWAY (6 mg/kg) for a median duration of 6.2 months (range: 0.7-28.5), were stomatitis (7.9%), fatigue (4.3%), anemia (3.2%), AST increased (2.7%), vomiting (1.6%), ALT increased (1.6%), nausea (1.4%), urinary tract infection (1.4%), COVID-19 (1.1%), decreased appetite (1.1%), neutropenia (1.1%) and pneumonia (1.1%). Grade 5 adverse events occurred in 0.7% of patients due to interstitial lung disease/pneumonitis, dyspnea and sepsis.

About TROPION-Breast01

TROPION-Breast01 is a global, randomized, multicenter, open-label phase 3 trial evaluating the efficacy and safety of intravenous DATROWAY (6 mg/kg) once per 21-day cycle versus investigator’s choice of single-agent chemotherapy (eribulin, capecitabine, vinorelbine or gemcitabine) in adult patients with unresectable or metastatic HR positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have progressed on and are not suitable for endocrine therapy per investigator assessment and have received at least one prior line of chemotherapy for unresectable or metastatic disease.

Following disease progression or discontinuation of DATROWAY or chemotherapy, patients had the option to receive a subsequent treatment at the discretion of their physician. Crossover between trial arms was not permitted.

The dual primary endpoints of TROPION-Breast01 are PFS as assessed by BICR and OS. Key secondary endpoints include ORR, DoR, investigator-assessed PFS, disease control rate, time to first subsequent therapy and safety. The PFS data and additional results for key secondary endpoints of TROPION-Breast01 were published in the Journal of Clinical Oncology and OS results were presented at a Virtual Plenary session hosted by the European Society for Medical Oncology in February 2025.

TROPION-Breast01 enrolled 732 patients in Africa, Asia, Europe, North America and South America. For more information visit ClinicalTrials.gov.

About Hormone Receptor Positive, HER2 Negative Breast Cancer

Breast cancer is the second most common cancer and one of the leading causes of cancer-related deaths worldwide.¹ More than two million breast cancer cases were diagnosed in 2022 with more than 665,000 deaths globally.¹ In Europe, approximately 557,000 cases of breast cancer are diagnosed annually.² While survival rates are high for those diagnosed with early breast cancer, only about 30% of patients diagnosed with or who progress to metastatic disease are expected to live five years following diagnosis.³

Approximately 70% of diagnosed cases are considered what has been historically called HR positive, HER2 negative breast cancer (measured as HER2 score of IHC 0, IHC 1+ or IHC 2+/ISH-).³ Endocrine therapy is widely given consecutively in the early lines of treatment for metastatic HR positive breast cancer.⁴ However, after initial treatment, further efficacy from endocrine therapy is often limited.⁴

About DATROWAY

DATROWAY (datopotamab deruxtecan) is a TROP2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, DATROWAY is one of six DXd ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. DATROWAY is comprised of a humanized anti-TROP2 IgG1 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

DATROWAY is approved in more than 30 countries for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease based on the results of the TROPION-Breast01 trial.

About the DATROWAY Clinical Development Program

A comprehensive global clinical development program is underway with more than 20 trials evaluating the efficacy and safety of DATROWAY across multiple cancers, including non-small cell lung cancer, triple negative breast cancer and HR positive, HER2 negative breast cancer. The program includes eight phase 3 trials in lung cancer and five phase 3 trials in breast cancer evaluating DATROWAY as a monotherapy and in combination with other anticancer treatments in various settings.

About the Daiichi Sankyo and AstraZeneca Collaboration

Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU^® in March 2019 and DATROWAY in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and DATROWAY.

About the ADC Portfolio of Daiichi Sankyo

The Daiichi Sankyo ADC portfolio consists of seven ADCs in clinical development crafted from two distinct ADC technology platforms discovered in-house by Daiichi Sankyo.

