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Sep 26
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Amphista Therapeutics Achieves First Milestone in Second Discovery Programme with Bristol Myers Squibb

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

 

PRESS RELEASE

 

Amphista Therapeutics Achieves First Milestone in Second Discovery Programme with Bristol Myers Squibb

 

Cambridge, UK, September 26th, 2023 – Amphista Therapeutics (“the Company” or “Amphista”), a leader in next generation targeted protein degradation (TPD) approaches, today announces it has achieved the first milestone in the second active discovery programme under its strategic collaboration and license agreement with Bristol Myers Squibb (NYSE: BMY), triggering a payment for achieving the milestone.

This  follows the announcement of the first milestone achieved in the first discovery programme  on May 4, 2023. Both programmes are part of the original agreement with Bristol Myers Squibb, which was announced in May 4, 2022 and included a $30 million upfront payment and the potential for up to $1.25 billion in performance-based milestone payments and payment for a limited expansion of the collaboration, as well as royalties on global net sales of products.

Louise Modis, Chief Scientific Officer of Amphista Therapeutics said: “The achievement of this first milestone in the second discovery programme under Amphista’s strategic collaboration with Bristol Myers Squibb is a testament to our innovative science and ability to discover and progress at pace differentiated, novel therapeutic molecules for our pharmaceutical partners. We have now delivered milestones on both active discovery programmes in our ongoing collaboration with the company and we look forward to continuing this strong momentum. Such collaborations further validate our unique approach and ambition to overcome challenges faced by other target protein degradation modalities.  With potential for CNS penetration, we continue to expand the range of potential applications for Amphista’s therapeutic modality.”

Under the agreement, Amphista is responsible for the discovery and development of small molecule protein degraders using EclipsysTM, its next-generation TPD platform. Bristol Myers Squibb is granted a global exclusive license to the resulting degraders and will be responsible for further development and commercialization activities.

ENDS

 

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  

 

For more information please contact:

Consilium Strategic Communications

Amber Fennell, Namrata Taak, Stella Lempidaki

Email: Amphista@consilium-comms.com

Tel: +44 (0)20 3709 5813

 

 

Aug 09
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Aleta Biotherapeutics Granted MHRA Clinical Trial Authorization for Biologic CAR T-Cell Engager ALETA-001

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

 

Aleta Biotherapeutics Granted MHRA Clinical Trial Authorization for Biologic CAR T-Cell Engager ALETA-001

 

  • Aleta’s lead program, ALETA-001 has clinical support from collaborator Cancer Research UK, which will initiate the Phase 1/2 Clinical Trial during H2 2023

 

  • ALETA-001, previously granted a UK Innovation Passport, was developed to address the urgent unmet patient need of relapse after CD19 CAR T-Cell Cancer Treatment

 

 

NATICK, Mass., August 8, 2023 – Aleta Biotherapeutics (Aleta), a privately-held immuno-oncology company with a platform of CAR T-Cell Engagers (CTE) which enable cell therapies to improve outcomes for patients with cancer, announces that the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has granted clinical trial authorization (CTA) to evaluate investigational biologic ALETA-001 in a Phase 1/2 clinical trial in the treatment of patients with B-cell malignancies who are relapsed/refractory to CD19 CAR T-cell therapy.

 

Aleta’s lead program, ALETA-001 has clinical support and funding from its collaboration partner, Cancer Research UK’s Centre for Drug Development, which has sponsored and will conduct the Phase 1/2 clinical trial.

 

“The authorization to clinically evaluate, in collaboration with Cancer Research UK, the potent activity of ALETA-001, our lead biologic CAR T-Cell Therapy Engager, marks important progress for the entire global oncology community – especially patients,” commented Paul Rennert, Ph.D., Chief Executive Officer and Chief Scientific Officer, Aleta Biotherapeutics. “Aleta’s CTEs act by transforming the expression of any cancer tumor cell to match the CAR T-cell therapies circulating in a patient’s blood, importantly restoring and increasing the effectiveness with which CAR T-Cells can kill cancer cells,” continued Dr. Rennert.

