PureTech Appoints Peel Hunt as Joint UK Corporate Broker

PureTech Appoints Peel Hunt as Joint UK Corporate Broker




PureTech Appoints Peel Hunt as Joint UK Corporate Broker

BOSTON–(BUSINESS WIRE)–PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) (“PureTech” or the “Company”), is pleased to announce that it has appointed Peel Hunt as Joint UK Corporate Broker alongside UBS.


About PureTech Health

PureTech is a clinical-stage biotherapeutics company dedicated to giving life to new classes of medicine to change the lives of patients with devastating diseases. The Company has created a broad and deep pipeline through its experienced research and development team and its extensive network of scientists, clinicians and industry leaders that is being advanced both internally and through its Founded Entities. PureTech’s R&D engine has resulted in the development of 29 therapeutics and therapeutic candidates, including three that have been approved by the U.S. Food and Drug Administration. A number of these programs are being advanced by PureTech or its Founded Entities in various indications and stages of clinical development, including registration-enabling studies. All of the underlying programs and platforms that resulted in this pipeline of therapeutic candidates were initially identified or discovered and then advanced by the PureTech team through key validation points.

For more information, visit www.puretechhealth.com or connect with us on X (formerly Twitter) @puretechh.

Contacts

PureTech
Public Relations

publicrelations@puretechhealth.com
Investor Relations

IR@puretechhealth.com

Fasikl Announces Breakthrough Pivotal Data Demonstrating Superiority of Felix NeuroAI Wristband over Sham Device for Essential Tremor

Fasikl Announces Breakthrough Pivotal Data Demonstrating Superiority of Felix NeuroAI Wristband over Sham Device for Essential Tremor




Fasikl Announces Breakthrough Pivotal Data Demonstrating Superiority of Felix NeuroAI Wristband over Sham Device for Essential Tremor

—Wristband is a first-of-its-kind investigational wearable designed to provide dynamic, all-day, personalized relief for people with essential tremor (ET); powered by advanced AI to continuously adapt stimulation based on real-time feedback—

—TRANQUIL study results presented in late-breaker at the 2025 American Academy of Neurology (AAN) annual meeting demonstrate clinically meaningful improvements in ET symptoms and ability to perform daily activities—

SAN DIEGO–(BUSINESS WIRE)–Fasikl, a trailblazing neuro-AI company redefining the intersection of advanced bioelectronic medicine and artificial intelligence (AI), today announced that its pivotal TRANQUIL study met its primary endpoint of superiority for the company’s Felix™ NeuroAI™ wristband over a sham device. The Felix wristband significantly reduced tremors and showed statistically and clinically significant improvement in the users’ ability to perform daily activities compared to those using the sham device.


“This wearable technology delivered significant improvement in tremor, with both clinicians and patients more likely to report benefit by the end of the study,” said Rajesh Pahwa, M.D., Laverne and Joyce Rider Professor of Neurology at the University of Kansas School of Medicine, Director of the Movement Disorder Program at The University of Kansas Health System, and the principal investigator for TRANQUIL. “The TRANQUIL study represents what could be a shift in how we approach essential tremor treatment, a condition for which new options are sorely needed. Although oral medications are often used to treat essential tremor, they can be frequently unsatisfactory, and while deep brain stimulation and focused ultrasound are effective, they require surgical intervention.”

At the end of the 90-day period the Felix group showed clear superiority over the sham group.

  • The Felix group experienced a statistically and clinically significant improvement in ET treatment, with a mean reduction of 6.9 points in modified Activities of Daily Living (mADL) scores compared to 2.7 points in the sham group (P < 0.0001).
  • Nearly two-thirds of participants using Felix achieved at least a 20% reduction in mADL scores versus just one-quarter in the sham group.
  • Efficacy benefits were consistent across age, gender, tremor severity, and ET medication use.