The ADC platform furthest in clinical development is Daiichi Sankyo’s DXd ADC Technology where each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADC portfolio currently consists of ENHERTU, a HER2 directed ADC, and DATROWAY, a TROP2 directed ADC, which are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck & Co., Inc, Rahway, NJ, USA. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo.

The second Daiichi Sankyo ADC platform consists of a monoclonal antibody attached to a modified pyrrolobenzodiazepine (PBD) payload. DS-9606, a CLDN6 directed PBD ADC, is the first of several planned ADCs in clinical development utilizing this platform.

Ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan, DS-3939 and DS-9606 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established.

About Daiichi Sankyo

Daiichi Sankyo is an innovative global healthcare company contributing to the sustainable development of society that discovers, develops and delivers new standards of care to enrich the quality of life around the world. With more than 120 years of experience, Daiichi Sankyo leverages its world-class science and technology to create new modalities and innovative medicines for people with cancer, cardiovascular and other diseases with high unmet medical need. For more information, please visit www.daiichisankyo.com.

Contacts

Media Contacts:

Global:
Jennifer Brennan

Daiichi Sankyo, Inc.

jennifer.brennan@daiichisankyo.com
+1 908 900 3183 (mobile)

EU:
Simone Jendsch-Dowé

Daiichi Sankyo Europe GmbH

simone.jendsch-dowe@daiichisankyo.com
+49 176 11780822

Japan:
Daiichi Sankyo Co., Ltd.

DS-PR_jp@daiichisankyo.com

Investor Relations Contact:
DaiichiSankyoIR_jp@daiichisankyo.com

MaaT Pharma Announces Positive Safety Interim Analysis from DSMB for Phase 2b Trial Evaluating MaaT033 for Patients Receiving Allo-HSCT




MaaT Pharma Announces Positive Safety Interim Analysis from DSMB for Phase 2b Trial Evaluating MaaT033 for Patients Receiving Allo-HSCT

• Following a milestone unblinded interim safety review, the independent Data Safety Monitoring Board (DSMB) has recommended that the study proceed without modification.
• MaaT033, a pooled donor-derived oral drug candidate developed for ambulatory use, continues to demonstrate a favorable safety profile and tolerability.

LYON, France–(BUSINESS WIRE)–$MAAT #Cancer–Regulatory News:

MaaT Pharma (EURONEXT: MAAT – the “Company”), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem Therapies^TM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, today announced the positive outcome of a key DSMB safety interim analysis for the Phase 2b trial PHOEBUS, the world’s largest randomized controlled trial evaluating microbiome therapy in oncology to date. The study compares the efficacy and safety of MaaT033 (experimental arm) to placebo in patients undergoing an Allogeneic Hematopoietic Stem Cell Transplantation (allo-HSCT).

Early after allo-HSCT, patients are highly vulnerable and face a significant risk of non-relapse mortality. Therefore, the study protocol includes a specific safety analysis that would trigger a stopping rule in case a pre-defined mortality excess in the experimental arm would be identified after 30 patients have been randomized to receive MaaT033 (approximately 60 enrolled in the study) and monitored for 90 days after allo-HSCT. This analysis is distinct from the ongoing safety assessments conducted every six months, whose positive outcomes have last been communicated on January 21, 2025. As a result of their unblinded analysis, the DSMB recommended the trial to proceed as planned, showing no excessive mortality related to MaaT033 as of today. This additional positive outcome further reinforces MaaT033’s safety profile and supports MaaT033’s integration in the allo-HSCT setting without significant risks of severe adverse events.

“We are pleased to report that MaaT033’s safety profile continues to be positive. The confirmed absence of a pre-specified mortality excess in patients receiving MaaT033 is of critical relevance”, said Gianfranco Pittari, MD, PhD, Chief Medical Officer of MaaT Pharma. “These patients would enormously benefit from innovative therapies enhancing hematopoietic stem cell transplantation outcomes while avoiding toxic effects.”