 

Dr. Nigel Blackburn, Cancer Research UK’s Director of Drug Development, stated,

“CAR-T cell therapy has been transformative for some cancer patients but there remains a critical need to ensure that this therapy is an option for all. ALETA-001 is working to address this treatment gap for people with blood cancers by advancing a potentially life-saving CAR-T cell therapy into the clinic. We are delighted to play a key role in this trial and look forward to seeing this treatment become available for more patients in the future.”

 

 

 

About CAR T-Cell Therapy Engager (CTE) ALETA-001

Aleta’s lead development program, ALETA-001, contains the CD19 target protein which is further linked to an CD20 antibody domain. ALETA-001 is designed to improve the effectiveness of CD19-directed CAR T therapies by increasing CD19 antigen density and restoring lost CD19 expression on the cancer cell. 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 I/II 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 Therapeutics is pioneering a transformation of cancer treatment by enabling CAR-T cell cancer therapies to work more effectively. The Company’s unique portfolio of multi-antigen 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 program, ALETA-001, is clinic-ready for the treatment of B-cell malignancies in patients who are relapsed/refractory to CD19-directed CAR T-cell therapy. Additional CTEs in the Aleta portfolio include:

  • ALETA-004: a preclinical, multi-antigen binding CTE designed to extend the cancer killing activity of CAR-T cells to treat advanced acute myeloid leukemia, and
  • ALETA-005: a discovery phase CTE that allows multiple myeloma tumors to present multiple antigen binding sites to B-cell maturation antigen (BCMA) CAR T-cells, stimulating optimal BCMA CAR-T cell killing of advanced multiple myeloma.

Aleta is further developing its CTE platform technology to address solid tumors including breast cancer, gastric cancer, and pediatric brain cancers.

 

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 25 years, taking over 150 potential new anti-cancer agents into clinical trials in patients. It currently has a portfolio of 21 new anti-cancer agents in preclinical development, Phase I or early Phase II 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. Two other drugs are in late development Phase III trials. www.cruk.org.uk/cdd

 

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Contacts:

Media:

Linda Phelan Dyson, MPH

973-986-5973

lpdyson@verizon.net

Jun 02
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Artax Biopharma Announces Positive Phase 1 Results for AX-158, Company’s First-in-Class Oral Immunomodulator Developed to Treat T Cell-Mediated Diseases

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

 

Artax Biopharma Announces Positive Phase 1 Results for AX-158, Company’s First-in-Class Oral Immunomodulator Developed to Treat T Cell-Mediated Diseases

 

Phase 1 Findings Demonstrated that Investigational Agent AX-158 Was Well Tolerated at All Doses, with a Profile that Strongly Supports Further Clinical Development Efforts

AX-158 is Being Developed for Once Daily Oral Dosing to Address Autoimmune Diseases by Providing Immunomodulation without Immunosuppression

 

Cambridge, Mass., June 1, 2023 – Artax Biopharma, Inc., a clinical-stage biotechnology     company focused on transforming the treatment of T Cell-mediated diseases, today announces positive Phase 1 clinical trial results for AX-158, the Company’s first-in-class oral small molecule immunomodulator to enter clinical development to treat T Cell-mediated diseases.

 

The Phase 1 clinical trial findings demonstrated that AX-158:

  • had no related adverse events in the clinical trial;
  • was well tolerated at all doses, up to the maximum of 50mg QD in the single ascending dose cohorts and up to the maximum of 15mg QD for 10 days in the multiple ascending dose cohorts; and
  • had a consistent, predictable pharmacokinetic (PK) profile and bioavailability among healthy volunteers, which supports the use of AX-158 as a once daily oral dosing regimen, which can be dosed independently of food.

 

Importantly, these positive results support the further evaluation of AX-158 at a once daily dose of 10mg in a Phase 2 clinical trial. AX-158 employs a first-in-class mechanism of action that selectively modulates T cell responses that play a critical role in healthy immune system function. AX-158 has the potential to treat T Cell-mediated diseases through immunomodulation without the risk of immunosuppression.

 

“With these strong Phase 1 results suggesting AX-158’s clean safety profile, we are eager to further evaluate safety and efficacy in Phase 2 clinical studies, and to ultimately making AX-158 available to patients managing complex T Cell-driven conditions, including autoimmune diseases, and induced T Cell pathologies,” stated Artax Biopharma Chief Executive Officer Joseph Lobacki. “AX-158 has the potential   to positively impact health outcomes through rebalancing immune system function, while maintaining a strong response to foreign pathogens and infections – providing true immunomodulation without immunosuppression.”

