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May 24
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Amphista Therapeutics unveils new differentiated mechanism of action for BRD9 degradation at 2024 Protein Degradation in Focus Symposium

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

 

Amphista Therapeutics unveils new differentiated mechanism of action for BRD9 degradation at 2024 Protein Degradation in Focus Symposium

  • First presentation of a novel mechanism of action for the degradation of BRD9 – completely differentiated from cereblon- or VHL-based PROTACs
  • Amphista’s bifunctional BRD9 degraders selectively induce BRD9 to DCAF16 proximity and serve as molecular glues, leading to strong and rapid degradation of BRD9
  • Discovery further underscores Amphista’s proprietary chemistry and high-quality science which is being applied to the discovery and development of additional targeted glues beyond BRD9

Cambridge, UK, May 23, 2024 – Amphista Therapeutics (“Amphista”), a leader in next generation targeted protein degradation (TPD) approaches, today announces the unveiling of a new mechanism of action for the degradation of BRD9, an emerging target in oncology, that is differentiated from cereblon- or VHL-based PROTACs, during an oral presentation at the 2024 Protein Degradation in Focus Symposium held in Dundee, UK.

This significant news for the TPD field builds upon an earlier announcement by Amphista of two compelling data sets demonstrating in vivo efficacy and central nervous system (CNS) activity of its mechanistically differentiated protein degraders. At today’s presentation titled “Degradation of BRD9 by a novel targeted glue”, Dr Andrea Testa, Scientific Co-Founder and Senior Director, Discovery Chemistry showed data from proteomic and genetic validation studies which demonstrate that Amphista’s bifunctional degraders induced degradation of BRD9 by target-assisted E3 ligase recruitment. This novel mechanism selectively induces the proximity of BRD9 to DCAF16 and serves as a molecular glue. DCAF16 is a cullin-RING E3 ligase which can facilitate degradation of proteins of interest via ternary complex formation. Induction of this complex leads to deep and rapid degradation of BRD9 in vivo.

Louise Modis, Chief Scientific Officer of Amphista Therapeutics, said: “The discovery of a novel mechanism for BRD9 degradation, differentiated from cereblon- or VHL-based PROTACs, is a testament to Amphista’s proprietary chemistry and know-how. Our ability to translate this novel approach into high-quality, drug-like compounds with oral bioavailability and activity in vivo is truly exciting. As we continue to spearhead our efforts to advance new TPD approaches, our goal remains clear – to expand the offering of TPD medicines beyond CRBN and VHL-based agents and bring effective treatments to patients in areas of high unmet need.”

In the results presented, Amphista’s sub-nanomolar BRD9 degraders showed activity in both solid and liquid cancer cell lines and demonstrated a high degree of selectivity over 8,000 proteins quantified by proteomics, including the closely related proteins BRD7 and BRD4. Notably, these compounds induced BRD9 degradation in a mouse xenograft model illustrating Amphista’s BRD9 degraders are orally bioavailable and active in vivo. This is the first-time in vivo degradation of BRD9 has been demonstrated via DCAF16, underscoring the potential therapeutic application of Amphista’s bifunctional degraders.

Building on this knowledge, Amphista is applying its proprietary scientific know-how and expertise to the development of targeted glues which induce degradation of different targets, expanding the opportunity beyond BRD9. Future announcements will provide details of additional pipeline targets as part of Amphista’s ambitions to develop a first- and/or best-in-class portfolio of degraders with leading physicochemical properties.

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases, including cancer and neurodegenerative disease, through the advancement of next generation targeted protein degradation (TPD) medicines. Amphista is applying its proprietary warhead chemistry and mechanistic know-how to generate bifunctional targeted glues with a differentiated mechanism and leading physicochemical properties. Its portfolio offers the potential for first- and/or best-in-class assets and expanding the offering of TPD medicines beyond CRBN and VHL-based agents. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s laboratories at the University of Dundee. Amphista is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, Eli Lilly & Company and The Dementia Discovery Fund, and also has strategic collaborations with Bristol Myers Squibb and Merck. For more information, please visit: www.amphista.com

Amphista and the Amphista logo are all trademarks or registered trademarks of Amphista Therapeutics Limited.

 

For more information please contact:

ICR Consilium
Amber Fennell, Namrata Taak
Email: Amphista@consilium-comms.com
Tel: +44 (0)20 3709 5813

May 08
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Ventus Medical announces the submission of a novel electronic nicotine replacement therapy in the UK

By Press Release, Private Companies, Ventus Medical
Press Release.

 

A novel NRT for quitting smoking and vaping has been submitted to the UK medicines regulator for approval

LIVERPOOL, UNITED KINGDOM, May 7, 2024 /EINPresswire.com/ — Ventus Medical LTD announces that it has submitted a UK Marketing Authorisation Application for ENHALE™, a next-generation smoking cessation and harm reduction product which will give tobacco product users a safer alternative to both smoking and vaping.

ENHALE™ features a certified medical device and easily replaceable cartridges with novel and proprietary electronic inhalation technology. ENHALE™ meets the rigorous safety standards required of a medicinal product.
• ENHALE™’s proprietary formulation and low-temperature vaporisation technology ensure no thermal degradants and safer nicotine delivery than traditional vapes, an important differentiator from every other product.
• Obtaining UK MHRA approval is the first step in enabling healthcare professionals to prescribe for the first time, an effective vape-like product that is regulated and certified to be safe.
• When approved, ENHALE™ will be available without prescription in the UK.

Commenting on this submission, Kaasim Mahmood, Chairman of Ventus Medical and General Partner at Advent Life Sciences, said, “Smoking remains the most preventable cause of premature death globally. There is an urgent need for regulated, safer alternatives to vaping products and cigarettes. Advent Life Sciences is proud to have supported Ventus Medical, a UK company, on its journey to this important milestone. ENHALE™ has been designed and developed following extensive consultation with physicians, healthcare professionals and smokers who require a medicinal alternative to tobacco products.”

David Lawson, co-founder of Ventus Medical, added, “After nine years of rigorous research and development, we have successfully developed aerosolization technology to the strictest standards meeting pharmaceutical and medical device requirements globally. Clinical data on ENHALE™ confirm it’s ability to reduce the urge to smoke by more than leading NRT’s. This is a great step forward for smoking cessation and tobacco harm reduction and will be the first innovation in NRT for decades.”

ENHALE™ features an intuitive, easy-to-use, certified medical device with replaceable single-dose nicotine cartridges. The product is unique in utilizing low-temperature vaporisation technology, which heats the nicotine formulation to much lower temperatures when compared to vape products. Operating at lower temperatures ensures that ENHALE™ can safely and effectively deliver nicotine without any thermal degradation or metals which are of particular concern in vaping products.

