Enigma Biomedical USA, Inc. Announces Candidate Selection of Novel 4R Tau PET Imaging Biomarkers




Enigma Biomedical USA, Inc. Announces Candidate Selection of Novel 4R Tau PET Imaging Biomarkers

KNOXVILLE, Tenn.–(BUSINESS WIRE)–Enigma Biomedical USA, Inc. (EB USA) today announced that it has selected two (2) four-repeat tau (4R Tau) protein PET imaging biomarkers to advance into Phase 1 (Ph1) studies. Previously, EB USA executed an Exclusive License and Option Agreement (License) with AbbVie for the development and potential commercialization of AbbVie’s next-generation F18 PET imaging biomarkers to assess the presence of 4R Tau in subjects with suspected neurodegenerative disease. These imaging biomarkers hold great promise as important new tools in advancing understanding of a range of neurodegenerative diseases in which the misfolded 4R Tau protein is implicated, including the tauopathies Progressive Supranuclear Palsy (PSP) and Corticobasal Degeneration.

Preclinical evaluations successfully identified two candidates to advance to Ph1 studies. These studies will examine several parameters, including safety, dosimetry, biomarker dynamic uptake and washout as well as determination of the optimal time for imaging. The studies are projected to commence in 3Q25.

“We are delighted that we have progressed this program to the clinical stage,” said Rick Hiatt, President and CEO of EB USA. “EB USA is committed to enabling the acceleration of promising technologies to advance the fight against debilitating neurodegenerative diseases. In this, we will build on demonstrated successes with the best-in-class neuroimaging biomarkers MK-6240 (Cerveau Technologies, sold to Lantheus Medical Imaging in 2023) and NAV-4694 (in development by Meilleur Technologies Inc., sold to Lantheus in 2024.) We believe our 4R Tau PET imaging biomarkers from AbbVie have unique properties and will prove useful in developing therapeutic agents in neurodegenerative disease. Our goal is to expand the availability of this novel investigational imaging technology to the broader scientific community.”

Hartmuth Kolb, Ph.D., Chief Science Officer of EB USA, states: “EB USA is excited to take the next steps in this important development process. The Ph1 studies are designed to measure an array of candidate characteristics in addition to safety and dosimetry, including metabolite formation and the dynamics of tracer uptake, retention and clearance. A final candidate for future development will be chosen based on increased uptake in PSP patients relative to that of healthy controls, the extent of off-target and non-specific binding as well as dynamic range, sensitivity and kinetics. We look forward to the first administration of these candidate biomarkers later this year.”

About Enigma Biomedical USA, Inc.

Enigma Biomedical USA, Inc.’s vision is to be the premier provider of imaging biomarkers for neurological pathologies, associated information technology, and related tools to accelerate the development, approval, and adoption of effective therapies to treat neurodegenerative diseases. EB USA’s neuroimaging biomarkers provide Pharma and Academic researchers with best-in-class tools for enabling Disease-Modifying Therapy development with the highest possible precision and accuracy.

Contacts

Enigma Biomedical USA, Inc.  

Rick Hiatt, 617-906-2715

Almirall’s 16th Skin Academy Brings Together Leading Experts to Advance Skin Science and Treatment Options in Medical Dermatology




Almirall’s 16th Skin Academy Brings Together Leading Experts to Advance Skin Science and Treatment Options in Medical Dermatology

• Almirall’s 16^th Skin Academy is a unique opportunity for collaboration in medical dermatology as it brings together leading experts from across the globe to advance science and innovative approaches for the treatment of skin diseases.
• The conference focuses on the latest advancements in medical dermatology, including the holistic and personalized treatment of atopic dermatitis and psoriasis.
• The comprehensive program covers a broad range of skin diseases such as actinic keratosis, androgenetic alopecia, onychomycosis and chronic spontaneous urticaria – advancing the understanding of these diseases and treatment options to support better patient outcomes.

BARCELONA, Spain–(BUSINESS WIRE)–Almirall, a global pharmaceutical company dedicated to medical dermatology hosts the 16^th edition of Skin Academy, a premier conference for healthcare professionals in the field of dermatology. This year’s event features a comprehensive program aimed at sharing state-of-the-art scientific knowledge on skin diseases, their treatment, and clinical best practice.

The Skin Academy is a unique opportunity for collaboration, bringing together a broad range of leading experts and clinicians from across the globe to create a network of peers that discuss and learn together to advance the treatment of skin diseases. The event will foster collaboration and co-creation of innovative approaches and solutions to better respond to the unmet needs of the millions of people living with dermatological conditions.

Prof. Dr. Ulrich Mrowietz, from the Psoriasis Center at the University Medical Center Schleswig-Holstein, Campus Kiel in Germany said: “deep collaboration across experts is essential for advancing medical dermatology and providing better treatment outcomes for patients. Almirall’s Skin Academy provides a unique platform for practitioners to explore latest scientific advances, clinical practice and discuss a broad range of skin diseases, with an emphasis on holistic, personalized patient care and wellbeing.”

The Chief Medical Officer of Almirall, Dr. Volker Koscielny, said: “this is the 16^th edition of the Skin Academy, and we are proud to continue to provide a leading platform for exchange and advancement in medical dermatology. Partnering with the dermatology community across Europe is based on our unique focus on medical dermatology and supported by our R&D capabilities as well as our strong collaborations. Skin Academy fosters collaboration and co-creation of innovative approaches and solutions that help the medical dermatology community to better respond to the unmet needs of millions of people living with these conditions.”

About Almirall’s Skin Academy

The Skin Academy, initiated and sponsored by Almirall, has become a cornerstone event for healthcare professionals in medical dermatology. Over the years, it has evolved into a key platform for sharing state-of-the-art scientific knowledge and clinical best practices in a wide range of skin diseases that affect millions of people. The event encourages collaboration and the development of new methods and solutions, aiming at addressing the unmet needs of patients and the medical dermatology community. The 16th edition continues this focus and Almirall’s commitment to educational advancement, bringing together over 800 participants, led by international experts, with common interests to create a network to discuss, educate, and learn to advance knowledge in medical dermatology.

About atopic dermatitis

As a highly prevalent and debilitating condition, atopic dermatitis will be a central focus at the conference, with experts offering new insights into its pathophysiology, the role of IL-13, and the importance of advanced treatment protocols. In addition, the discussion will include a comprehensive analysis of efficacy and safety of leading treatments over time, as well as real-world evidence from clinical practice. Another key topic is the dermatologist-patient relationship, which is crucial for treatment success, especially based on the role of shared decision-making in treatment planning.

About psoriasis

Psoriasis will be another focus area for the 16th Skin Academy, and will be addressed in a range of sessions specifically dedicated to biologic treatments. The speakers will address unmet medical needs, the importance of individualized treatment for long-term disease control and share real world evidence showing the effectiveness and safety of targeted IL-23 inhibition, as well as highlighting the importance of striving for patient wellbeing as treatment outcome. Moreover, as difficult-to-treat areas of the skin remain of special importance for patients suffering from psoriasis, current treatment challenges and approaches to address these will be discussed, including advancements in topical treatments and new efficacy data with IL-23 inhibition in psoriasis of the scalp, which affects 40-90% of patients with psoriasis¹²³⁴.

About Actinic Keratosis

The conference will address the crucial relationship between patients and their physicians, which is essential for treatment success and patient satisfaction, particularly in managing chronic skin diseases. It will highlight the importance of early treatment in actinic keratosis (AK) to prevent its progression to squamous cell carcinoma. Experts will cover advancements in AK treatment, real-world evidence, and the role of patient satisfaction in treatment outcomes.

The conference program further includes topics on other important dermatological conditions, including androgenetic alopecia, onychomycosis, and chronic spontaneous urticaria. The event will also feature poster sessions, clinical case discussions, and Q&A sessions with experts, ensuring an engaging and interactive experience for all participants.