 

About Artax Science and Immunomodulation

A healthy immune system eliminates harmful foreign pathogens, while being tolerant of self-tissues and organs. The T Cell Receptor (TCR) is central to healthy T Cell function and a well-functioning immune system. When TCR signaling becomes dysregulated, T Cells behave abnormally. This behavior results in T Cell-driven conditions, including autoimmune diseases, and induced T Cell pathologies in which medical treatments result in immune reactions (such as stem cell transplants resulting in acute graft-versus-host- disease or immuno-oncology treatments resulting in immune related-adverse events). Artax believes immunomodulation – a mechanism through which our investigational agents assist in modulating the immune system to ameliorate T Cell-mediated diseases while not impacting patients’ ability to properly fight foreign pathogens – holds great potential.

About Artax-158

AX-158 is a first-in-class, oral small molecule immunomodulating agent in clinical development for the treatment of T Cell-mediated diseases. AX-158 employs a novel mechanism of action that selectively modulates, or adjusts, T Cell responses that play a critical role in immune system function. Nck is a protein that naturally amplifies T Cell signaling directly at the TCR, contributing to T Cell mediated disease. AX-158 is a Nck SH3.1 domain inhibitor which selectively blocks the role of Nck in T Cells.  Immunomodulation assists the immune system to maintain healthy control and eliminates the underlying driver of T Cell-mediated diseases. Importantly, Artax believes that preclinical data suggests AX-158 will not be immunosuppressive and so will not impact the immune system’s ability to mount a strong response to foreign pathogens and infections.

About Artax Biopharma

Artax Biopharma is a clinical-stage biotechnology company transforming T Cell-mediated disease treatment by developing innovative small molecules that modulate the immune system without causing immunosuppression. Artax science holds broad potential to treat T Cell-mediated diseases such as autoimmune diseases, induced T cell pathologies (such as acute graft versus host disease and immune- oncology treatment-related adverse events) and T Cell malignancies, while simultaneously allowing the body to fight foreign pathogens. For more information, please visit www.artaxbiopharma.com.

                                                                #  #  #

Contacts:

Maria Nichol, DPhil, EPA, CPA

Vice President, Business Development

+44 7901 666564

mnichol@artaxbiopharma.com

Media:

Linda Phelan Dyson

+1 973-986-5873

lpdyson@verizon.net

May 04
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Amphista Therapeutics Achieves First Discovery Milestone in Collaboration with Bristol Myers Squibb

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

 

Amphista Therapeutics Achieves First Discovery Milestone in Collaboration with Bristol Myers Squibb

4 May 2023

CAMBRIDGE, UK, May 4, 2023 – Amphista Therapeutics, a leader in next generation targeted protein degradation (TPD) approaches, today announced the delivery of the first discovery milestone under its collaboration and license agreement with Bristol Myers Squibb, triggering a payment for achieving the milestone.

Nicola Thompson, CEO of Amphista, said, “we are absolutely delighted with the progress and success of our collaboration with BMS. Our ability to deliver our first milestone within the first year of our collaboration exemplifies the strength of our Eclipsys TM platform and our ambition to be a world-leading, next generation protein degradation company.”

The collaboration and license agreement with Bristol Myers Squibb was announced on May 4, 2022 and included a $30 million upfront payment, the potential for up to $1.25 billion in performance-based milestone payments and payment for a limited expansion of the collaboration, as well as royalties on global net sales of products. Amphista is responsible for the discovery and development of small molecule protein degraders using Eclipsys TM, its next-generation TPD platform. Bristol Myers Squibb is granted a global exclusive license to the resulting degraders and will be responsible for further development and commercialization activities.

Amphista’s next generation bifunctional molecules use a novel approach that makes use of a wider range of the body’s own innate protein degrading mechanisms than those used by most other TPD companies. This proprietary approach offers the potential to overcome many of the limitations seen with current TPD approaches, providing the opportunity to treat a wider range of diseases. Amphista is focused on biological targets with a high level of clinical or genetic validation.

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 labs 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.