About Ventus Medical
Ventus Medical is dedicated to the development of inhalation technology and is a certified medical device manufacturer based in the UK. The founding team of Ventus Medical were responsible for the first and only approved medical vape product (e-VOKE), which was licensed in 2013 by the MHRA.

David Lawson
Ventus Medical LTD
+44 333 344 0201
david.lawson@ventusmedical.com

Apr 15
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AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations

By AviadoBio, Press Release, Private Companies
Press Release.

 

AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations

• AVB-101 is an investigational gene therapy designed to deliver a functional copy of the GRN gene directly to the brain, thus restoring progranulin levels and potentially stopping disease progression

• Milestone marks first-in-human intrathalamic gene therapy delivery for any adult neurodegenerative disease

• ASPIRE-FTD trial will enroll patients at sites in Europe and the United States

LONDON — APRIL 15, 2024 — AviadoBio, a pioneering gene therapy company dedicated to developing and delivering potentially transformative medicines for neurodegenerative disorders, today announced that the first patient has been treated in the Phase 1/2 ASPIRE-FTD trial evaluating AviadoBio’s investigational gene therapy, AVB-101, in people with frontotemporal dementia (FTD) with progranulin (GRN) gene mutations (FTD-GRN). Clinical trial sites are currently open in Poland, Spain, and the Netherlands with additional sites expected to open in multiple countries, including the United States.

“AviadoBio is committed to bringing forward an innovative gene therapy treatment for FTD-GRN and this moment marks an important milestone for the FTD community and our company,” said Lisa Deschamps, CEO. “We are thankful for the dedication of the ASPIRE-FTD clinical investigators studying AVB-101 and immensely grateful for the families who participate in clinical trials for new treatment options that may change the future for generations of families living with FTD-GRN.”

FTD is a devastating form of early-onset dementia that typically leads to death within three to 10 years from diagnosis.¹,² People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.³ It is a leading cause of dementia in people under the age of 65 and is often misdiagnosed.⁴People with FTD who have disease-causing GRN mutations produce a reduced amount of progranulin protein. AVB-101 is a potential one-time therapy designed to stop disease progression by delivering a functional copy of the GRN gene to restore appropriate progranulin levels to affected areas of the brain.

“There is a critical need for new treatments for people living with FTD-GRN. The lack of effective disease-modifying treatments makes this diagnosis particularly difficult for patients and their families who don’t currently have any available treatment options for this devastating and progressive disease,” said David Cooper, M.D., Chief Medical Officer. “Treating the first patient in ASPIRE-FTD marks an important step forward in understanding AVB-101’s potential, which has already shown promise in preclinical studies.”

AVB-101 is delivered using a minimally invasive, stereotactic neurosurgical procedure directly to the part of the brain called the thalamus. The thalamus has extensive connections to other parts of the brain, including the frontal and temporal lobes, which play a critical role in FTD and the symptoms that impact patients and their families. This targeted delivery method aims to safely and effectively cross the blood-brain barrier, delivering targeted treatment directly to the brain to restore progranulin levels in the frontal and temporal cortex where it is needed most, while at the same time minimizing the dose required and thereby limiting any potential systemic exposure.

The Interventional Neurotherapy Center (INC) at Mazowiecki Szpital Bródnowski Hospital is the first and only center in Europe currently performing MRI-guided infusions of gene therapies. “Our team is excited to participate in this important scientific research and to perform the first intrathalamic administration of AVB-101 in the ASPIRE-FTD global trial,” said Prof. Mirosław Ząbek, M.D., Ph.D., the chairperson of the department of neurosurgery at INC.

“As a neurologist and founder of the Neuro-Care Clinic, I’ve seen the difficult struggles of my patients with dementia and their families,” said Gabriela Klodowska, M.D., Ph.D. “It means a lot to our patients with FTD to bring this type of study offering a potential one-time treatment for patients with a GRN mutation to Poland and to be the first to enroll patients in this global study.”

In November 2023, the U.S. Food and Drug Administration (FDA) granted Fast Track designation to AVB-101. Fast Track is a process designed to facilitate the development and expedite the review of new drugs to treat serious conditions and fill an unmet medical need. This designation is intended to bring new medicines to patients earlier. It has also received orphan drug designation by both the FDA and European Commission.

More information about the ASPIRE-FTD study can be found at https://clinicaltrials.gov/study/NCT06064890.

  1. Onyike CU. Neuroepidemiology. 2011;37:166–167
  2. Riedl L et al. Neuropsychiatr Dis Treat. 2014;10:297–310
  3. Pressman P and Miller BL. Biol Psychiatry. 2014;75(7):574–581
  4. Hendriks S, Peetoom K, Bakker C, et al. Global Prevalence of Young-Onset Dementia: A Systematic Review and Meta-analysis. JAMA Neurol. 2021;78(9):1080–1090. doi:10.1001/jamaneurol.2021.216
  5. Hogan DB et al. Can J Neurol Sci. 2016;43 (Suppl 1):S96–S109
  6. Moore KM et al. Lancet Neurol. 19(2):145–156
  7. Kansal K et al. Dement Geriatr Cogn Disord. 2016;41:109–122
  8. Galvin JE et al. Neurology. 89(20):2049–2056
  9. Young JJ et al. Ther Adv Psychopharmacol. 2018;8(1):33–48
  10. Kuang, L., et. al. Frontotemporal dementia non-sense mutation of progranulin rescued by aminoglycosides. Human Molecular Genetics 2020;29(4):624-634

About ASPIRE-FTD

ASPIRE-FTD is an open-label, multi-center, Phase 1/2 dose-escalation study designed to evaluate the safety and preliminary efficacy of AVB-101 in patients with FTD-GRN. In the study, patients will receive a single administration of AVB-101 delivered as a one-time infusion into the thalamus via a stereotactic neurosurgical procedure at expert neurosurgical centers throughout Europe and the United States.

About AVB-101

An investigational gene therapy, AVB-101 contains a correct (non-mutated) version of the GRN gene. It is designed to restore levels of progranulin in the brain, potentially slowing or stopping the progression of FTD-GRN. AVB-101 will be delivered as a one-time infusion directly into the brain via a minimally invasive surgical procedure, performed by a study neurosurgeon at a specialist neurosurgical center.