Patient enrollment is ongoing in France, Germany, Belgium, Spain, Netherlands and the United Kingdom. The Phoebus trial is an international, multi-center, randomized, double-blinded study comparing MaaT033 (a standardized, oral, freeze-dried, multi-donor microbiotherapy) to placebo in patients receiving an allo-HSCT. The trial is expected to enroll 387 patients and is set to be conducted in up to 60 clinical investigational sites (NCT05762211).

Building on the demonstrated safety and efficacy profile of MaaT013, the capsule formulation MaaT033, a high-value asset and the second candidate from MaaT Pharma’s native ecosystem platform, is designed to reach a larger patient population through its oral administration. By enabling outpatient use, MaaT033 also supports optimized patient care.

Next steps:

• The next DSMB unblinded interim analysis with mortality monitoring is scheduled for Q3 2025 at the 120-patient mark.
• The routine DSMB review for ongoing safety, conducted every six months, is also expected for Q3 2025.

About MaaT033

MaaT033, a donor-derived, high-richness, high-diversity oral Microbiome Ecosystem Therapy^TM containing anti-inflammatory Butycore^TM species, is currently being developed as an adjunctive therapy to improve overall survival in patients receiving HSCT and other cellular therapies. It aims to ensure optimal microbiota function and to address a larger patient population in a chronic setting. MaaT033 has been granted Orphan Drug Designation by the European Medicines Agency (EMA).

About MaaT Pharma

MaaT Pharma is a leading, late-stage clinical company focused on developing innovative gut microbiome-driven therapies to modulate the immune system and enhance cancer patient survival. Supported by a talented team committed to making a difference for patients worldwide, the Company was founded in 2014 and is based in Lyon, France. As a pioneer, MaaT Pharma is leading the way in bringing the first microbiome-driven immunomodulator in oncology. Using its proprietary pooling and co-cultivation technologies, MaaT Pharma develops high diversity, standardized drug candidates, aiming at extending life of cancer patients. MaaT Pharma has been listed on Euronext Paris (ticker: MAAT) since 2021.

Forward-looking Statements

All statements other than statements of historical fact included in this press release about future events are subject to (i) change without notice and (ii) factors beyond the Company’s control. These statements may include, without limitation, any statements preceded by, followed by, or including words such as “target,” “believe,” “expect,” “aim”, “intend,” “may,” “anticipate,” “estimate,” “plan,” “project,” “will,” “can have,” “likely,” “should,” “would,” “could” and other words and terms of similar meaning or the negative thereof. Forward-looking statements are subject to inherent risks and uncertainties beyond the Company’s control that could cause the Company’s actual results or performance to be materially different from the expected results or performance expressed or implied by such forward-looking statements.

Contacts

MaaT Pharma – Investor Relations
Guilhaume DEBROAS, Ph.D.

Head of Investor Relations

+33 6 16 48 92 50

invest@maat-pharma.com

Rx Communications Group – U.S. Investor Relations
Michael Miller

Managing Director

+1-917-633-6086

mmiller@rxir.com

MaaT Pharma – Media Relations
Pauline RICHAUD

Senior PR & Corporate Communications Manager

+33 6 14 06 45 92

media@maat-pharma.com

Catalytic Agency – U.S. Media Relations
Heather Shea

Media relations for MaaT Pharma
+1 617-286-2013
heather.shea@catalyticagency.com

GenSight Biologics Announces the Filing of its 2024 Universal Registration Document




GenSight Biologics Announces the Filing of its 2024 Universal Registration Document

PARIS–(BUSINESS WIRE)–Regulatory News:

GenSight Biologics (Euronext: SIGHT, ISIN: FR0013183985, PEA-PME eligible), a biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders, announced the filing on April 7, 2025 of its 2024 Universal Registration Document (URD) in English with the French market authority (Autorités des Marchés Financiers, or AMF).

The Universal Registration Document includes in particular:

• The 2024 annual financial report;
• The management report;
• The report on corporate governance; and
• The description of the share buyback program.