About Almirall

Almirall is a global pharmaceutical company dedicated to medical dermatology. We closely collaborate with leading scientists, healthcare professionals, and patients to deliver our purpose: to transform the patients’ world by helping them realize their hopes and dreams for a healthy life. We are at the forefront of science to deliver ground-breaking, differentiated medical dermatology innovations that address patients’ needs.

Almirall, founded in 1944 and headquartered in Barcelona, is publicly traded on the Spanish Stock Exchange (ticker: ALM, total revenue in 2024: €990 MM, over 2000 employees globally). Almirall products help to improve the lives of patients every day and are available in over 100 countries.

For more information, please visit https://www.almirall.com/

Contacts

Corporate Communications
corporate.communication@almirall.com
Phone: (+34) 93 291 35 08

Investor Relations
investors@almirall.com
Phone: (+34) 93 291 30 87

Pint Pharma Announces ANVISA’s Approval of BESREMi® (ropeginterferon alfa-2b) for the Treatment of Polycythemia Vera




Pint Pharma Announces ANVISA’s Approval of BESREMi® (ropeginterferon alfa-2b) for the Treatment of Polycythemia Vera

SÃO PAULO–(BUSINESS WIRE)–Pint Pharma and PharmaEssentia announced today that ANVISA (Brazilian Health Regulatory Agency) has approved BESREMi® (ropeginterferon alfa-2b) for the treatment of adult patients with Polycythemia Vera (PV).

Polycythemia Vera (PV) is a rare, chronic, debilitating, and potentially fatal myeloproliferative neoplasm, originating from a disease-initiating stem cell in the bone marrow. This results in a persistent increase in red blood cells, white blood cells, and platelets. PV can lead to cardiovascular complications such as thrombosis and embolism, and in a significant number of patients it can progress to secondary myelofibrosis or leukemia.

The global incidence of PV is estimated at approximately 2.8 cases per 100,000 people per year. In Brazil, between 2016 and 2020, DataSUS recorded 1,843 cases of Polycythemia Vera, representing an incidence of 0.16 per 100,000 inhabitants. Despite being a rare disease, these numbers suggest the possibility of underdiagnosis and underreporting, highlighting a challenge in identifying affected patients.

According to Fernanda Bertasi, General Manager of Pint Pharma in Brazil, “ANVISA’s approval of BESREMi® represents a transformative milestone for patients with Polycythemia Vera in Brazil. This new therapeutic option brings real hope, offering innovation and progress in disease treatment. This is an essential step in providing better health and well-being for these patients.”

Valnei Canutti, Hematologist and Chief Scientific Officer at Pint Pharma, emphasized that “BESREMi® is an innovative, monopegylated interferon with extended action due to its unique pharmacokinetic properties. Its approval was based on robust evidence from the PEGINVERA clinical study, which demonstrated superior and more sustained hematologic and clinical responses compared to standard therapy.”

For David Ricardo Muñoz Guzmán, CEO of Pint Pharma, “This therapeutic advancement reaffirms Pint Pharma’s commitment to being a community-centered company. Our mission goes beyond innovation and high technology, focusing on solutions that truly impact patients’ lives by expanding access to treatments and improving healthcare quality.”

Ko-Chung Lin, Ph.D., Founder and CEO of PharmaEssentia, added, “The approval of BESREMi® by ANVISA is a testament to the power of strong cross-border partnerships in advancing healthcare and reinforces our mission to transform care for PV patients worldwide. We deeply appreciate the collaboration with the Pint Pharma team, whose expertise and dedication have been instrumental in bringing BESREMi® to patients in Brazil.”

BESREMi® is exclusively registered, marketed, and distributed by Pint Pharma in Brazil.

About BESREMi® (ropeginterferon alfa-2b) in Polycythemia Vera (PV)

BESREMi® is an innovative, monopegylated, long-acting interferon that has marketing authorization in over 40 countries, with approval from the European Medicines Agency (EMA) in 2019, the U.S. Food and Drug Administration (FDA) in 2021, and the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan in 2023.. With its exclusive pegylation technology, BESREMi® offers extended activity in the body and is designed for administration once every two weeks (or once every four weeks if hematologic stability is maintained for at least 1.5 years), allowing for flexible dosing tailored to individual patient needs.

About PharmaEssentia

PharmaEssentia Corporation (TWSE: 6446) headquartered in Taipei, Taiwan, is a global and rapidly growing biopharmaceutical innovator. Leveraging deep expertise and proven scientific principles, PharmaEssentia aims to deliver effective new biologics for challenging diseases in the areas of hematology, oncology, and immunology with one approved product and a diversifying pipeline. Founded in 2003 by a team of Taiwanese American executives and renowned scientists from U.S. biotechnology and pharmaceutical companies, today PharmaEssentia is expanding its global presence with operations in the U.S., Japan, China, and Korea, along with a world-class biologics production facility in Taichung, Taiwan.

About Pint Pharma

Pint Pharma is a biopharmaceutical company that specialises in the commercialisation of innovative therapies with the potential to transform outcomes in rare disease and other patient communities across the Latin American region. By collaborating with world-class biotech and pharma companies, Pint Pharma is committed to improving the lives of Latin American patients in areas of significant unmet medical need. The company is headquartered in Europe and counts with over 250 employees across its territories in Latin America including Brazil, Mexico, Argentina, Colombia, Chile, Peru and Ecuador. Pint Pharma licensed from PharmaEssentia the rights to commercialize BESREMi® in Brazil.

Contacts

Jorge Camacho

Chief of Portfolio Strategy

e-mail: jorge.camacho@pint-pharma.com

Valnei Canutti

Chief of Scientific Affairs

e-mail: valnei.canutti@pint-pharma.com

Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran), the First RNAi Therapeutic to Reduce Cardiovascular Death, Hospitalizations and Urgent Heart Failure Visits in Adults with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)




Alnylam Announces FDA Approval of AMVUTTRA® (vutrisiran), the First RNAi Therapeutic to Reduce Cardiovascular Death, Hospitalizations and Urgent Heart Failure Visits in Adults with ATTR Amyloidosis with Cardiomyopathy (ATTR-CM)

− Novel Mechanism of Action Delivers Rapid Knockdown of Transthyretin, Addressing the Disease at its Source –

− Proven Consistency of Effect on Cardiovascular Outcomes, Function, and Quality of Life in ATTR-CM Population Representative of Today’s Patients –

– Only Therapeutic Approved in the U.S. to Address Both Cardiomyopathy and Polyneuropathy Manifestations of ATTR Amyloidosis −

– Alnylam Offers Multiple Programs to Support Broad and Seamless Patient Access; Majority of Patients Expected to Pay $0 in Out-of-Pocket Costs for AMVUTTRA –

– Alnylam to Host Conference Call Today at 6:00 pm ET –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the U.S. Food and Drug Administration (FDA) approval of the supplemental New Drug Application (sNDA) for its RNAi therapeutic, AMVUTTRA^® (vutrisiran), for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. The approval expands the indication for AMVUTTRA, which now becomes the first and only therapeutic approved by the FDA for the treatment of ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

ATTR-CM is a devastating, rapidly progressive, and ultimately fatal disease estimated to affect approximately 150,000 people in the U.S. and over 300,000 people worldwide. There is no cure for ATTR-CM, and the deposition of misfolded transthyretin (TTR) fibrils causes irreversible damage over time which can lead to premature death. Today, most patients remain undiagnosed and untreated, and a significant portion of those who are treated with commonly used therapies experience disease progression.

“The FDA approval of AMVUTTRA for ATTR-CM marks a pivotal advancement for patients, providing a new and clinically differentiated treatment option that has been shown to improve outcomes, including cardiovascular mortality, and reduce progression for those living with this devastating disease,” said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. “I would like to extend my deepest gratitude to the patients who participated in our clinical trials, their families and caregivers, the clinical researchers, regulators, and my colleagues at Alnylam who made this approval possible. Today represents a significant milestone in our nearly twenty years of partnership with the ATTR amyloidosis community, but we are not stopping here. We will continue to innovate for patients with ATTR amyloidosis so they can live longer, better, healthier lives.”