Amphista, the Amphista logo and Eclipsys are all trademarks of Amphista Therapeutics Limited

Media contacts

Lynn Granito
Berry & Company Public Relations
lgranito@berrypr.com
+1 212 253 8881

May 02
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Eloxx Pharmaceuticals Announces FDA Clearance to Begin Single Ascending Dose Study of ZKN-013

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

 

May 2, 2023
ZKN-013 is in development for potential treatment of recessive dystrophic and junctional epidermolysis bullosa (RDEB and JEB) and Familial
Adenomatous Polyposis (FAP)
ZKN-013 is Eloxx’s first drug candidate from its TURBO-ZM™ platform to gain clearance for clinical testing
WATERTOWN, Mass., May 02, 2023 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) (“Eloxx” or the “Company”), a leader in
ribosomal RNA-targeted genetic therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the
Company’s Investigational New Drug (IND) application to initiate a single ascending dose (SAD) clinical trial in healthy volunteers for ZKN-013 for the
potential treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by
mutations in Collagen7 gene.
“FDA clearance to begin our planned single ascending dose trial is an important milestone towards providing a potential treatment option for patients
with RDEB and JEB,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “This update is also a momentous event for Eloxx, as
ZKN-013, our lead TURBO-ZM™ based molecule, is the first program developed from hit to lead and is now poised to enter clinical development.
Beyond ZKN-013, Eloxx has promising TURBO-ZM based discovery programs in cystic fibrosis (fully funded by the Cystic Fibrosis Foundation) and in
certain cMYC-overexpressing cancers.”
Further SAD and multiple ascending dose (MAD) testing is expected to be conducted following the completion of the planned dose cohorts in the SAD
study and discussion with the FDA. The MAD testing could potentially include RDEB patients given the strong benefit/risk in patients cited by the FDA.
ZKN-013 Preclinical Activity in RDEB, JEB and FAP
ZKN-013 has demonstrated robust functional preclinical activity in RDEB and JEB patient cells and in APCmin (multiple intestinal neoplasia) mice.
Eloxx also plans to develop ZKN-013 for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and
progression to colon cancer. In January 2023, Eloxx published its preclinical results that showed treatment with ZKN-013 demonstrated a decrease in
intestinal polyps and adenomas, resulting in increased survival.
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. Eloxx also has preclinical programs
focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant
polycystic kidney disease, as well as rare ocular genetic disorders.
For more information, please visit www.eloxxpharma.com.
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 present and historical facts contained in this press release, including without limitation, statements regarding clinical testing of
ZKN-013 for the treatment of RDEB and JEB and the expected timing of trials for our other product candidates and the potential of our product
candidates to treat nonsense mutations 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 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 preclinical studies and clinical trials and other research
and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic
on our clinical trials, operations, vendors, suppliers, and employees; 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 financial 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 Annual Report on Form 10-K for the
fiscal year ended December 31, 2022, 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.
Contact
Investors
John Woolford
john.woolford@westwicke.com
443.213.0506
Media
Laureen Cassidy
laureen@outcomescg.com
SOURCE: Eloxx Pharmaceuticals, Inc.

Apr 21
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Orphalan announces US commercial launch of Cuvrior™ for the treatment of Wilson disease

By Orphalan, Press Release, Private Companies
Press Release.

 

– Cuvrior™ now available in the US for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to penicillamine

– Oral administration offers dosing flexibility and portability

Paris, France, April 20, 2023 – Orphalan SA (“Orphalan” or “the Company”), an international orphan drug development and commercialization company, today announces the commercial launch in the US of Cuvrior™, a new trientine tetrahydrochloride (TETA-4HCl). Cuvrior™ is now available for the treatment of adult patients with stable Wilson disease who are de-coppered and tolerant to D-penicillamine.

Cuvrior™ was approved by the United States Food and Drug Administration (FDA) and was granted Orphan Drug Exclusivity (ODE) in April 2022 for this patient group. Physicians can now discuss the availability of Cuvrior™ as a treatment option with their patients and can access important information here about prescribing Cuvrior™.

Compared with the standard of care, Cuvrior™ facilitates precise dosing by providing a small, scored tablet in a blister pack. Additionally, its room temperature stability eliminates the need for refrigeration, making it a more practical solution for patients with busy lifestyles.