The Universal Registration Document may be consulted on the Company’s website (www.gensight-biologics.com), “Investors” section, and on the AMF’s website (www.amf-france.org).

About GenSight Biologics

GenSight Biologics S.A. is a clinical-stage biopharma company focused on developing and commercializing innovative gene therapies for retinal neurodegenerative diseases and central nervous system disorders. GenSight Biologics’ pipeline leverages two core technology platforms, the Mitochondrial Targeting Sequence (MTS) and optogenetics, to help preserve or restore vision in patients suffering from blinding retinal diseases. GenSight Biologics’ lead product candidate, GS010 (lenadogene nolparvovec) is in Phase III in Leber Hereditary Optic Neuropathy (LHON), a rare mitochondrial disease that leads to irreversible blindness in teens and young adults. Using its gene therapy-based approach, GenSight Biologics’ product candidates are designed to be administered in a single treatment to each eye by intravitreal injection to offer patients a sustainable functional visual recovery.

Contacts

GenSight Biologics
Chief Financial Officer

Jan Eryk Umiastowski

jeumiastowski@gensight-biologics.com

Niagen Bioscience Confirms Its Operations Remain Unaffected by New Tariffs




Niagen Bioscience Confirms Its Operations Remain Unaffected by New Tariffs

The Company’s flagship patented nicotinamide riboside (NR) ingredient, Niagen^®, is manufactured, encapsulated, and bottled in the U.S. and is currently classified as exempt from tariffs, minimizing exposure to tariff-related risks

LOS ANGELES–(BUSINESS WIRE)–$NAGE #Bioscience—Niagen Bioscience, Inc. (NASDAQ: NAGE) (formerly ChromaDex Corp.), the global authority on NAD+ (nicotinamide adenine dinucleotide) with a focus on the science of healthy aging, reaffirms that the manufacturing of its Niagen^® ingredient (patented nicotinamide riboside or NR) and Tru Niagen^® and pharmaceutical-grade Niagen^® products remain resilient amid the recently enacted global tariffs.

Manufactured in the U.S. by the Company’s partner, W.R. Grace, Niagen is rigorously tested, encapsulated, bottled, and packaged domestically as Tru Niagen using premium materials. With only a small percentage of materials sourced internationally, the cost to manufacture final Niagen products remains largely insulated from global price volatility.

“We made the decision to build a U.S.-based supply chain for Niagen to safeguard long-term resilience, trust, and quality,” said Rob Fried, CEO of Niagen Bioscience. “That foresight is proving valuable now. We remain confident in our ability to deliver Niagen products to customers and long-term stability to shareholders.”

Currently, most vitamins—including nicotinamide riboside (NR)— remain exempt from the recently enacted tariffs. This exemption enables Niagen Bioscience to continue international operations without disruption. In 2024, approximately 24% of the Company’s revenue came from international sales, with over 12% attributed to A.S. Watson in Hong Kong—a duty-free market not subject to tariffs. As a result, this key international revenue stream remains unaffected by recent trade policy changes.

At this time, the Company anticipates no interruption in its ability to deliver Niagen products or maintain costs and pricing.

For additional information on Niagen Bioscience, visit www.niagenbioscience.com.

About Niagen Bioscience:

Niagen Bioscience, Inc. (NASDAQ: NAGE), formerly ChromaDex Corp., is the global leader in NAD+ (nicotinamide adenine dinucleotide) science and healthy-aging research. As a trusted pioneer of NAD+ discoveries, Niagen Bioscience™ is dedicated to advancing healthspan through precision science and innovative NAD+-boosting solutions.

The Niagen Bioscience team, composed of world-renowned scientists, works with independent investigators from esteemed universities and research institutions around the globe to uncover the full potential of NAD+. A vital coenzyme found in every cell of the human body, NAD+ declines with age and exposure to everyday lifestyle stressors. NAD+ depletion is a key contributor to age-related changes in health and vitality.