AMVUTTRA is an RNAi therapeutic that works upstream to deliver rapid knockdown of TTR, addressing the disease at its source, with only four convenient subcutaneous doses per year. By rapidly knocking down TTR production, AMVUTTRA substantially decreases deposition of TTR fibrils, which form amyloid and cause irreversible cardiovascular damage and premature death in patients with ATTR-CM.

“This FDA approval provides an opportunity to further transform ATTR-CM treatment with a new mechanism of action. The HELIOS-B clinical trial found that vutrisiran allowed patients to live longer, experience fewer hospitalizations, and improve how they function and feel,” said Ronald Witteles, M.D., HELIOS-B Investigator, Professor of Medicine at Stanford University School of Medicine and Co-Director of the Stanford Amyloid Center. “The trial enrolled patients who mirror the real-world population with this disease, and I am very encouraged by vutrisiran’s ability to demonstrate meaningful clinical benefits across both cardiovascular outcomes and multiple measures of disease progression. This is a very exciting day for patients with this challenging disease.”

This approval is based on the HELIOS-B Phase 3 clinical trial which evaluated AMVUTTRA for the treatment of ATTR-CM. The trial achieved statistical significance compared to placebo on all 10 pre-specified primary and secondary endpoints. The results were presented at the European Society of Cardiology Congress and simultaneously published in The New England Journal of Medicine. In the overall population, AMVUTTRA reduced the risk of all-cause mortality (ACM) and recurrent cardiovascular (CV) events by 28% during the double-blind treatment period of up to 36 months. Mortality in this population was significantly reduced by 36% through 42 months in a pre-specified secondary endpoint analysis which included up to 36 months of the double-blind period plus six months of open-label extension. In the monotherapy population, AMVUTTRA significantly reduced the risk of ACM and recurrent CV events by 33% in the double-blind period and significantly reduced the risk of mortality by 35% through 42 months. As compared to patients treated with placebo, patients treated with AMVUTTRA also experienced preservation of functional capacity and quality of life, as well as early improvements in biomarkers NT-proBNP and troponin I, which are predictive of cardiovascular outcomes. The safety and tolerability of AMVUTTRA are well-established, as demonstrated in the positive HELIOS-A clinical trial for AMVUTTRA in hATTR-PN which resulted in FDA approval in 2022 and over 5,000 patient-years exposure to-date, globally. In that study, the most common adverse reactions in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%). No new safety concerns were identified in the HELIOS-B clinical trial of patients with ATTR-CM.

“Despite recent advances, there remains a significant need for patients living with ATTR-CM and I’ve witnessed, firsthand, the impact that ATTR amyloidosis can have on families, including diminished quality of life and the loss of loved ones,” said Muriel Finkel, President of the Amyloidosis Support Groups. “The availability of this groundbreaking treatment option is a significant moment for patients living with ATTR amyloidosis. It represents a beacon of hope for our community.”

AMVUTTRA in hATTR-PN is covered by insurers for ~99% of patients with the majority paying $0 out-of-pocket. Similar broad coverage and out-of-pocket costs are expected in ATTR-CM given comparable payer dynamics and the clinical value demonstrated in the HELIOS-B clinical trial. Alnylam has a proven track record of working closely with payers to ensure broad patient access to our medicines, including pioneering innovative value-based agreements linked to clinical outcomes that ensure value and predictability for payers.

Alnylam offers multiple support services and resources for patients prescribed its products through Alnylam Assist^®, the only in-house patient support program in ATTR-CM. Alnylam Assist^® is designed to provide one-on-one support for patients and their healthcare teams to help navigate the treatment journey, and includes a Quick Start program that provides eligible patients who encounter coverage delays or delays in treatment with an initial dose at no cost. In addition, Alnylam Assist^® offers support with insurance coverage, financial assistance, and disease and treatment education for a seamless start to the AMVUTTRA treatment experience. Physicians and patients can learn more about Alnylam’s patient support services by visiting AlnylamAssist.com/amvuttra or calling 1-833-256-2748.

Marketing authorization applications based on HELIOS-B data are currently under review by several global health agencies including the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA), and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025.

Visit AMVUTTRA.com/attr-cm for more information.

Investor Webcast Information

Alnylam management will discuss the FDA approval of AMVUTTRA for ATTR-CM via webcast today at 6:00 pm ET. A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events. An archived webcast will be available on the Company’s website approximately two hours after the event.

INDICATIONS AND IMPORTANT SAFETY INFORMATION

Indications

AMVUTTRA^® (vutrisiran) is indicated for the treatment of the:

• cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits.
• polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation

AMVUTTRA treatment leads to a decrease in serum vitamin A levels.

Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.

Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions

In a study of patients with hATTR-PN, the most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (15%), arthralgia (11%), dyspnea (7%), and vitamin A decreased (7%).

In a study of patients with ATTR-CM, no new safety issues were identified.

For additional information about AMVUTTRA, please see the full Prescribing Information.

About AMVUTTRA® (vutrisiran)

AMVUTTRA^® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection by a healthcare professional, AMVUTTRA is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and is approved in the U.S. for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. For more information about AMVUTTRA, including the full U.S. Prescribing Information, visit AMVUTTRA.com.

About ATTR

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.^1-4

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam Pharmaceuticals (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO^® (patisiran), AMVUTTRA^® (vutrisiran), GIVLAARI^® (givosiran), OXLUMO^® (lumasiran), and Leqvio^® (inclisiran), which is being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram.

Alnylam Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding Alnylam’s expectations regarding the safety and efficacy of AMVUTTRA for the treatment of ATTR-CM and hATTR-PN; that FDA approval of AMVUTTRA for ATTR-CM represents a pivotal advancement for patients, providing a new and clinically differentiated treatment option for ATTR-CM; the ability of AMVUTTRA to improve outcomes and reduce progression for patients with ATTR-CM; Alnylam’s ability to continue to innovate for patients with ATTR amyloidosis; the ability of AMVUTTRA or any of Alnylam’s other products or product candidates to enable patients with ATTR amyloidosis to live longer, better and/or healthier lives; the potential for AMVUTRA to transform the treatment of ATTR-CM; the ability of AMVUTTRA to provide ATTR-CM patients with benefits comparable to the clinical benefits observed in the HELIOS-B clinical trial; Alnylam’s expectations that AMVUTTRA in ATTR-CM will have coverage and out-of-pocket cost dynamics similar to AMVUTTRA’s coverage and out-of-pocket cost dynamics in hATTR-PN; and the potential receipt and timing of any future marketing authorizations or the timing of any future regulatory submissions for vutrisiran, should be considered forward looking statements.

Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam’s ability to successfully execute on its “Alnylam P⁵x25” goals; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing Alnylam’s views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

¹ Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638.

² Gertz MA. Am J Manag Care. 2017;23(7):S107-S112.

³ Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9.

⁴ Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.

Contacts

Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom

(Investors and Media)

+1-617-682-4340

Josh Brodsky

(Investors)

+1-617-551-8276

Aptar Recognized with Prestigious ‘A’ Score on the CDP Climate Change Assessment




Aptar Recognized with Prestigious ‘A’ Score on the CDP Climate Change Assessment

CRYSTAL LAKE, Ill.–(BUSINESS WIRE)–AptarGroup, Inc. (NYSE: ATR), a global leader in drug and consumer product dosing, dispensing and protection technologies, has been recognized for its leadership on climate change topics by the global environmental non-profit CDP, securing a place on its prestigious “A List.”

Aptar was recognized for its actions to cut emissions, mitigate climate risks and further the low-carbon economy, based on the data reported by the Company through CDP’s 2024 corporate questionnaire. In 2024, over 700 financial institutions with over 142 trillion USD in assets requested companies to disclose data on environmental impacts, risks, and opportunities through CDP’s platform. A record-breaking 24,800 companies responded. Through significant demonstrable action on climate, Aptar is a leader in corporate environmental ambition, action and transparency worldwide.