About Frontotemporal Dementia (FTD) and FTD with GRN Mutations (FTD-GRN

FTD is a devastating form of early-onset dementia that typically leads to death within seven to 13 years of symptom onset and three to 10 years from diagnosis.¹,²  People with FTD commonly experience personality changes, behavioral disturbance, loss of language, apathy, and reduced mobility.³

FTD is a leading cause of dementia in people under the age of 65⁴ with an estimated prevalence at any one time of up to 4.6 cases per 1,000 people.⁵ FTD typically strikes younger than Alzheimer’s disease and the majority of FTD cases occur between 45 and 68 years of age.⁶,⁷ Given the early onset, FTD can have a substantially greater impact on work, family, and finances than Alzheimer’s disease.⁸ Genetic FTD cases account for about one-third of cases and are associated with autosomal dominant mutations in three genes, including the GRN (progranulin) gene.⁹ Approximately 11,000 people in the U.S. and EU5 are living with FTD-GRN with approximately 2,200 new cases per year.¹,¹⁰ Some FTD cases may be misidentified, and diagnostic delay is common. As disease education, genetics knowledge, and research and treatment options grow, these numbers are expected to increase.

About AviadoBio

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

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

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

Mar 13
Love0

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

By Eloxx Pharmaceuticals, Press Release, Private Companies
Press Release.

 

Almirall and Eloxx Pharmaceuticals Enter into Exclusive Agreement to license ZKN-013 for rare dermatological diseases

  • Almirall obtains exclusive global rights to develop and commercialize ZKN-013 for the treatment of rare dermatological and other diseases associated with nonsense mutations
  • ZKN-013 is a phase I ready oral therapy designed to overcome nonsense mutations that cause a premature stop codon resulting in nonfunctional protein production for example in recessive Dystrophic Epidermolysis Bullosa (RDEB), Junctional Epidermolysis Bullosa (JEB) and familial adenomatous polyposis (FAP)

BARCELONA, Spain and WATERTOWN, Mass., March 13, 2024 (GLOBE NEWSWIRE) — Almirall, S.A. (BME:ALM) and Eloxx Pharmaceuticals, Inc. (OTC: ELOX) announced today that the companies have entered into an exclusive license agreement for the asset ZKN-013. Under the agreement, Almirall obtains global rights to develop and commercialize ZKN-013, including for the use in orphan dermatological diseases. ZKN-013 is a potentially promising oral, nonsense mutation readthrough drug, which enables the host cells to produce functional proteins which counteract the root cause of these rare dermatological and potentially other diseases.

The drug candidate is expected to shortly enter into Phase I development in healthy volunteers.

As part of this agreement, Eloxx will receive an upfront of $3 million, and additional payments throughout the potential development phases, including regulatory and sales milestones of up to $470 million, as well as tiered royalties based on any potential future global sales.

“We are very excited about this agreement with Almirall to develop and distribute ZKN-013, our lead TURBO-ZM™ based molecule, as we believe it has the potential to have a significant impact on the treatment of these painful and debilitating diseases,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “In addition to advancing development of ZKN-013, this agreement will allow Eloxx to remain focused on fully maximizing the potential of ELX-02 in rare kidney diseases and continue funded discovery efforts on our TURBO-ZM™ platform.”

“This license agreement is aligned with Almirall’s R&D strategy to develop novel treatments to help people with dermatological conditions, including rare diseases,” said Karl Ziegelbauer, Executive Vice President R&D and Chief Scientific Officer of Almirall. “We look forward to progressing the development of ZKN-013 to find a potentially impactful solution for patients suffering from rare and devastating diseases caused by nonsense mutations.”

RDEB /JEB are rare skin diseases characterized by defects in the Collagen7 gene which is essential for the correct formation of the skin structure and barrier function. ZKN-013 has demonstrated robust functional preclinical activity in RDEB/JEB patient cells and in APCmin (multiple intestinal neoplasia) mice. The studies demonstrated that ZKN-013 induces the production of functional, full-length COL7 in RDEB patient cells.

ZKN-013 is also being developed for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and progression to colon cancer. FAP is a rare GI disease with patients progressing to colon cancer caused by mutations in the APC gene.

ZKN-013 is Eloxx’s lead TURBO-ZM based molecule. Eloxx’s TURBO-ZM platform uses chemistry technology to develop novel Ribosome Modulating Agents to target the human ribosome to develop new potential therapeutics. Ribosomes form the translation machinery that generates functional proteins from genetic sequences; modulating the ribosome subunits provides a therapeutic approach to address a number of different diseases.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZM™ chemistry technology platform in an effort to develop novel Ribosome Modulating Agents (RMAs) and its library of Eukaryotic Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in Phase 2 clinical development for the treatment of Alport syndrome in patients with nonsense mutations.

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

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). Almirall (total revenue in 2023: €898.8 MM, 1900 employees globally) has a direct presence in 21 countries and affiliates in over 70 others.

For more information, please visit almirall.com

Corporate Communications contact Almirall: Investors’ Relations contact
Corporate communications team Pablo Divasson del Fraile
corporate.communication@almirall.com investors@almirall.com
Phone: (+34) 659 614 173 Phone: (+34) 93 291 30 87
Eloxx Contacts:
Investors:
John Woolford
john.woolford@westwicke.com
443.213.0506
Media:
Laureen Cassidy
laureen@outcomescg.com

Eloxx Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this press release, including without limitation, statements regarding the potential future payments and future benefits under the license agreement, achievement of key milestones under the license agreement and the expected timeline for clinical development and efficacy of ZKN-013 are forward-looking statements. Forward-looking statements can be identified by the words “aim,” “may,” “will,” “would,” “should,” “expect,” “explore,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplate,” “believe,” “estimate,” “predict,” “potential,” “seeks,” or “continue” or the negative of these terms similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections based on information currently available to us. Forward-looking statements are subject to known and unknown risks, uncertainties and assumptions, and actual results or outcomes may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to: our ability, or our licensees’ ability, to progress any product candidates in preclinical or clinical trials; the uncertainty of clinical trial results and the fact that positive results from preclinical studies are not always indicative of positive clinical results; the scope, rate and progress of our, and our licensees’ preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;; our ability to obtain the capital necessary to fund our operations; the cost of filing, prosecuting, defending and enforcing any patent claims and other intellectual property rights; our ability to obtain financing in the future through product licensing, public or private equity or debt financing or otherwise; our ability to meet the continued listing requirements of the Nasdaq Capital Market; general business conditions, regulatory environment, competition and market for our products; and business ability and judgment of personnel, and the availability of qualified personnel and other important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website at www.sec.gov and the “Financials & Filings” page of our website at https://investors.eloxxpharma.com/financials-filings.