Distinguished by state-of-the-art laboratories, rigorous scientific and quality protocols, and collaborations with leading research institutions worldwide, Niagen Bioscience sets the gold standard for research, quality, and innovation. There’s a better way to age.

At the heart of its clinically proven product portfolio is Niagen^® (patented nicotinamide riboside, or NR), the most efficient, well-researched, high-quality, and legal NAD+ booster available. Niagen powers the Company’s consumer supplement, Tru Niagen^®, the number one NAD+ boosting oral supplement in the United States^† (available at www.truniagen.com), and Niagen Plus™, featuring pharmaceutical-grade intravenous (IV) and injectable Niagen products (www.niagenplus.com). Pharmaceutical-grade Niagen IV and injections are compounded and distributed by U.S. FDA-registered 503B outsourcing facilities and are available exclusively at clinics with a prescription.

Niagen Bioscience’s robust patent portfolio protects NR and other NAD+ precursors. Niagen Bioscience maintains a website at www.niagenbioscience.com, where copies of press releases, news, and financial information are regularly published.

^†Based on the top-selling dietary supplement brands by revenue per the largest U.S. e-commerce marketplace (as of 1/1/2024 – 12/31/2024).

Forward Looking Statements:

This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities and Exchange Act of 1934, as amended. Statements that are not a description of historical facts constitute forward-looking statements and may often, but not always, be identified by the use of such words as “expects,” “anticipates,” “intends,” “estimates,” “plans,” “potential,” “possible,” “probable,” “believes,” “seeks,” “may,” “will,” “should,” “could” or the negative of such terms or other similar expressions. Risks that contribute to the uncertain nature of these forward-looking statements include: the potential impact of newly imposed tariffs on the Company’s business operations, supply chain, financial performance, and strategic initiatives, our history of operating losses and need to obtain additional financing; the growth and profitability of our product sales; our ability to maintain sales, marketing and distribution capabilities; changing consumer perceptions of our products; our reliance on a single or limited number of third-party suppliers; and the risks and uncertainties associated with our business and financial condition. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and actual results may differ materially from those suggested by these forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement and Niagen Bioscience undertakes no obligation to revise or update this release to reflect events or circumstances after the date hereof.

Contacts

Niagen Bioscience Media Contact:
Kendall Knysch, Senior Director of Media Relations & Partnerships

310.405.5227

kendall.knysch@niagenbio.com

Niagen Bioscience Investor Relations Contact:
Ben Shamsian

Lytham Partners

646-829-9701

shamsian@lythampartners.com

AGC Biologics Strengthens Single-Use Technology Network and Adds 5,000 L Vessels from Thermo Scientific to New Yokohama, Japan Facility




AGC Biologics Strengthens Single-Use Technology Network and Adds 5,000 L Vessels from Thermo Scientific to New Yokohama, Japan Facility

CDMO’s new site introducing the most advanced and large-scale single-use bioreactors in the region

YOKOHAMA, Japan & SEATTLE–(BUSINESS WIRE)–AGC Biologics, your friendly CDMO expert, today announced a strategic decision about the large-scale single-use technology it will use at its new Yokohama, Japan facility. The CDMO is introducing two 5,000 L Thermo Scientific DynaDrive Single-Use Bioreactors (S.U.B.), which makes the site one of the most advanced in Japan for large-scale mammalian-based biologics production, with GMP operations beginning in 2027.

According to 2024 data from BDO Group’s bioTRAK Database, single-use technology represents 51 percent of all mammalian bioreactor technology globally, outpacing the traditional stainless-steel alternative. Attributes driving this trend include fewer cleaning requirements, lower cost barrier to entry and timeline for setup, flexible configurations, scalability to meet changing product demands, and shorter clinical production timelines. AGC Biologics has the second largest global network of single-use manufacturing capacity by volume in the industry, according to bioTRAK. The new bioreactor technology ensures the company stays ahead of industry-wide demand for single-use systems.