“We are proud to be recognized by CDP as a leader on climate related topics. Our actions that demonstrate our continuous efforts in this area, and the products and solutions we provide, play an important role in improving everyday life for consumers and patients around the world,” said Stephan B. Tanda, Aptar President and CEO. “As we focus on the future, we intend to continue our efforts on reducing climate risks and increasing actions that lead to a more sustainable future.”

Aptar recognizes that caring for the environment means reducing climate emissions. Aptar has set formalized science-based targets for Scope 1 and Scope 2 emissions reductions that are in line with requirements to keep global warming at 1.5° Celsius by 2030. In addition, the Company has a renewable electricity target, as well as a Scope 3 target. Aptar’s targets have been validated by the Science Based Targets Initiative (SBTi). The Company also improved its risk and opportunities disclosures in accordance with the Task Force on Climate-Related Financial Disclosures (TCFD). In addition, Aptar received the ISO 14046 certification, assuring the reduction in greenhouse gas emissions in all scopes, especially as it increased renewable electricity purchases. Aptar has signed Power Purchase Agreements (PPA) in Europe and the US to provide more localized sources of renewable energy dedicated to Aptar.

“It is encouraging that Aptar’s progress is recognized by CDP, especially given our ever-changing global environment. As companies across the globe aim to become more transparent, we believe it is important to share our progress with the world and reduce our carbon emissions year-over-year. This is why our focus at Aptar has been on continuous improvement and collaborating with our teams around the world as we make the next steps on our journey,” said Beth Holland, Chief Sustainability Officer.

A detailed and independent methodology is used by CDP to assess these companies, allocating a score of A to D- based on the comprehensiveness of disclosure, awareness and management of environmental risks as well as demonstration of best practices associated with environmental leadership, such as setting ambitious and meaningful targets. CDP disclosure and the A List rankings help organizations on their journey of continuous improvement. CDP, in collaboration with other leading frameworks, set the bar on what qualifies as environmental leadership and increased transparency in line with emerging science, stakeholder feedback, and market needs.

Paul Simpson, Chief Delivery Officer of CDP, said, “As the founder of environmental reporting, CDP is dedicated to building a world where people, planet and profit are truly balanced. We greatly appreciate the support of Aptar in our efforts to continue pioneering transparency, powering corporate environmental action. Transparency drives action at all levels…. Disclosure is far more than a box to tick. It’s a tool to see clearly, act decisively and create change.”

About Aptar

Aptar is a global leader in drug and consumer product dosing, dispensing and protection technologies. Aptar serves a number of attractive end markets including pharmaceutical, beauty, food, beverage, personal care and home care. Using market expertise, proprietary design, engineering and science to create innovative solutions for many of the world’s leading brands, Aptar in turn makes a meaningful difference in the lives, looks, health and homes of millions of patients and consumers around the world. Aptar is headquartered in Crystal Lake, Illinois and has over 13,000 dedicated employees in 20 countries. For more information, visit www.aptar.com.

About CDP

CDP is a global non-profit that runs the world’s environmental disclosure system for companies, cities, states and regions. Founded in 2000 and working with more than 700 financial institutions with over $142 trillion in assets, CDP pioneered using capital markets and corporate procurement to motivate companies to disclose their environmental impacts, and to reduce greenhouse gas emissions, safeguard water resources and protect forests. Over 24,000 organizations around the world disclosed data through CDP in 2023, with more than 23,000 companies – including listed companies worth two thirds global market capitalization – and over 1,100 cities, states and regions. Fully TCFD aligned, CDP holds the largest environmental database in the world, and CDP scores are widely used to drive investment and procurement decisions towards a zero carbon, sustainable and resilient economy. CDP is a founding member of the Science Based Targets initiative, We Mean Business Coalition, The Investor Agenda and the Net Zero Asset Managers initiative. Visit www.cdp.net or follow us @CDP to find out more.

This press release contains forward-looking statements, including with regard to our sustainability goals and targets. Expressions or future or conditional verbs such as “will” are intended to identify such forward-looking statements. Forward-looking statements are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934 and are based on our beliefs as well as assumptions made by and information currently available to us. Accordingly, our actual results may differ materially from those expressed or implied in such forward-looking statements due to known or unknown risks and uncertainties that exist in our operations and business environment including, but not limited to: the successful integration of acquisitions; the regulatory environment; and competition, including technological advances. For additional information on these and other risks and uncertainties, please see our filings with the Securities and Exchange Commission, including the discussion under “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” in our Form 10-Ks and Form 10-Qs. We undertake no obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Contacts

Aptar Investor Relations Contact:
Mary Skafidas

mary.skafidas@aptar.com
+1 347 351 6407

Aptar Media Contact:
Katie Reardon

katie.reardon@aptar.com
+1 815 479 5671

Poxel Announces Positive Results from a Preclinical Study for PXL065, a Proprietary Deuterium-Stabilized R-Stereoisomer of Pioglitazone, in Hypertrophic Cardiomyopathy




Poxel Announces Positive Results from a Preclinical Study for PXL065, a Proprietary Deuterium-Stabilized R-Stereoisomer of Pioglitazone, in Hypertrophic Cardiomyopathy

• Hypertrophic cardiomyopathy (HCM) is the most common human genetic cardiac disorder and can lead to serious complications including heart failure and sudden cardiac death
• The preclinical study was financed through a grant by DZHK¹ and conducted at the TUM University Hospital German Heart Center under a research collaboration² between Poxel and the TUM University Hospital German Heart Center
• PXL065 demonstrated significant benefits in a HCM mouse model preventing pathological myocardial remodeling, including hypertrophy and fibrosis in the heart
• The top-line results from this mouse model support the clinical development of PXL065 as a potential disease-modifying treatment for symptomatic and asymptomatic HCM

LYON, France–(BUSINESS WIRE)–Regulatory News:

POXEL SA (Euronext : POXEL – FR0012432516), a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including metabolic dysfunction-associated steatohepatitis (MASH) and rare metabolic disorders, today announces the positive top-line results for PXL065 from a preclinical study conducted in a mouse model of hypertrophic cardiomyopathy. PXL065 is a novel, proprietary deuterium-stabilized R-stereoisomer of pioglitazone which is known to reduce inflammation and fibrosis, improve mitochondrial function, and restore metabolic balance.

Thomas Kuhn, CEO of Poxel, stated: “We are very pleased with these top-line results, which illustrate the potential of PXL065 for treating hypertrophic cardiomyopathy, the most common genetic cardiac disorder. Current therapeutic options for this disease are limited, with either low efficacy, a difficult safety profile or addressing a limited patient population. There is therefore a high medical need for novel, effective and well-tolerated treatments that would prevent disease progression and help to avoid invasive procedures such as heart surgery. We look forward to initiating the clinical development of PXL065 in this indication based on these promising results.”

Prof. Dr. Cordula Wolf, Director of the Center for Rare Congenital Heart Diseases at the TUM University Hospital German Heart Center, added: “The findings of the pre-clinical study conducted in collaboration with Poxel represent a major step in the development of a novel therapeutic approach to treat hypertrophic cardiomyopathy, a severe and progressive disease that can lead to life-threatening cardiac events. Results obtained during this preclinical study showed that PXL065 may have the potential to improve the clinical outcomes for patients suffering from this genetic condition by reducing left ventricular hypertrophy, decreasing cardiac fibrosis and improving the underlying pathophysiological mechanisms. The profile of PXL065 also compares well versus standard of care, including mavacamten, with a highly differentiated mechanism of action. Building on the data package available for this novel compound, in our view the study results support the development of PXL065 as a disease modifier and long-term treatment for HCM patients.”