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Almirall Legal warning
This document includes only summary information and is not intended to be exhaustive. The facts, figures and opinions contained in this document, in addition to the historical ones, are “forward-looking statements”. These statements are based on the information currently available and the best estimates and assumptions that the Company considers reasonable. These statements involve risks and uncertainties beyond the control of the Company. Therefore, actual results may differ materially from those declared by such forward-looking statements. The Company expressly waives any obligation to revise or update any forward-looking statements, goals or estimates contained in this document to reflect any changes in the assumptions, events or circumstances on which such forward-looking statements are based, unless required by the applicable law.

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Feb 28
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Pulsed Field Ablation Innovator Argá Medtech Announces €54 Million Oversubscribed Series B Capital Raise

By Argá Medtech, Press Release, Private Companies
Press Release.

 

Pulsed Field Ablation Innovator Argá Medtech Announces €54 Million Oversubscribed Series B Capital Raise

– Funding will advance US and EU clinical studies of innovative pulsed field ablation system for treating atrial fibrillation  – 

Lausanne, Switzerland and San Diego, CA, USA — February 27, 2024 — Argá Medtech, a private company developing Coherent Sine-Burst Electroporation™ (CSE™), a next-generation cardiac ablation system for treating cardiac arrhythmias, including atrial fibrillation (AF), today announced the closure of a €54M oversubscribed Series B funding. The round was led by the existing investors, Advent Life Sciences (UK) and Earlybird Health (Germany), as well as new investor Gilde Healthcare (the Netherlands) and an undisclosed strategic investor. The financing enables Argá Medtech to advance the development of its innovative CSE™ Pulsed Field Ablation (PFA) system for treating AF through the execution of an IDE study in the US and a CE Mark study in the EU. With the funding, the company will also expand its US offices in San Diego, CA, in anticipation of its US clinical activities.

“We are pleased to secure the support of such marquee investors who believe that Argá Medtech will revolutionize the atrial fibrillation ablation field,” said David Neale, CEO of Argá Medtech. “This financing enables us to advance toward our goal of validating the CSE™ PFA system in Europe and the US as we work to deliver a safe, fast, and effective treatment to millions of people affected by cardiac rhythm disorders and atrial fibrillation. We are proud of our accomplishments to date, including conducting a 48-patient first-in-human study in Europe, which demonstrated the high performance of our platform in treatingatrial fibrillation.”

Argá Medtech’s CSE™ ablation system enables electrophysiologists to treat any region in the heart safely and efficiently using a single, multi-configurable catheter while titrating lesion depths according to the location within the heart.

The company’s CSE™ PFA platform combines its proprietary CSE™ PFAgenerator with a multi-configurable catheter to provide unmatched flexibility in AF treatment. Argá Medtech’s CSE™ system uses a sinusoidal/sine wave, while other PFA platforms are typically powered by square wave energy sources. This approach offers several advantages, such as allowing physicians to configure the energy delivery and titrate the depth to the location in the heart. Additionally, the waveform is delivered through a versatile, all-in-one catheter that can be shaped to create circular, linear, or focal ablation lesions, eliminating the need to perform catheter exchanges to achieve the desired lesion set. Thus, the procedure is simplified, reducing the risk of introducing air bubbles as one catheter is removed and another is reinserted, as well as reducing costs.

AF is the most common heart arrhythmia, affecting about 38 million patients worldwide1, rendering them five times more likely to have a stroke.2 While ablation using thermal energy has proven effective in alleviating AF symptoms, higher risks are associated with its use.3In contrast, PFA energy delivery offers a tissue-selective, precise ablation of the intended heart tissue while preserving nearby tissues and minimizing the risks of thermal injury, such as those to the esophagus or the phrenic nerve.

About Argá Medtech SA

Argá Medtech SA is developing a Coherent Sine-Burst Electroporation™ (CSE™), Pulsed Field Ablation (PFA) system to treat patients with cardiac arrhythmias. Argá Medtech is setting the standard for the next-generation PFA systems with its versatile catheter design able to create circular, linear, and focal lesions, coupled with a unique CSE™ PFA waveform that allows energy titration based on tissue depth to achieve durable lesions. The company was co-founded in June 2020 by veteran medical device executive David Neale, CEO, and serial entrepreneur Randy Werneth, CTO, and has offices in Lausanne, Switzerland and San Diego, CA, USA. With over 50 years of combined experience in creating and commercializing cardiac and arrhythmia management products, the leadership team is well connected in electrophysiology and understands the current challenges faced by physicians and patients.

To learn more, visit https://argamedtech.com.

References: 

1. Lippi, Giuseppe et al. “Global epidemiology of atrial fibrillation: An increasing epidemic and public health challenge.” International journal of stroke: official journal of the International Stroke Society vol. 16,2 (2021): 217-221. doi:10.1177/1747493019897870. https://pubmed.ncbi.nlm.nih.gov/31955707/

2. World Stroke Organization. Stroke and Atrial Fibrillation. Accessed February 16, 2024.  https://www.world-stroke.org/world-stroke-day-campaign/prevent-stroke/stroke-and-atrial-fibrillation

3. Aldaas OM, Malladi C, Aldaas AM, Han FT, Hoffmayer KS, Krummen D, Ho G, Raissi F, Birgersdotter-Green U, Feld GK, Hsu JC. Safety and acute efficacy of catheter ablation for atrial fibrillation with pulsed field ablation vs thermal energy ablation: A meta-analysis of single proportions. Heart Rhythm O2. 2023 Sep 12;4(10):599-608. doi: 10.1016/j.hroo.2023.09.003. PMID: 37936671; PMCID: PMC10626185. https://pubmed.ncbi.nlm.nih.gov/37936671/

Media Contact: 

Finn Partners

Glenn Silver

glenn.silver@finnpartners.com

Feb 27
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Curve Therapeutics raises £40.5 million to turbocharge discovery platform and advance breakthrough pipeline

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

 

Curve Therapeutics raises £40.5 million to turbocharge discovery platform and advance breakthrough pipeline

  • Series A financing led by Pfizer Ventures with new investors Columbus Venture Partners and British Patient Capital which join founding investor Advent Life Sciences and seed investor Epidarex Capital
  • Goal to advance proprietary discovery platform and progress pipeline of assets addressing challenging cancer targets into the clinic

Southampton, UK, 27 February 2023 – Curve Therapeutics (“Curve” or the “Company”), a private biotechnology company pioneering a revolutionary intracellular screening platform addressing complex and challenging disease targets, today announces the close of its successful £40.5 million Series A financing. Pfizer Ventures led the round with participation from Columbus Venture Partners and British Patient Capital, which join founding investor Advent Life Sciences and co-lead from the seed round, Epidarex Capital.