Secondary outcomes further highlighted the impact of Felix. Both clinicians and patients reported greater symptom improvement with Felix, and quality of life scores improved more substantially in the treatment group. Importantly, no serious device-related adverse events were reported, confirming the device’s strong safety profile.

The Felix NeuroAI Wristband is a first-of-its-kind investigational wearable designed to deliver all-day, personalized relief for people with ET. Powered by advanced AI, Felix continuously adapts stimulation based on real-time feedback, providing dynamic, individualized therapy. Unlike surgical or pharmacologic interventions, Felix is non-invasive and wearable, connecting seamlessly to Fasikl’s cloud-based AI platform for ongoing optimization and learning.

“The TRANQUIL study marks a breakthrough as the first multicenter, double-blind, sham-controlled, non-surgical randomized controlled trial in essential tremor to meet its primary endpoint in over two decades,” said Zhi Yang, Ph.D., CEO of Fasikl. “Felix is a transformative advancement in AI-powered therapeutics, offering a safe, non-invasive, and personalized treatment option for ET patients and their physicians. With the efficacy and safety of Felix now validated and presented, we believe this represents a pivotal moment in the treatment of essential tremor and the broader landscape of movement disorders.”

About Essential Tremor (ET)

Essential tremor (ET) is the most common type of tremor disorder, affecting approximately 7 million people in the United States.¹ It causes involuntary, rhythmic shaking, most often in the hands, which can make everyday activities like drinking from a glass or tying shoelaces difficult.² Over time, ET can worsen and significantly impact a person’s quality of life.²

The TRANQUIL Study

The TRANQUIL study, a randomized, double-blind, sham-controlled clinical trial, enrolled 125 adults across 12 clinical sites in the U.S. and China. Participants were randomized 2:1 to receive either the Felix™ NeuroAI™ Wristband or a sham device and wore it during waking hours for 90 days. The study’s primary endpoint was change in the modified Activities of Daily Living (mADL) score from the Tremor Research Group Essential Tremor Rating Assessment Scale (TETRAS), a commonly used tremor assessment that combines 10 activities of daily living (ADLs) and 2 clinical assessments of tremor.3 The mADL has a maximum score of 52.

About Fasikl

Based in Minneapolis, Fasikl, Inc. is a pioneering neuro-AI company dedicated to developing advanced, AI-powered neural technologies. A 2019 spin-off from the University of Minnesota, Fasikl leverages its proprietary platform to capture neural signals directly from peripheral nerves, enabling intelligent, adaptive therapy. The company is on a mission to transform the treatment landscape for movement disorders, paralysis, and other neurological impairments through cutting-edge neuromodulation innovations. Fasikl’s portfolio, currently under FDA review, includes the Felix™ NeuroAI™ Wristband and the MindForce™ Nerve-Computer Interface. To learn more, visit fasikl.com.

References:

  1. Louis ED, Ottman R. How many people in the USA have essential tremor? Deriving a population estimate based on epidemiological data. Tremor Other Hyperkinet Mov (N Y). 2014;4:259. https://doi.org/10.7916/D8TT4P4B
  2. Mayo Foundation for Medical Education and Research. (2025, March 15). Essential tremor. Mayo Clinic. https://www.mayoclinic.org/diseases-conditions/essential-tremor/symptoms-causes/syc-20350534
  3. Elble R, Comella C, Fahn S, Hallett M, Jankovic J, Juncos JL, Lewitt P, Lyons K, Ondo W, Pahwa R, Sethi K, Stover N, Tarsy D, Testa C, Tintner R, Watts R, Zesiewicz T. Reliability of a new scale for essential tremor. Mov Disord. 2012 Oct;27(12):1567-9.