Hypertrophic Cardiomyopathy (HCM) is a genetic disorder marked by myocardial hypertrophy, cardiac fibrosis, ventricular dysfunction, arrhythmias, and an increased risk of sudden cardiac death. It is caused by mutations in sarcomere protein genes, leading to altered cell metabolism, including oxidative stress and mitochondrial dysfunction. The estimated prevalence of HCM is 0.2%, or 1/500 adults, and its incidence is around 5 per 100,000 person-years.

In connection with the mechanism of action of PXL065 on the inhibition of the mitochondrial pyruvate carrier (MPC) and on the inhibition of Acyl CoA Synthetase 4 (ACSL4) thus acting on oxidative stress, inflammation and fibrosis, PXL065 was tested in an established mouse model of hypertrophic cardiomyopathy. After 10 weeks of treatment, a significant reduction in myocardial hypertrophy associated with a significant reduction in cardiac fibrosis was demonstrated, highlighting the potential of PXL065 in this pathology.

This preclinical study was funded by the German Center for Cardiovascular Research (DZHK) and conducted at the TUM University Hospital German Heart Center by leading HCM expert Prof. Dr. Cordula Wolf. Poxel and the TUM University Hospital German Heart Center collaborated on the pre-clinical study based on Poxel’s existing data and patent portfolio on PXL065 and prior research conducted by Prof. Dr. Cordula Wolf and her group on the disease mechanisms and therapeutic use of TZD’s in HCM. The top-line results available indicate that PXL065 prevents pathological myocardial remodeling in a HCM mouse model, including hypertrophy and cardiac fibrosis. A detailed analysis of the transcriptome and proteomics will help evaluate the mechanistic pathways of PXL065. The deuterium-stabilized R-Pioglitazone may thus provide a promising therapeutic approach for the long-term treatment of HCM.

The full results of this preclinical study have been submitted for presentation at an upcoming scientific meeting.

In parallel of finalizing the analysis of this study and pending additional financing, Poxel plans to define the best patient population for HCM that could benefit from PXL065 based on the data available and its mechanism of action and also to elaborate the associated clinical development and regulatory plan under advice from leading HCM experts including Prof. Dr. Cordula Wolf.

About Poxel SA

Poxel is a clinical stage biopharmaceutical company developing innovative treatments for chronic serious diseases with metabolic pathophysiology, including metabolic dysfunction-associated steatohepatitis (MASH) and rare disorders. For the treatment of MASH, PXL065 (deuterium-stabilized R-pioglitazone) met its primary endpoint in a streamlined Phase 2 trial (DESTINY-1). In rare diseases, development of PXL770, a first-in-class direct adenosine monophosphate-activated protein kinase (AMPK) activator, is focused on the treatment of adrenoleukodystrophy (ALD) and autosomal dominant polycystic kidney disease (ADPKD). TWYMEEG^® (Imeglimin), Poxel’s first-in-class product that targets mitochondrial dysfunction, is now marketed for the treatment of type 2 diabetes in Japan by Sumitomo Pharma and Poxel expects to receive royalties and sales-based payments. Poxel has a strategic partnership with Sumitomo Pharma for Imeglimin in Japan. Listed on Euronext Paris, Poxel is headquartered in Lyon, France, and has subsidiaries in Boston, MA, and Tokyo, Japan.

For more information, please visit: www.poxelpharma.com

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. The Company does not endorse or is not otherwise responsible for the content of external hyperlinks referred to in this press release.

¹ DZHK: German Center for Cardiovascular Research (Deutsches Zentrum für Herz-Kreislauf-Forschung)

² The research collaboration between Poxel and the TUM University Hospital German Heart Center currently entails evaluating the potential of PXL065 for the treatment of HCM in pre-clinical studies under a research grant obtained from DZHK

Contacts

Investor relations / Media

NewCap

Nicolas Fossiez, Aurélie Manavarere / Arthur Rouillé

investors@poxelpharma.com
+33 1 44 71 94 94

Alto Neuroscience Reports Full-Year 2024 Financial Results and Recent Business Highlights




Alto Neuroscience Reports Full-Year 2024 Financial Results and Recent Business Highlights

– Favorable outcome from interim analysis of ongoing ALTO-300 Phase 2b MDD trial; topline results expected in mid-2026 –

– Completed enrollment in ALTO-203 Phase 2 proof-of-concept MDD trial; on track to report topline data in the second quarter of 2025 –

– Novel transdermal formulation of ALTO-101 demonstrated greater drug exposure and improved tolerability profile; topline data from Phase 2 trial of ALTO-101 in schizophrenia expected in the second half of 2025 –

– Company’s precision psychiatry approach underpins recently granted patents covering the use of ALTO-100 and ALTO-300 in biomarker defined populations –

– Strong cash position of approximately $169 million expected to fund planned operations into 2028, and through at least four upcoming clinical study readouts –

MOUNTAIN VIEW, Calif.–(BUSINESS WIRE)–$ANRO #PrecisionPsychiatry–Alto Neuroscience, Inc. (“Alto”) (NYSE: ANRO) a clinical-stage biopharmaceutical company focused on the development of novel precision medicines for neuropsychiatric disorders, today reported financial results for the full-year ended December 31, 2024, and highlighted recent progress across its pipeline of clinical-stage product candidates.

“In 2024 we continued to advance our mission to change the way medicines are developed for neuropsychiatric conditions,” said Amit Etkin, M.D., Ph.D., founder and chief executive officer of Alto Neuroscience. “Using our proprietary platform we have evaluated brain-based biomarkers in over 2,000 participants and have completed six clinical studies across our pipeline programs since our founding. Further, we believe the outcome of the recent interim analysis of the ongoing ALTO-300 trial is suggestive of antidepressant activity, and we believe the sample re-estimation improves the overall probability of success in that trial.”

Dr. Etkin continued, “Successfully completing our IPO in 2024 has provided us with a strong balance sheet to support several key clinical milestones in the coming years. As a scientific founder with a sincere passion to move the field forward, I am very proud of all our team has accomplished and I look forward to building on this momentum.”

Pipeline Highlights

ALTO-100: Enrollment ongoing in Phase 2b in Bipolar Depression; Phase 2b in MDD complete.

ALTO-100, a first-in-class, oral small molecule believed to work through enhancing neuroplasticity, is in development for the treatment of bipolar depression (BPD) in patients characterized by a cognitive biomarker.

• Enrollment in the randomized, double-blind, placebo-controlled Phase 2b trial remains ongoing with topline data expected in the second half of 2026. The Company expects to enroll approximately 200 patients with BPD. Patients will be evaluated over a six-week treatment period and the primary endpoint is the change from baseline on the Montgomery-Åsberg Depression Rating Scale (MADRS) in the patient population characterized by a cognitive biomarker.
• In October 2024, the Company reported topline results from the Phase 2b trial evaluating ALTO-100 as a treatment for major depressive disorder (MDD). The cognitive biomarker-defined MDD (biomarker positive) patient group treated with ALTO-100 (n=196) did not demonstrate a statistically significant improvement in depressive symptoms compared to placebo in the modified intention to treat (mITT) population. ALTO-100 continued to demonstrate a favorable safety and tolerability profile, with no new safety signals observed in this study.
  • In a pre-specified analysis of biomarker positive patients taking ALTO-100 or placebo adjunctively to a stable background antidepressant (n=61), ALTO-100 demonstrated a clinically meaningful effect compared to placebo at week 6 (Cohen’s d=0.47, p=0.09). The pre-specified analysis of this population was not powered to achieve statistical significance.
    • Detailed analysis of the study results revealed a significantly lower rate of study medication compliance within the monotherapy group compared to the adjunctive group (56% in monotherapy vs. 100% in adjunctive)
  • It was observed that in a subset of patients that had evidence of taking ALTO-100 through blood sample analysis, the patients with the ALTO-100 cognitive biomarker demonstrated a greater improvement compared to placebo and demonstrated a greater improvement than those without the biomarker.
  • Based on the clinically meaningful signal in the adjunctive subgroup and the evidence of biomarker enrichment in the compliant subset of patients, the Company made the decision to continue the Phase 2b trial of ALTO-100 as an adjunctive treatment in BPD.