Curve’s powerful Microcycle® platform enables the direct discovery of biologically active molecules against targets that have been difficult to address using conventional drug discovery methods. Curve has built a discovery pipeline of assets including a first-in-class dual-inhibitor of HIF-1 and HIF-2 that addresses survival mechanisms in more than half of solid tumours, and a first-in-class inhibitor of ATIC dimerization that targets an important vulnerability in multiple cancers. The financing will enable the Company to progress development of these assets rapidly towards clinical development and to expand the discovery platform beyond challenging and complex intracellular protein targets.

Simon Kerry, PhD, MBA, Chief Executive Officer of Curve Therapeutics, said: “This financing will enable us to expand our team, progress our lead assets into the clinic and to expand our drug discovery platform. We welcome our new investors alongside our existing strong syndicate and look forward to working together to take Curve to its next stage of growth.”

Professor Ali Tavassoli, Chief Scientific Officer of Curve Therapeutics, added: “Curve’s Microcycle platform is a powerful tool for drug discovery, enabling an unparalleled advantage in the discovery of functional hits and leads. We look forward to maximising the potential of our platform to further develop a rich pipeline of programmes with the potential to treat unmet clinical needs in a diverse range of diseases, including cancer.”

Dr. Marie-Claire Peakman, Partner at Pfizer Ventures, commented: “We are delighted to have led this financing and to work with great co-investors and leadership team.  Curve’s platform provides an exciting new approach designed to tackle tough, high priority targets which have been difficult to progress in the past. We are eager for the Company to progress the platform with the goal of identifying new therapeutics for challenging diseases.” 

Curve’s platform allows functional screening and enrichment of highly diverse, gene-encoded, Microcycle libraries within the cytoplasm of mammalian cells to identify library members that have a desired biological activity against a therapeutic target. Importantly, the compact size of Microcycles enables their transformation to non-peptide small molecules for lead optimisation and development, an unparalleled advantage compared to other cyclic peptide technologies.

Curve occupies state of the art laboratories at the Southampton Science Park, a move prompted by the expansion of the Company’s research team which included the recent appointments of Dr Monika Ermann as Vice President of Drug Discovery, and Dr Sally Price as Vice President of Biology. With four regional Universities and pioneering medical institutions such as the Centre for Cancer Immunology in Southampton, Hampshire is an attractive location for life science companies, and just an hour from key biotech centres in Oxford and London.

For more information, please contact:

Curve Therapeutics
Simon Kerry
Chief Executive Officer
info@curvetx.com

Optimum Strategic Communications
Mary Clark, Vici Rabbetts, Joshua Evans
+44 (0) 208 078 4357
curve@optimumcomms.com

About Curve Therapeutics

Curve Therapeutics is a private biotechnology company pioneering a revolutionary intracellular screening platform to enable the discovery of innovative therapeutics that address complex and challenging disease targets with the potential to transform the lives of patients. Curve originated from world-leading Microcycle® research conducted by Professor Tavassoli’s group in the Department of Chemistry at the University of Southampton, UK. Curve is backed by blue chip investors including Advent Life Sciences, Epidarex Capital, Pfizer Ventures, Columbus Venture Partners and British Patient Capital. Curve has a US$1.7bn global research collaboration with MSD the trade name of Merck & Co., Inc., Kenilworth, NJ USA, to discover and validate modulators of up to five therapeutic targets using its Microcycle® technology, initially for oncology and neurology indications. For more information visit: www.curvetx.com

About Curve’s Microcycle® platform

Curve has developed an IP-protected, mammalian cell platform technology for functional screening and enrichment of diverse hexameric cyclic peptide Microcycle® libraries to identify those library members that have the desired biological activity against a therapeutic target. Curve’s platform allows direct screening for biologically active library members inside mammalian cells and facilitates small molecule hit-to-lead programmes. A key advantage of the technology is that both the library and the target are present in all of their native conformations within a cell. Uniquely, the compact size and rigid structure of Microcycles® enables the design of non-peptide small molecule leads. The platform can be used for a wide range of therapeutically relevant targets, including protein-protein and protein-DNA interactions and has been used by Curve to develop a pipeline of cancer programmes against targets including a dual HIF-1/HIF-2 inhibitor and an inhibitor of ATIC homodimerization.

Feb 21
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Aleta Biotherapeutics and Cancer Research UK’s Centre for Drug Development Announce First Patient Dosed in ALETA-001 Phase 1/2 Clinical Trial

By Aleta Biotherapeutics, Press Release, Private Companies
Press Release.

 

Aleta Biotherapeutics and Cancer Research UK’s Centre for Drug
Development Announce First Patient Dosed in ALETA-001 Phase 1/2 Clinical
Trial in Patients with Relapsed/Refractory B-Cell Malignancies

 

  •  The Phase 1/2 clinical trial will evaluate safety, dose, PK/PD, and early efficacy
    signals of Aleta’s first-in-class biologic CAR T-Cell Engager, ALETA-001
  • ALETA-001 entering the clinic is a critical milestone toward treatment availability to
    improve CAR T-cell patient response when a complete response is not achieved or
    maintained

NATICK, Mass., and LONDON, UK, February 21, 2024 – Aleta Biotherapeutics (Aleta),
a clinical stage, immuno-oncology company with a CAR T-Cell Engager (CTE) platform
that enables cell cancer therapies to work more effectively, and Cancer Research UK’s
Centre for Drug Development, today announce the first patient was dosed in a Phase 1/2
clinical trial. This trial is evaluating the Company’s first-in-class biologic CAR T-Cell
Engager, ALETA-001, for the treatment of patients with B-cell malignancies who are
relapsed/refractory to CD19-targeting CAR T-cell therapy.

Cancer Research UK’s Centre for Drug Development is sponsoring and conducting the Phase
1/2 clinical trial of ALETA-001, the lead agent in Aleta’s portfolio.

“It is very exciting and meaningful to have ALETA-001 now in the clinic. Clinical
evaluation of ALETA-001 is a key milestone toward a much-needed treatment option for the
many cancer patients whose CD19-targeted CAR T-cell therapies ultimately stop working.
ALETA-001 restores and increases the effectiveness with which CAR T-cells can kill cancer
cells, and we believe that it will enable more patients to successfully benefit from cell
therapies,” stated Dr. Paul Rennert, President and Chief Scientific Officer, Aleta
Biotherapeutics.