“The new Yokohama site is designed to utilize single-use bioreactors to offer large-scale GMP manufacturing. The cutting-edge technology from Thermo Scientific makes this one of the most advanced sites in Japan for large-scale manufacturing and further expands our robust global network of single-use manufacturing sites on three continents,” said Christoph Winterhalter, Chief Business Officer, AGC Biologics. “This location offers the capabilities to meet the commercial needs of customers in Japan and Asia, as well as global customers seeking capacity using the latest technology.”

“Flexibility and the means to scale production cost-effectively is key for any developer of therapies today, and CDMOs need the best technology possible to help them accomplish that. The DynaDrive 5,000 L Single-Use Bioreactor delivers seamless scalability, exceptional flexibility, and superior process intensification capabilities – embodying best-in-class performance. AGC Biologics’ new site in Yokohama will be a premier site in Japan for large-scale single-use technology thanks to our collaboration,” said Daniella Cramp, Senior Vice President and President of Thermo Fisher Scientific’s Bioproduction business.

The new AGC Biologics Yokohama facility will offer support from initial process development work for early-phase projects to late-phase and commercial production. It will support mammalian expression, cell therapy, and messenger RNA (mRNA) drug products.

The Yokohama facility is expected to help maximize operational and supply chain efficiencies across AGC Biologics’ global network by adding another site in the region. The CDMO currently operates one site in Japan, its Chiba facility, offering mammalian expression and microbial fermentation services.

AGC Biologics is a part of AGC Inc.’s Life Science Company. The Life Science company operates over 10 facilities worldwide focused on biopharmaceuticals, advanced therapies, small molecule active pharmaceutical ingredients, and agrochemicals.

To learn more about AGC Biologics’ global network of services for protein biologics and cell and gene therapies, please visit www.agcbio.com.

About AGC Biologics

AGC Biologics is a leading global biopharmaceutical Contract Development and Manufacturing Organization (CDMO) with a strong commitment to delivering the highest standard of service as we work side-by-side with our clients and partners, to provide friendly and expert services. We provide world-class development and manufacturing of mammalian and microbial-based therapeutic proteins, plasmid DNA (pDNA), messenger RNA (mRNA), viral vectors, and genetically engineered cells. Our global network spans the U.S., Europe, and Asia, with locations in Seattle, Washington; Boulder and Longmont, Colorado; Copenhagen, Denmark; Heidelberg, Germany; Milan, Italy; and Chiba and Yokohama, Japan. We currently employ more than 2,800 Team Members worldwide. AGC Biologics is a part of AGC Inc.’s Life Science Business. The Life Science Business runs 10+ facilities focused on biopharmaceuticals, advanced therapies, small molecule active pharmaceutical ingredients, and agrochemicals. To learn more, visit www.agcbio.com.

Contacts

Media Contact
Nick McDonald

AGC Biologics

nmcdonald@agcbio.com

AGC Inc. Media Contact
info-pr@agc.com

Quibim Announces New Board of Directors To Promote Growth And Strategic Expansion




Quibim Announces New Board of Directors To Promote Growth And Strategic Expansion

NEW YORK & VALENCIA, Spain & CAMBRIDGE, England–(BUSINESS WIRE)–#AIinHealthcare—Quibim, the global pioneer in imaging biomarkers for precision medicine, is pleased to announce the formation of its new Board of Directors. This strategic move aligns with Quibim’s commitment to innovation and growth in the healthcare sector. It follows the recent FDA 510(k) clearance of its flagship product, QP-Prostate CAD, and the close of a $50M Series A funding round in January. Quibim’s advanced imaging solutions are currently utilized across more than 180 sites worldwide.