ALTO-300: Completed interim analysis, resulting in a recommendation to continue the Phase 2b adjunctive MDD trial.

ALTO-300, also known as agomelatine, is an oral, small molecule designed to act as a melatonin agonist and 5-HT2C antagonist, is being developed as an adjunctive treatment in the United States for patients with MDD, characterized by an EEG biomarker. Agomelatine is an approved antidepressant medication in Europe and Australia, but has not been approved in the United States.

• Topline data from the double-blind, placebo-controlled, randomized Phase 2b trial is expected in mid-2026. In the study, patients who have had an inadequate response to their current antidepressant are randomized to receive ALTO-300 or placebo over a six-week treatment period. The study medication is being taken in addition to a patient’s background antidepressant. The primary outcome is the change from baseline in MADRS score in patients with the EEG biomarker.
• In February 2025, the Company reported a favorable outcome from the planned interim analysis for the Phase 2b trial of ALTO-300 as an adjunctive treatment for patients with MDD. Based on the results of the interim analysis, the Phase 2b trial is continuing with a target enrollment of approximately 200 biomarker positive patients for the final analysis sample.
  • Prior to the interim analysis, a blinded committee conducted an in-depth site and patient eligibility review that resulted in the prospective exclusion of sites and patients from the analysis population. Following the eligibility review, the biomarker positive population in the interim analysis consisted of 87 patients.
• The Company presented clinical advancements at multiple scientific conferences that provide further insight into the patient phenotype being studied in the Phase 2b trial. Approximately 50% of patients with MDD are estimated to be positive for the ALTO-300 EEG biomarker.
  • In February 2025, a poster was presented at the International Society for CNS Clinical Trials and Methodology (ISCTM) 21^st Annual Scientific Meeting, demonstrating the successful standardization of EEG enrichment marker acquisition through a platform that facilitates the identification of patients more likely to respond to ALTO-300.
  • In December 2024, a poster was presented at the 63rd Annual Meeting of the American College of Neuropsychopharmacology (ACNP) from a reverse-translation preclinical study demonstrating that the ALTO-300 biomarker is a measure of reduced neural signal stability and can be generated by activating the 5-HT2C receptor. These findings mechanistically tie the EEG biomarker to ALTO-300’s mechanism of action as a 5-HT2C antagonist.

ALTO-203: Enrollment completed in Phase 2 proof-of-concept MDD trial; data expected in the second quarter of 2025.

ALTO-203, a novel, oral small molecule designed to uniquely act as a histamine H3 inverse agonist, is being developed for the treatment of MDD associated with increased levels of anhedonia.

• In February, the Company completed enrollment in the Phase 2 proof-of-concept (POC) trial in MDD patients with higher levels of anhedonia.
• The trial enrolled 69 patients and consists of two sequential double-blind, placebo-controlled treatment periods with two dose levels of ALTO-203 as a monotherapy.
• The proof-of-concept trial is designed to evaluate the subjective and objective effects of ALTO-203 in patients with MDD.
• The Company expects to report topline data in the second quarter of 2025.

ALTO-101: Enrollment ongoing in Phase 2 proof-of-concept CIAS trial.

ALTO-101, a brain-penetrant PDE4 inhibitor designed as a novel transdermal formulation, is being developed for the treatment of Cognitive Impairment Associated with Schizophrenia (CIAS). Through this unique formulation, ALTO-101 is designed to retain the desired brain effects shown with the oral formulation and avoid tolerability challenges and adverse effects known to be associated with PDE4 inhibitors.

• Enrollment remains ongoing in the Phase 2 POC trial in CIAS, with topline data expected in the second half of 2025.
• The Phase 2 POC trial consists of a dose-escalating treatment with ALTO-101 and is designed to enroll approximately 70 adult participants with schizophrenia between the ages of 21 and 55.
  • The primary outcome in the study is the effect of ALTO-101 on theta band activity, the EEG measure shown to be best related to CIAS in replicated analyses of large schizophrenia datasets. Objective cognitive performance will also be evaluated.
• In April 2024, the Company reported positive Phase 1 results for ALTO-101 in healthy subjects.
  • The Company’s proprietary transdermal formulation of ALTO-101 exhibited greater systemic drug exposure than orally administered ALTO-101 while also demonstrating a significant reduction in typical class-related adverse events. All adverse events reported in the study were mild in severity, and there were no reported serious adverse events.
  • The transdermal formulation demonstrated favorable adhesion properties and there were no application site reactions that led to removal or intolerance.

Business Highlights

• In the first quarter of 2025, the Company strengthened its patent estate:
  • Development of ALTO-300 in the U.S. is protected by granted patents covering the use of ALTO-300 as an adjunctive therapy and patient selection characterized by an EEG biomarker. As granted, not accounting for any potential patent term extensions, the patent is expected to cover the use of ALTO-300 in the EEG-defined patient population until 2044.
  • Development of ALTO-100 in the U.S. is protected by granted patents covering methods of treating BPD and MDD in patients with objectively determined impaired learning and/or memory with a specific dose range of ALTO-100. As granted, not accounting for any potential patent term extensions, the patents are expected to cover the use of ALTO-100 in the specified patient populations until 2043.
• In July 2024, the Company received an $11.7 million funding award from the Wellcome Trust to advance the clinical development of ALTO-100 through a Phase 2b study in patients with BPD.
• In February 2024, the Company successfully completed its upsized initial public offering resulting in net proceeds of approximately $133 million.

Upcoming Milestones and Events

Near-Term Expected Milestones

• 2Q 2025 — ALTO-203 Phase 2 POC MDD trial topline data
• 2H 2025 — ALTO-101 Phase 2 POC CIAS trial topline data
• Mid 2026 — ALTO-300 Phase 2b MDD trial topline data
• 2H 2026 — ALTO-100 Phase 2b bipolar depression trial topline data

Upcoming Conferences

• Members of the Company’s management team are expected to present at the following upcoming conferences:
  • Annual Congress of the Schizophrenia International Research Society (SIRS): March 29-April 2, 2025
  • Society of Biological Psychiatry (SOBP) Annual Meeting: April 24-26, 2025
  • American Society of Clinical Psychopharmacology (ASCP) Annual Meeting: May 27-30, 2025

Full-Year 2024 Financial Highlights

Cash Position: As of December 31, 2024 the Company had cash, cash equivalents, and restricted cash of approximately $169 million, compared to approximately $83 million in cash, cash equivalents, and restricted cash as of December 31, 2023.

The Company expects its cash balance to support planned operations into 2028.

R&D Expenses: Research and development expenses for the full year ended December 31, 2024 were $47.0 million, as compared to $30.3 million for the same period in 2023. The increase was primarily attributable to increased personnel costs; including $1.5 million of non-cash, stock-based compensation; as well as costs associated with the Phase 2b clinical study for ALTO-300, and the Phase 2 POC studies for ALTO-203 and ALTO-101.

G&A Expenses: General and administrative expenses for the full year ended December 31, 2024 were $21.6 million, as compared to $7.5 million for the same period in 2023. The increase was primarily attributable to costs associated with higher headcount to support expanded clinical development efforts, growing operational requirements, and costs associated with operating as a public company. $3.3 million of the increase is reflective of non-cash, stock-based compensation.

Net Loss: The Company incurred a net loss of $61.4 million for the full year ended December 31, 2024, as compared to $36.3 million for the year ended December 31, 2023.

About Alto Neuroscience

Alto Neuroscience is a clinical-stage biopharmaceutical company with a mission to redefine psychiatry by leveraging neurobiology to develop personalized and highly effective treatment options. Alto’s Precision Psychiatry Platform™ measures brain biomarkers by analyzing EEG activity, neurocognitive assessments, wearable data, and other factors to better identify which patients are more likely to respond to Alto product candidates. Alto’s clinical-stage pipeline includes novel drug candidates in bipolar depression, major depressive disorder, schizophrenia, and other mental health conditions. For more information, visit www.altoneuroscience.com or follow Alto on X.