The ALETA-001 Phase 1/2 clinical trial is an open-label, dose-expansion trial which will
evaluate safety and tolerability, pharmacokinetic and pharmacodynamic effects, and early
signals of clinical efficacy of ALETA-001 as a single agent. Chief Investigator Dr. Sridhar
Chaganti is leading the trial from University Hospital Birmingham NHS Foundation Trust in
Birmingham, UK, and will enroll patients who are relapsed/refractory following treatment
with available CD19-targeting CAR T-cell therapies. For more information on this trial,
please visit clinicaltrials.gov (NCT06045910)

Dr. Lars Erwig, Cancer Research UK’s Director of Drug Development, said, “A
significant number of patients with blood cancers unfortunately relapse following CD19-
directed CAR T-cell therapy. ALETA-001 is being developed to provide patients with these
cancers a better chance for a successful treatment outcome. At Cancer Research UK, we are
very excited to study the clinical potential of ALETA-001 to transform a patient’s treatment
journey – which can possibly be lifesaving in many cases.”

“Aleta-001 is a uniquely designed new molecule with a novel mechanism of action, coating
tumor cells densely with the target antigen, thereby stimulating the tumor killing ability of
activated CAR T-cells,” commented Dr. Sridhar Chaganti, Chief Investigator, University
Hospital Birmingham NHS Foundation Trust, Birmingham, UK. “This is an important
new strategy being investigated to improve outcomes for patients with relapsed or refractory
lymphomas who experience disease progression after CAR T-cell therapy and have a poor
prognosis.”

About CAR T-Cell Therapy Engager (CTE) ALETA-001
Aleta’s lead clinical stage program, ALETA-001, was designed specifically for the treatment
of B-cell malignancies in patients who have received a CD19-directed CAR T-cell therapy
and are at risk of treatment failure. Developed to improve the effectiveness of CD19-directed
CAR T-cell therapies by increasing CD19 antigen density and restoring lost CD19
expression on the cancer cell, ALETA-001 contains the CD19 target protein which is further
linked to an CD20 antibody domain. This allows CD19+/CD20+ cancer cells to be easily
recognized and killed by CD19-directed CAR T-cells that were previously administered and
are already circulating within a patient.

Aleta previously secured landmark clinical support and funding from Cancer Research UK
for the ALETA-001 Phase 1/2 clinical trials, and ALETA-001 has received a UK Innovation
Passport under the Innovative Licensing and Access Pathway (ILAP) from the U.K.
Medicines and Healthcare products Regulatory Agency (MHRA). ILAP designation is
granted to medicines that address life-threatening or seriously debilitating conditions, and
where there exists a significant patient or public health need.

About Aleta Biotherapeutics
Aleta Biotherapeutics is pioneering a transformation of cancer treatment by enabling CAR Tcell
cancer therapies to work more effectively. The Company’s unique portfolio of multiantigen
CAR T Engagers (CTEs) are simple, potent biologic therapies designed to transform
the expression of cancer tumor cells to match CAR T-cell therapies circulating in patient
blood. These CTEs are being developed to produce deeper and more durable responses in
patients receiving CAR T-cell therapies through increasing cancer target density, and
preventing resistance and escape from therapy, thus increasing the speed and effectiveness
with which CAR T-cells can kill cancer cells.

Aleta’s lead clinical stage biologic, ALETA-001 was designed specifically for the treatment
of B-cell malignancies in patients who have received a CD19-directed CAR T-cell therapy
and are at risk of treatment failure. Aleta is further developing its CTE platform technology
to address acute myeloid leukemia, multiple myeloma, and solid tumors including breast
cancer, gastric cancer, and pediatric brain cancers. aletabio.com

About Cancer Research UK’s Centre for Drug Development
Cancer Research UK has an impressive record of developing novel treatments for cancer.
The Cancer Research UK Centre for Drug Development has been pioneering the
development of new cancer treatments for 30 years, taking over 160 potential new anticancer
agents into clinical trials in patients. It currently has a portfolio of over 20 new anticancer
agents in preclinical development, Phase 1, or early Phase 2a clinical trials. Six of
these new agents have made it to market including temozolomide for brain cancer,
abiraterone for prostate cancer and rucaparib for ovarian cancer. 13 other drugs are still
undergoing active development. www.cruk.org.uk/cdd

Contacts:
Media:
Linda Phelan Dyson, MPH
Corporate Communications
+1 973-986-5973
lpdyson@verizon.net

Jan 24
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Amphista Therapeutics announces new data demonstrating in vivo efficacy and CNS activity of its mechanistically differentiated targeted protein degraders

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

 

Amphista Therapeutics announces new data demonstrating in vivo efficacy and CNS activity of its mechanistically differentiated targeted protein degraders

 

  • First demonstration of efficacy in disease-relevant in vivo models for an orally delivered bifunctional non-cereblon / non-VHL-based protein degrader
  • Deep degradation of target protein is achieved in a rapid, sustained, and highly selective manner
  • Successful achievement of efficient CNS delivery for multiple degraders and demonstration of significant degradation of target protein in the brain

Cambridge, UK, January 24th, 2024 – Amphista Therapeutics (“the Company” or “Amphista”), a leader in next generation targeted protein degradation (TPD) approaches, today announces the achievement of two compelling new data sets with its next generation bifunctional degraders demonstrating in vivo efficacy and the ability to target and degrade proteins in the central nervous system (CNS).

Amphista’s degraders, which leverage a novel protein degrading mechanism, with advantages beyond first generation cereblon or VHL-based approaches, have demonstrated:

–  Sustained degradation of target protein and anti-tumor efficacy: once daily oral dosing led to statistically significant reduction in tumor burden and survival advantage in multiple in vivo disease models.

–  Rapid degradation of target protein: significant levels of degradation were induced rapidly after dosing.

– Highly selective degradation of target protein: statistically significant degradation of target protein vs >8000 other proteins, including closely related homologs, when dosed at 100x DC50.

Amphista also discloses significant progress in the advancement of its technology for the treatment of neurodegenerative diseases, a key focus of the Company. These data show:

  • Amphista degraders can be rationally designed to achieve CNS penetrance with examples achieved across multiple targets.
  • Rapid degradation of target protein achieved in the brain in vivo (dog and non-human primate, and in multiple brain regions) when dosed intravenously.

Louise Modis, Chief Scientific Officer of Amphista Therapeutics, said: “We are delighted by the progress we have made in advancing our pipeline and demonstrating the potential of our unique TPD technology. To our knowledge, this is the first time an orally delivered bifunctional non-cereblon / non-VHL-based protein degrader has shown efficacy in disease-relevant in vivo models. Combined with our progress in demonstrating in vivo degradation of targets within the CNS, we are extremely excited to have achieved these key preclinical development milestones and we look forward to providing additional updates across our pipeline throughout

the year.”