The new Board of Directors is composed of six members:

• Dr. Ángel Alberich-Bayarri, CEO & Co-Founder of Quibim
• Tom Tynes, Founder of T2 MedTech (Independent Director)
• Pierre Socha, Partner at Amadeus Capital
• Romain Lavault, General Partner at Partech
• Guillem Masferrer, Partner at Asabys Partners
• José Mesa, Partner at Columbus Venture Partners and Buenavista’s representative on the Board

One of the new appointments to the Board of Directors is Tom Tynes, who joins as an independent director. Tom brings a wealth of experience, having led the formation, profitable growth, and success of several medical device companies over the past 30 years. During his career, Tom has played a pivotal role in developing numerous transformative innovations and technologies in diagnostic imaging and minimally invasive image-guided intervention, resulting in several patented, market-leading solutions that have driven major advances in clinical practice guidelines. While Tom was a pioneer in breast imaging and intervention – most notably through the development of DynaCAD, the industry-leading computer-aided detection (CAD) system for breast MRI – his expertise later expanded to other high-impact clinical areas, including lung and prostate cancer. Tom will play a significant role in advising and guiding Quibim’s expansion into the US market and supporting Quibim’s long-term vision and growth plans.

Tom Tynes commented on his new role: “I am thrilled to join Quibim’s Board of Directors at such a pivotal time. I look forward to leveraging my experience to help drive Quibim’s expansion into the US market and support its innovative approach to transforming patient care.”

The additions of Guillem Masferrer and José Mesa to the Board represent the new investors from Quibim’s recent Series A funding round of $50M which was announced in January this year. Their extensive experience and contacts in life sciences and biotech will be instrumental in driving Quibim’s strategic initiatives. Pierre Socha and Romain Lavault, represent the long-standing investors and bring a wealth of expertise that will continue to guide Quibim’s advancements in the healthcare space.

Dr. Ángel Alberich-Bayarri, CEO of Quibim, stated: “The formation of our new Board of Directors marks a significant milestone for Quibim. With the combined expertise of our new and existing members, we are well-positioned to accelerate our growth and continue our mission of transforming patient data into patient care. These changes are a natural progression following our Series A funding and are aligned with our strategic priorities to enhance our market presence and technological capabilities. We are confident that the new Board will drive Quibim towards achieving its mission to turn imaging into a catalyst for precision health and revolutionize diagnostics and improve patient outcomes.”

About Quibim

Quibim is a company designing pioneering tools that unlock imaging data to improve patient outcomes. With offices in New York (United States), Cambridge (UK), Valencia, Madrid, and Barcelona (Spain), the company was born from the ambition of turning imaging into a catalyst for precision health. Quibim leads the forefront of imaging biomarkers research in life sciences, pioneering the development of advanced algorithms that transform imaging data into actionable predictions in oncology, immunology, and neurology. The company leverages the capabilities of MRI, CT, and PET imaging to create regulatory-cleared Medical Devices that are seamlessly integrated into the workflows of healthcare providers worldwide. More than 180 sites are using Quibim products globally.

Quibim has developed several products, including:

• QP-Prostate® CAD is the company’s flagship product for identifying prostate cancer. Now includes an industry-leading lesion detection capability.
• QP-Brain®, which provides quantification and visualization of imaging findings. This is useful for identifying patterns of brain atrophy and neurodegenerative diseases like Alzheimer’s, multiple sclerosis, and vascular and frontotemporal dementia at earlier stages and makes reporting more effective. US, UK, and EU regulators recently launched and cleared it.
• QP-Insights®, an all-in-one web platform that manages, stores, and quantitatively analyzes medical images and other clinical data, recently launched and already in use by leading bio-pharmaceutical companies and European research consortiums.
• QP-Liver® improves the diagnosis of diffuse liver diseases by accurately quantifying tissue fat and iron from MRI scans.

Quibim has also announced several high-profile partnerships recently, including Merck KGaA, to develop imaging biomarker models to predict responses of cancer patients to immunotherapy and inform decisions in its global drug development efforts. Additionally, Quibim partnered with Philips, a leader in the health technology industry, to launch AI-based imaging and reporting solutions for MR prostate exams.