Forward-Looking Statements

This press release may contain forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as “aims,” “anticipates,” “believes,” “could,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “look forward,” “may,” “plans,” “possible,” “potential,” “seeks,” “will” and variations of these words or similar expressions that are intended to identify forward-looking statements, although not all forward-looking statements contain these words. Forward-looking statements in this press release include, but are not limited to, statements regarding Alto’s expectations with regard to the potential benefits, activity, effectiveness and safety of its product candidates and Precision Psychiatry Platform (“Platform”); Alto’s expectations with regard to the design and results of its research and development programs and clinical trials, including the timing of enrollment and the timing and availability of data from such trials; Alto’s clinical and regulatory development plans for its product candidates, including the timing or likelihood of regulatory filings and approvals for its product candidates; Alto’s business strategy, financial position and the sufficiency of its financial resources to fund its operations through expected milestones; and other statements that are not historical fact. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation, progress and completion of clinical trials and clinical development of Alto’s product candidates; the risk that Alto may not realize the intended benefits of its Platform; availability and timing of results from clinical trials; whether initial or interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; the risk that clinical trials may have unsatisfactory outcomes; the risk that Alto’s projections regarding its financial position and expected cash runway are inaccurate or that its conduct of its business requires more cash than anticipated; and other important factors, any of which could cause Alto’s actual results to differ from those contained in the forward-looking statements, which are described in greater detail in Alto’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 filed with the Securities and Exchange Commission (“SEC”) as well as in other filings Alto may make with the SEC in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Alto expressly disclaims any obligation to update any forward-looking statements contained herein, whether because of any new information, future events, changed circumstances or otherwise, except as required by law.

Availability of Information on Alto’s Website

Alto routinely uses its investor relations website to post presentations to investors and other important information, including information that may be material. Accordingly, Alto encourages investors and others interested in Alto to review the information it makes public on its investor relations website.

Contacts

Investor Contact:
Nick Smith

investors@altoneuroscience.com

Media Contact:
Mari Purpura

media@altoneuroscience.com

Allurion Announces Initial Results on the Combination of the Allurion Program With Low-dose GLP-1 Therapy to Optimize Muscle Mass and GLP-1 Adherence




Allurion Announces Initial Results on the Combination of the Allurion Program With Low-dose GLP-1 Therapy to Optimize Muscle Mass and GLP-1 Adherence

Average Total Weight Loss of 20% With 15% Increase in Lean Body Mass After 8 Months of Combined Therapy

NATICK, Mass.–(BUSINESS WIRE)–$ALUR–Allurion Technologies, Inc. (“Allurion” or the “Company”) (NYSE: ALUR), a company dedicated to ending obesity, today announced initial results on the combination of the Allurion Program with low-dose GLP-1 therapy to optimize muscle mass and GLP-1 adherence.

52 patients treated with the Allurion Balloon were started on 0.25mg semaglutide after completing their first month of balloon therapy. The dose of semaglutide was increased to no greater than 1.0mg over the subsequent 6 months. After 8 months of this combination approach, average total body weight loss was 20.3%, and lean body mass increased by 15% from 59.6% to 68.5%. All patients remained adherent to the GLP-1 medication through 8 months.

“I believe combining the Allurion Balloon with low-dose GLP-1 therapy has several advantages,” said Dr. Luigi Flagiello, bariatric surgeon at Clinica Ruesch who oversaw this case series. “These results indicate that a combination approach increases weight loss and that by using lower doses of GLP-1s, patients do not experience as many side effects—including muscle wasting—and have improved adherence. Patients may also be benefiting from two distinct physiological impacts: satiety induction from the balloon and reduced hunger from the GLP-1.”

Previous studies in patients undergoing GLP-1 therapy have demonstrated reductions in lean mass of approximately 40% as a proportion of total weight lost¹ and have also shown that 30% of patients discontinue GLP-1 therapy within the first month and 58% discontinue before reaching a clinically meaningful health benefit², due in part to side effects, dose escalation required for continued weight loss, and cost. Semaglutide dosing is typically increased to 2.4mg, over two times higher than the maximum dose of 1.0mg used in the combination approach.

“Muscle wasting and lack of adherence are significant challenges for GLP-1s, and this initial data suggests that a combination approach that leverages Allurion’s full program—which includes the balloon, our Virtual Care Suite, and our behavior change program—may be a compelling solution,” said Dr. Shantanu Gaur, Founder and CEO of Allurion. “We plan on building upon this promising initial data and are optimistic that this could become a new standard of care for patients who want metabolically healthy weight loss.”

Additional data on the combination approach is being collected as part of this case series and is expected to be presented at upcoming medical meetings.

About Allurion

Allurion is dedicated to ending obesity. The Allurion Program is a weight-loss platform that combines the Allurion Gastric Balloon, the world’s first and only swallowable, Procedureless^TM gastric balloon for weight loss, the Allurion Virtual Care Suite, including the Allurion Mobile App for consumers and Allurion Insights for healthcare providers featuring the Iris AI Platform, and the Allurion Connected Scale. The Allurion Virtual Care Suite is also available to providers separately from the Allurion Program to help customize, monitor, and manage weight-loss therapy for patients regardless of their treatment plan. The Allurion Gastric Balloon is an investigational device in the United States.

For more information about Allurion and the Allurion Virtual Care Suite, please visit www.allurion.com.

Forward-Looking Statements

This press release contains certain forward-looking statements within the meaning of the U.S. federal and state securities laws. These forward-looking statements generally are identified by the words “believe,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “strategy,” “future,” “opportunity,” “plan,” “target,” “may,” “should,” “will,” “would,” “will be,” “will continue,” “will likely result,” and similar expressions and include statements regarding the expected efficacy of a combination approach of the Allurion Program and GLP-1 use for weight loss management, the timing and results of additional and future studies and trials involving such treatment plans, market acceptance of such new combined therapies, the uniqueness of Allurion’s product and service offerings and other statements about future events that reflect the current beliefs and assumptions of Allurion’s management based on information currently available to them and, as a result, are subject to risks and uncertainties. Forward-looking statements are predictions, projections and other statements about future events that reflect the current beliefs and assumptions of Allurion’s management based on information currently available to them and, as a result, are subject to risks and uncertainties. Many factors could cause actual future results or developments to differ materially from the forward-looking statements in this press release, including but not limited to (i) the ability of Allurion to obtain regulatory approval for and successfully commercialize the Allurion Program, (ii) the timing of and results from its clinical studies and trials, including those involving a combination approach to weight loss treatment, (iii) the evolution of the markets in which Allurion competes and the increasing acceptance of GLP-1 drugs, (iv) the ability of Allurion to defend its intellectual property and satisfy regulatory requirements, (v) the impact of acts of war and terrorism, including the Russia-Ukraine war and Israel-Hamas war and political instability in general on Allurion’s business, (vi) Allurion’s expectations regarding its market opportunities, (vii) the outcome of any legal proceedings against Allurion, (viii) the risk of economic downturns and a Allurion’s ability to respond to a changing regulatory landscape in the highly competitive industry in which it operates, and (ix) uncertainties related to market conditions and economic conditions in general, including tariffs, trade wars, recessions, interest rates and currency fluctuations. The foregoing list of factors is not exhaustive. You should carefully consider the foregoing factors and the other risks and uncertainties described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K filed on March 26, 2024 and Amendment No. 1 thereto filed on April 29, 2024, the Company’s Quarterly Report on Form 10-Q filed during fiscal 2024 and other documents filed by Allurion from time to time with the U.S. Securities and Exchange Commission. These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and Allurion assumes no obligation and does not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise. Allurion does not give any assurance that it will achieve its expectations.