Beverley Carr, Interim Chief Executive Officer of Amphista Therapeutics, said: “The last six months have been

transformative for Amphista and these new compelling in vivo data strengthen our belief that our technology has the potential to deliver differentiated protein degrader molecules with class-leading physicochemical properties, enabling us to target a wider tissue and indication scope than traditional protein degrader approaches. These data are testament to the strength of our scientific team and we are on track with our portfolio priorities, exploring the tremendous potential of our platform across multiple indications in CNS and

oncology.”

About Amphista Therapeutics

Amphista Therapeutics is focused on transforming the lives of patients with severe diseases including cancer. The company is applying its proprietary Amphista degrader platform to advance new approaches in targeted protein degradation (TPD). They aim to address the challenges faced by earlier stage TPD research and to realise the full therapeutic potential of this transformational approach. Founded by Advent Life Sciences, Amphista is a spin-out of TPD expert Professor Alessio Ciulli’s laboratories at the University of Dundee. The Company has raised over $60M to date and is funded by leading life science investors including Forbion, Gilde Healthcare, Novartis Venture Fund, Advent Life Sciences, Eli Lilly & Company and The Dementia Discovery Fund. For more information, please visit: www.amphista.com

Amphista and the Amphista logo are all trademarks of Amphista Therapeutics Limited.

For more information please contact:

Amphista Therapeutics

Beverley Carr, Interim CEO info@amphista.com

 

ICR Consilium

Amber Fennell, Namrata Taak

Email: Amphista@consilium-comms.com

Tel: +44 (0)20 3709 5813

Jan 18
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Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease

By Orphalan, Press Release, Private Companies
Press Release.

 

Orphalan announces China NMPA’s approval of its trientine tetrahydrochloride product for the treatment of Wilson disease

Paris, France 18 January, 2024 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the approval of its trientine tetrahydrochloride product by China’s National Medical Products Administration (NMPA). This product, already marketed as Cuprior® in the EU, the UK, Saudi Arabia, Switzerland, and Colombia, and as Cuvrior® in the US, represents the first trientine approved by the NMPA. It offers a new treatment option for Wilson disease in children aged five years and older and adults intolerant to penicillamine therapy.
China is home to an estimated 80,000 individuals with Wilson disease, of whom approximately half have their diagnosis confirmed, and 25,000 are receiving regular treatment. The introduction of new treatment alternatives such as Orphalan’s trientine tetrahydrochloride, particularly for patients who are intolerant to currently available therapies, presents a significant opportunity to meet the needs of a broader segment of the Wilson disease population in China. Orphalan anticipates its product to be available in the country in the coming months.

Dr Naseem Amin, Chief Executive Officer at Orphalan, said: “We are pleased with the marketing authorisation approval of our trientine tetrahydrochloride product in China, which offers a well-tolerated and effective treatment option for patients with Wilson disease. This approval is a crucial step towards providing a much-needed treatment option for the Wilson disease community in China and reaffirms our commitment to improving the experience for patients living with rare diseases worldwide.”

Yan Qin, Director of Wuhan Tongxin (Wilson disease patient group in China) added: “We were thrilled when we heard that NMPA approved Orphalan’s trientine tetrahydrochloride in China. It is a significant step for those of us living with Wilson disease, representing a much-needed alternative treatment option. This approval, a result of Orphalan’s patient-focused approach, offers us a chance to better manage our condition with a new, effective treatment.”

Wilson disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Early treatment with legacy agents such as D-penicillamine (DPA), trientine HCI, and zinc salts can effectively slow the progression of the disease, however these options may not be suitable for long-term care due to adverse events associated with DPA and the need for close monitoring during treatment with zinc salts and DPA.

About Orphalan
Orphalan is an international orphan drug development and commercialization company headquartered in Paris. Founded in 2011, the Company develops and deliver innovative therapies for people living with rare and debilitating diseases. Our trientine tetrahydrochloride product has been approved for the treatment of Wilson disease and is available in more than 20 countries, branded as Cuprior® in EU, the UK, Saudi Arabia, Switzerland and Columbia, and as Cuvrior® in the United States. For more information visit www.orphalan.com and follow us on LinkedIn.

For more information, please contact:
Orphalan:
Géraldine van den Broek, Head of Corporate & BD
Tel: +33 (0)1 42 49 82 64
info@orphalan.com
ICR Consilium:
Tracy Cheung, Sukaina Virji, Davide Salvi
Tel: +44 (0) 203 709 5700
orphalan@consilium-comms.com

Jan 09
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Bayer’s elinzanetant meets all primary and key secondary endpoints in pivotal OASIS 1 and 2 Phase III studies

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

 

Bayer’s elinzanetant meets all primary and key secondary endpoints in pivotal OASIS 1 and 2 Phase III studies

 

OASIS 1 and 2 pivotal studies evaluating investigational compound elinzanetant met all primary endpoints demonstrating a statistically significant reduction in frequency and severity of moderate to severe vasomotor symptoms (VMS) compared to placebo in postmenopausal women / Both studies also showed superiority over placebo for all key secondary endpoints with a statistically significant reduction in frequency of VMS at week 1, as well as improvement of sleep disturbances and menopause-related quality of life / The safety profile observed in both studies is overall consistent with previously published data on elinzanetant / OASIS 1 and 2 are two of three Phase III clinical studies investigating the efficacy and safety of elinzanetant, a first dual neurokinin-1,3 (NK-1,3) receptor antagonist, as a non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily

Berlin, January 8, 2024 – Bayer today announced positive top-line results of the pivotal Phase III studies OASIS 1 and 2 evaluating the efficacy and safety of the investigational compound elinzanetant versus placebo. Elinzanetant successfully met all four primary endpoints in both studies demonstrating statistically significant reductions in the frequency and severity of moderate to severe vasomotor symptoms (VMS, also known as hot flashes) from baseline to week 4 and 12 compared to placebo.

Both studies also achieved all three key secondary endpoints showing a statistically significant reduction in the frequency of VMS from baseline to week 1, as well as statistically significant improvements in sleep disturbances and menopause-related quality of life compared to placebo. The safety profile observed in the OASIS 1 and 2 studies is overall consistent with previously published data1,2 on elinzanetant.

“We are excited about the positive results of these two pivotal Phase III studies for elinzanetant, reinforcing its potential as a non-hormonal treatment option in menopause management,” said Dr. Christian Rommel, member of the Executive Committee of Bayer AG’s Pharmaceutical Division and Global Head of Research and Development. “We want to thank the women participating in the OASIS studies, their families, and all study investigators as well as their clinical and nursing staff for their time and commitment to advance menopause research.”