 

Contacts

Media Contact:
Jenna Kane

Health+Commerce

480-388-9587

jennakane@healthandcommerce.com

New Coalition Calls for Urgent Action to Address Rising Stroke Crisis

New Coalition Calls for Urgent Action to Address Rising Stroke Crisis




New Coalition Calls for Urgent Action to Address Rising Stroke Crisis

GENEVA–(BUSINESS WIRE)–#StrokeActionNow–Launching today, the world’s first multisector advocacy movement dedicated to stroke – the Global Stroke Action Coalition – has issued an urgent call to action to address growing inequities in stroke. Already a leading cause of death and disability, without intervention, the global burden of stroke is projected to rise by a further 50% over the next 25 years, claiming 100 million lives and costing US$1.6 trillion each year.


At this afternoon’s launch, health and economic policy specialists, clinical experts and people with lived experience of stroke, will highlight key data and recommendations for action to address human and financial impact of stroke. Drawing on both data and the lived experiences of people affected by stroke, the Coalition will share their inaugural policy document Stroke Action Now which sets out evidence-based examples of interventions that can significantly advance progress on a disease that is largely:

  • Preventable: 80% of the current stroke burden is linked to 10 modifiable risk factors. Identifying and managing hypertension alone could cut the rate of stroke in half.
  • Treatable: Advances in clot removal and clot-busting technologies (thrombectomy and thrombolysis) can dramatically improve patient health outcomes, minimizing disability and reducing the economic impact of stroke.
  • Recoverable: Access to rehabilitation helps people regain independence and reduces the risk —and cost— of long-term disability.

Despite the clear opportunities, prevention, treatment and rehabilitation services are only available to a fraction of stroke patients. For example, only 3% of medically eligible patients currently receive thrombectomy, with 20-40% of healthcare settings worldwide yet to implement basic stroke rehabilitation services.

“The global burden of stroke has doubled in the past 30 years,” explains Coalition co- Chair, Professor Bo Norrving. “During that same period huge advances have been made across the care pathway that offer us an incredible opportunity to reduce inequitable health outcomes and make significant progress towards global health and development targets. Committing to the development of National Stroke Plans should be a key priority for governments as part of their forward strategy for prevention and control of NCDs. We can’t afford to wait another 30 years to turn this around. Millions of lives depend on governments taking action now.”

Coalition leaders are calling on governments attending the 4th High-Level UN Meeting on NCDs in September this year, to commit to five actions:

1. Making stroke a priority in NCD prevention and control strategies so that it becomes an explicit and integral part of national and internal health agendas.

2. Developing National Stroke Action Plans that address the entire care pathway and include measurable targets that respond to in-country needs.

3. Committing to funding evidence-based interventions and exploring innovative financing models, such as taxing harmful substances, to build domestic resources.

4. Implementing robust stroke monitoring systems that measure changes in the stroke burden and the provision and outcomes of stroke care.

5. Including stroke survivors and caregivers in policy development and prioritize meaningful representation at all levels of decision-making.

The Coalition’s call comes at a defining moment. The upcoming UN High-Level Meeting is a rare opportunity for world leaders to shape the next 25 years of action on NCDs. Stroke must not be overlooked. The Coalition urges governments to seize this moment and ensure that stroke is recognized, prioritized, and acted upon.

Notes

Members of the Coalition include the World Stroke Organization, American Stroke Association, Asia Pacific Stroke Organization, Bayer, Boehringer Ingelheim, European Stroke Organisation, Heart & Stroke Foundation South Africa, Ipsen, March of Dimes Canada, Medtronic, Philips, Stroke Association UK and Society of Vascular and Interventional Neurology.

Contacts

strokecoalition@infinitycommunications.ch

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




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




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




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




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.

 ____________________

References:

1 Bray F, et al. CA Cancer J Clin. 2024; 10.3322/caac.21834.

2 Globocan 2022. Europe. Accessed April 2025.

3 National Cancer Institute. SEER Cancer Stat Facts: Female Breast Cancer Subtypes. Accessed April 2025.

4 Manohar P, et al. Cancer Biol Med. 2022 Feb 15; 19(2):202–212.

 

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




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 TherapiesTM (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 TherapyTM containing anti-inflammatory ButycoreTM 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




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




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 #BioscienceNiagen 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