¹ Wilding et al. NEJM. 2021, 384, 989-1002; 10.1056/NEJMoa2032183

² https://www.bcbs.com/about-us/association-news/most-americans-stop-weight-loss-drugs-before-seeing-meaningful-benefit

Contacts

Global Media and Investor Inquiries
investors@allurion.com

RevBio Initiates its Pivotal Clinical Trial in Europe for its Dental Implant Stabilization Product




RevBio Initiates its Pivotal Clinical Trial in Europe for its Dental Implant Stabilization Product

The Company Received Approval in Multiple European Countries to Initiate this Key Study Necessary for Commercial Product Approval

LOWELL, Mass.–(BUSINESS WIRE)–#BoneRepair–RevBio, Inc., announced that it has received regulatory and ethics committee approvals in multiple European countries to conduct its pivotal clinical trial for its dental implant stabilization product. The successful completion of this pivotal clinical trial will result in the CE marking approval for the product, which will allow the company to begin commercial sales in Europe. As of the date of this press release, the company has already enrolled 30 of an expected 75 patients in this clinical trial.

“The results from this ongoing international multicenter study are exceeding expectations, showcasing remarkable outcomes. We are profoundly optimistic and incredibly enthusiastic about what the future holds,” said Prof. Dr. med. Dent., Patrick Schmidlin, head of the Division of Periodontology at the University of Zurich, who serves as one of the investigators in RevBio’s pivotal clinical trial. “This breakthrough material promises to open a new chapter in dental care, redefining what is possible in regenerative dentistry.”

RevBio received regulatory and ethics committee approvals for five clinical sites consisting of one in Switzerland, two in Belgium, one in Spain, and one in the United Kingdom. Each site is expected to enroll between 12-25 patients for a total of approximately 75 patients. The investigators who are approved to enroll patients in this clinical trial are Prof. Dr. med. Dent., PhD, Patrick Schmidlin, head of the Department of Periodontology at the University of Zurich, Ana Castro, BDS MSc PhD, Clinical Head Department Periodontology at the Catholic University of Leuven, France Lambert, DDS, PhD, Professor and Head of Periodontology, Oral Surgery, and Implant Surgery at the University of Liege, Arturo Llobell, DDS, MS, a periodontist in private practice in Valencia, Spain, and Azim Malik, BDS, MFDS RCSEd, DipPCD RCSI, DClinDent, MPerio RCSEd, a periodontist and implant specialist in private practice in London, United Kingdom.

When teeth are extracted due to damage from traumatic injuries, tooth decay, or gum disease, the current standard of care consists of multiple staged surgical procedures to restore a patient’s dentition with prosthetic crowns supported by dental implants. Frequently, extraction sites are too large for dental implants to achieve primary stability through conventional mechanical engagement. Instead, patients must undergo a costly, complex, and lengthy process including a preliminary bone grafting surgery before receiving a dental implant. The use of TETRANITE to stabilize an unstable implant will allow for the immediate placement of dental implants which otherwise could not be placed until the initial bone graft has healed to form new bone. As a result, the TETRANITE^® biomaterial will help reduce the duration and complexity of these dental implant procedures, lessen patient pain and recovery time, and reduce the overall cost of care thereby providing greater patient access for the treatment of tooth loss.

“The approval to conduct this pivotal clinical trial and the successful enrollment of the first 30 patients is a watershed moment for RevBio,” said Alan Pollack, DDS, RevBio’s Senior Director of Dental Clinical Operations. “This clinical trial has the potential to significantly accelerate the timeframe when this promising bone adhesive technology can be used in the field of dentistry.”

About RevBio, Inc.

RevBio, Inc., is a clinical stage medical device company engaged in the development and commercialization of TETRANITE^®, a patented, synthetic, injectable, self-setting, and osteoconductive bone adhesive biomaterial. The company is initially developing this technology for use in the dental, cranial, and broader orthopaedic markets as well as applications in the animal health market. RevBio’s TETRANITE technology is not yet approved for commercial use.

Contacts

Michael Tiedemann

mtiedemann@revbio.com

CEL-SCI Announces Pricing of $2.5 Million Offering




CEL-SCI Announces Pricing of $2.5 Million Offering

VIENNA, Va.–(BUSINESS WIRE)–$CVM–CEL-SCI Corporation (“CEL-SCI” or the “Company”) (NYSE American: CVM), a Phase 3 cancer immunotherapy company, today announced the pricing of a best-efforts offering of 16,000,000 shares of its common stock (or pre-funded warrants (“Pre-Funded Warrants”) in lieu thereof). Total gross proceeds from the offering, before deducting the placement agent’s fees and other offering expenses, are expected to be approximately $2,560,000. The offering is expected to close on March 18, 2025, subject to satisfaction of customary closing conditions.

The Company intends to use the net proceeds from the offering to fund the continued development of Multikine, general corporate purposes, and working capital.

ThinkEquity is acting as sole placement agent for the offering.

The securities will be offered and sold pursuant to a shelf registration statement on Form S-3 (File No. 333-265995), including a base prospectus, filed with the U.S. Securities and Exchange Commission (the “SEC”) on July 1, 2022, and declared effective on July 15, 2022. The offering will be made only by means of a written prospectus. A final prospectus supplement and accompanying prospectus describing the terms of the offering will be filed with the SEC on its website at www.sec.gov. Copies of the prospectus supplement and the accompanying prospectus relating to the offering may also be obtained, when available, from the offices of ThinkEquity, 17 State Street, 41^st Floor, New York, New York 10004.

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

About CEL-SCI Corporation

CEL-SCI believes that boosting a patient’s immune system before surgery, radiotherapy and chemotherapy have damaged it should provide the greatest possible impact on survival. Multikine is designed to help the immune system “target” the tumor at a time when the immune system is still relatively intact and thereby thought to be better able to mount an attack on the tumor.

Multikine (Leukocyte Interleukin, Injection), given right after diagnosis and before surgery, has been dosed in over 740 patients and received Orphan Drug designation from the FDA for neoadjuvant therapy in patients with squamous cell carcinoma (cancer) of the head and neck. Based on the data from the completed randomized controlled Phase 3 study, the FDA concurred with CEL-SCI’s target patient selection criteria and gave the go-ahead to conduct a confirmatory Registration Study. The study will enroll 212 newly diagnosed locally advanced primary treatment naïve resectable head and neck cancer patients with no lymph node involvement (determined via PET scan) and with low PD-L1 tumor expression (determined via biopsy), representing about 100,000 patients annually.

The Company has operations in Vienna, Virginia, and near/in Baltimore, Maryland.

Forward-Looking Statements

This press 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 Exchange Act of 1934, as amended. When used in this press release, the words “intends,” “believes,” “anticipated,” “plans” and “expects,” and similar expressions, are intended to identify forward-looking statements. Such statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected. Such statements include, but are not limited to, statements about the expected proceeds, use of proceeds and closing of the offering. Factors that could cause or contribute to such differences include an inability to duplicate the clinical results demonstrated in clinical studies, timely development of any potential products that can be shown to be safe and effective, receiving necessary regulatory approvals, difficulties in manufacturing any of the Company’s potential products, inability to raise the necessary capital and the risk factors set forth from time to time in CEL-SCI’s filings with the Securities and Exchange Commission, including but not limited to its report on Form 10-K for the year ended September 30, 2024. The Company undertakes no obligation to publicly release the result of any revision to these forward-looking statements which may be made to reflect the events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.

* Multikine (Leukocyte Interleukin, Injection) is the trademark that CEL-SCI has registered for this investigational therapy. This proprietary name is subject to FDA review in connection with the Company’s future anticipated regulatory submission for approval. Multikine has not been licensed or approved for sale, barter or exchange by the FDA or any other regulatory agency. Similarly, its safety or efficacy has not been established for any use.

Contacts

Gavin de Windt

CEL-SCI Corporation

(703) 506-9460