Elinzanetant is a first dual neurokinin-1,3 (NK-1,3) receptor antagonist, in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily.

“Menopausal symptoms such as hot flashes and sleep disturbances can be highly disruptive and broadly impact health and quality of life, still many women cope in silence and remain untreated,” said JoAnn Pinkerton, M.D., Professor and Director of Midlife Health at UVA Health. “It’s important that we continue to research for solutions that address the unmet needs of women, and I am looking forward to the unveiling of the full results.”

OASIS 1 and 2 (NCT05042362 and NCT05099159) are the first two Phase III studies in the OASIS clinical development program with results, and the details are planned to be presented at upcoming scientific congresses. The results of the third Phase III study OASIS 3 (NCT05030584) are expected in the coming months. Bayer plans to submit data from the OASIS 1, 2 and 3 studies to health authorities for approval of marketing authorizations for treatment of moderate to severe VMS associated with menopause.

About the OASIS 1 and 2 studies
OASIS 1 and 2 are double-blind, randomized, placebo-controlled multicenter studies investigating the efficacy and safety of elinzanetant administered orally once daily in women with moderate to severe VMS associated with menopause. OASIS 1 and 2 randomized 396 and 400 postmenopausal women between 40 and 65 years across 184 sites in 15 countries.

About the OASIS Clinical Development Program
The Phase III clinical development program of elinzanetant, OASIS, currently comprises four Phase III studies: OASIS 1, 2, 3 and 4. The OASIS 1, 2 and 3 studies investigate the efficacy and safety of elinzanetant 120 mg in women with moderate to severe VMS associated with menopause. The OASIS 4 study is an expansion of the clinical phase III program and investigates the efficacy and safety of elinzanetant in women with moderate to severe VMS caused by endocrine therapy for treatment or prevention of breast cancer.

The design and dosing of the Phase III clinical development program is based on the positive data from two Phase II studies (RELENT-1 and SWITCH-1). RELENT-1 was a Phase Ib/IIa study investigating the safety, pharmacokinetics and preliminary efficacy of elinzanetant. SWITCH-1 was a Phase IIb study investigating the efficacy and safety of four different doses of elinzanetant compared to placebo in women with VMS.

About Elinzanetant
Elinzanetant is a first dual neurokinin-1,3 (NK-1,3) receptor antagonist, in late-stage clinical development for the non-hormonal treatment of moderate to severe VMS associated with menopause, administered orally once daily. Elinzanetant may address moderate to severe VMS by modulating a group of estrogen sensitive neurons in the hypothalamus region of the brain (the KNDy neurons) which, with the decrease of estrogen, become hypertrophic and lead to a hyperactivation of the thermoregulatory pathway, consequently disrupting body heat control mechanisms resulting in VMS. Elinzanetant may also decrease sleep disturbances associated with menopause.

About Vasomotor Symptoms
Vasomotor symptoms (VMS; also referred to as hot flashes) result from hyperactivation of the thermoregulatory pathway mediated by hypertrophy of the KNDy neurons. This is due to a decrease of estrogen, which can result from the progressive reduction of ovarian function due to natural menopause or medical intervention by bilateral oophorectomy or endocrine therapy.

VMS are reported by up to 80% of women at some point during the menopausal transition and are one of the leading causes for seeking medical attention during this phase of a woman’s life. Over one-third of menopausal women report severe symptoms, which can last 10 years or more after the last menstrual period, with relevant impact on quality of life.

VMS may also be caused by endocrine therapy, for the treatment or prevention of breast cancer, impacting quality of life and treatment adherence. For these women, there are currently no approved treatment options.

About Menopause
By 2030, the world population of women experiencing menopause is projected to increase to 1.2 billion, with 47 million new women entering this phase each year. Menopause is a transitional phase in women’s lives, related to the progressive decline of ovarian function, and which usually occurs in women during their 40s or early 50s. It can also be the result of surgical or medical treatment, for example breast cancer treatment. The hormonal decline can lead to various symptoms which can substantially affect a woman’s health, quality of life, healthcare utilization and work productivity. The most frequently reported and disruptive symptoms during the menopausal transition are VMS, sleep disturbances and mood changes. Addressing these symptoms is key to maintaining functional ability and quality of life in menopause which is highly relevant from both a healthcare and socio-economic perspective.

About Women’s Healthcare at Bayer
Women’s Health is in Bayer’s DNA and as a global leader in women’s healthcare Bayer has a long-standing commitment to delivering science for a better life by advancing a portfolio of innovative treatments. Bayer offers a wide range of effective short- and long-acting birth control methods as well as therapies for menopause management and gynecological diseases. Bayer is also focusing on innovative options to address the unmet medical needs of women worldwide and to broadening treatment choices such as in menopause. Additionally, Bayer intends to provide 100 million women per year in low-and-middle income countries by 2030 with access to family planning by funding multi-stakeholder aid programs for capacity building and by ensuring the supply of affordable modern contraceptives. This is part of the comprehensive sustainability measures and commitments from 2020 onwards and in line with the Sustainable Development Goals of the United Nations.

About Bayer
Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2022, the Group employed around 101,000 people and had sales of 50.7 billion euros. R&D expenses before special items amounted to 6.2 billion euros. For more information, go to www.bayer.com.

Find more information at https://pharma.bayer.com
Follow us on Facebook: http://www.facebook.com/bayer
Follow us on LinkedIn: Bayer | Pharmaceuticals

Forward-Looking Statements
This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

References:

1 Simon JA, Anderson RA, Ballantyne E, Bolognese J, Caetano C, Joffe H, Kerr M, Panay N, Seitz C, Seymore S, Trower M, Zuurman L, Pawsey S. Efficacy and safety of elinzanetant, a selective neurokinin-1,3 receptor antagonist for vasomotor symptoms: a dose-finding clinical trial (SWITCH-1). Menopause. 2023 Mar 1;30(3):239-246.

2 Trower M, et al. Effects of NT-814, a dual neurokinin 1 and 3 receptor antagonist, on vasomotor symptoms in postmenopausal women: a placebo-controlled, randomized trial. Menopause: The Journal of The North American Menopause Society. 2020; 27 (5): 498-505.

Contact for global media inquiries:
Katja Wiggers, phone +49 30 221541614
Email: katja.wiggers@bayer.com

Contact for US media inquiries:
Courtney Ambrosi, phone 1 (908) 798-1107
Email: courtney.ambrosi@bayer.com

Contact for investor inquiries:
Bayer Investor Relations Team, phone +49 214 30-72704
Email: ir@bayer.com
www.bayer.com/en/investors/ir-team