Contacts

quibim@camarco.com

ZCG-Backed Unimed Acquires Lagaay Medical Group




ZCG-Backed Unimed Acquires Lagaay Medical Group

Strengthens Global Leadership as the Leading Maritime Health and Compliance Solutions Provider

NEW YORK–(BUSINESS WIRE)–Universal Marine Medical Supply International (“Unimed” or the “Company”), the leading global provider of health and compliance solutions to the maritime industry, today announced its acquisition of Lagaay Medical Group (“Lagaay”), a global distributor of pharmaceutical products and medical devices, from B&S Group S.A. (“B&S”). Unimed is backed by affiliates of ZCG Private Equity, the private equity arm of ZCG, a privately held global firm.

Headquartered in the Netherlands, Lagaay has more than 140 years of experience providing high-quality medical supplies and pharmaceuticals for the marine industry. The company currently offers 10,000 medical SKUs across 100 active countries. This strategic acquisition expands Unimed’s presence in Rotterdam – the largest port in Europe – further enhancing Unimed’s ability to serve global clients with comprehensive healthcare and compliance solutions as part of its long-term growth strategy.

“This transaction marks a significant step in strengthening Unimed’s leadership in the maritime medical industry while deepening our partnerships with clients worldwide,” said Søren Andersen, Chief Executive Officer of Unimed. “We have long respected Lagaay’s business and global reach, particularly its strong presence in Rotterdam. We look forward to welcoming their team to Unimed, expanding our technology-driven subscription model to more clients, and continuing to execute on our growth strategy.”

“Joining forces with Unimed is the right next step for Lagaay, we have always perceived Unimed as a logical partner to capture synergies and strengthen the market position of Lagaay in the maritime medical industry,” said Peter van Mierlo, Chief Executive Officer of B&S. “We are proud of all that we have achieved with Lagaay’s management over the past 4,5 years, growing its client base and solutions, and focusing on operational excellence. We wish the team continued success in this next chapter.”

“We are pleased to announce this strategic transaction as Unimed continues to capitalize on both organic and inorganic growth opportunities,” said James Zenni, Founder, President and Chief Executive Officer of ZCG. “With this expansion, Unimed is even more well positioned to lead the maritime industry by delivering the platform of choice to clients globally. We look forward to supporting the combined Unimed and Lagaay leadership and the experienced teams as they integrate two companies.”

The transaction is expected to close in the coming days, subject to the satisfaction of customary closing conditions.

In connection with the acquisition, Unimed will launch a new branding and product suite at the Seatrade Cruise Global conference later this month, marking a new chapter in its evolution as a premier healthcare provider for the maritime sector. Attendees will get a first look at the refreshed identity, reflecting Unimed’s commitment to innovation and excellence.

About Unimed

Unimed is the industry leader in medical supply, equipment and oxygen distribution to global maritime industries, specializing in the delivery of integrated healthcare and compliance solutions to clients worldwide.

Unimed is a portfolio company of ZCG Private Equity, the private equity arm of ZCG (www.zcg.com), a leading privately held global firm comprised of private markets asset management, business consulting services, and technology development and solutions.

For more information, please visit https://universalmarinemedical.com/.

About ZCG

ZCG is a leading, privately held global firm comprised of private markets asset management, business consulting services, and technology development and solutions.

For nearly three decades, ZCG has invested tens of billions of dollars in private equity and credit-related strategies, through multiple economic cycles and across many industries, including consumer products, steel, steel processors, agriculture, consumer food, gaming, hospitality, manufacturing, specialty services, and automotive.

ZCG’s investors include prominent global sovereign wealth funds, endowments, pension funds, insurance companies, foundations, family offices, wealth management firms, and other financial institutions in North America, Europe, Asia, Africa, and the Middle East.

ZCG has a global team comprised of approximately 400 professionals. The company is headquartered in New York, with affiliate offices located in Pune and Mumbai, India, and Riyadh, KSA.

For more information, please visit www.zcg.com.

Contacts

Tim Ragones / Kate Thompson / Madeline Jones

Joele Frank, Wilkinson Brimmer Katcher

212-355-4449