Wilson disease is a rare inherited disorder of copper transport primarily affecting the liver and brain. Since 2019, Orphalan has commercialized its trientine tetrahydrochloride product in Europe under the name of Cuprior® and approximatively 1,000 Wilson disease patients have received treatment, with a positive track record on safety and efficacy.

Cuvrior™ was approved by the FDA on the basis of data from the Company’s phase III CHELATE trial, the first prospective randomized trial comparing penicillamine with TETA-4HCl. During the trial, an innovative assay was developed to measure non-caeruloplasmin bound copper (NCC), the free and potentially toxic pool of copper in the blood. Using this NCC measurement in patients previously receiving maintenance penicillamine therapy, TETA-4HCl was determined to be non-inferior to penicillamine at the primary endpoint of the study (24 weeks), with the same observation at the end of the extension phase of the study (one year from randomization).

Dmitry Paramonov, President Orphalan US, commented:

“We are thrilled to introduce Cuvrior™ in the US, a safe and innovative treatment option for patients and caregivers managing Wilson disease. Our distribution partners have Cuvrior™ stocked and ready for immediate use. At Orphalan, our focus is on providing targeted solutions for rare diseases, as demonstrated by the successful launch of Cuprior® in Europe. We are eager to expand availability of Cuvrior™ to as many patients as possible, underscoring our dedication to making a meaningful impact on the lives of rare disease patients worldwide.”

 

                                                                         ENDS                        

About Orphalan

Orphalan is a pioneering, international orphan drug development and commercialization company. Founded in 2011, the company develops and delivers innovative therapies for people living with orphan diseases, and is initially focused on Wilson disease, a rare genetic disorder that can be life-threatening if untreated. Orphalan commercializes Cuprior®, its trientine tetrahydrochloride product for the treatment of Wilson disease in Europe, and has launched Cuvrior™ in the US. For more information visit www.orphalan.com and follow us on LinkedIn.

About Cuvrior™

Cuvrior™ is an innovative new oral formulation of trientine and the first new advance in Wilson disease in over 30 years. Cuvrior™’s FDA approval was supported by a global phase III trial, CHELATE, which met its primary efficacy endpoint by demonstrating that Cuvrior™ was non-inferior to penicillamine as measured by serum non-caeruloplasmin copper assay (NCC) [1,2].

About Wilson Disease

Wilson disease is a rare genetic disease that is characterized by gradual accumulation of dietary copper over time, possibly to life-threatening levels. Affected individuals are unable to effectively excrete copper naturally through the digestive tract. Common symptoms associated with Wilson disease include progressive liver disfunction, neurological disorders such as severe tremors, and mental health deterioration. About 1 in 15 patients eventually need a liver transplant. Wilson disease affects nearly 1 in every 30,000 people worldwide.

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

Consilium Strategic Communications: 
Mary-Jane Elliott, Tracy Cheung, Davide Salvi
Tel: +44 (0) 203 709 5700
orphalan@consilium-comms.com

References

[1] Trientine tetrahydrochloride versus penicillamine for maintenance therapy in Wilson disease (CHELATE): a randomized, open-label, non-inferiority, phase III trial, Schilsky et al, Lancet Gastroenterol Hepatol 2022, Published Online September 29, 2022

[2] CuvriorTM USPI, 215760s000lbl.pdf (fda.gov)

Apr 10
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FDA Authorizes Marketing of MISHA™ Knee System for People Suffering from Knee Osteoarthritis

By Moximed, Press Release, Private Companies
Press Release.

 

FREMONT, Calif.–(BUSINESS WIRE)–Moximed, a medical device company on a mission to improve the standard of care for people with knee osteoarthritis (OA), today announced that the U.S. Food and Drug Administration (FDA) granted marketing authorization of the MISHA™ Knee System, an implantable shock absorber (ISA) for the knee. The MISHA Knee System is indicated to treat people with medial knee OA who failed to find relief from non-surgical or surgical treatment, continue to experience pain that interferes with daily activities, and are ineligible for, or unwilling to undergo, joint replacement due to age or absence of advanced OA.

“I’ve been lucky enough to have been exposed to many major orthopedic innovations, most notably total knee arthroplasty (TKA). TKA is a phenomenal operation, but it has its limitations. I still see a clinical void between arthroscopic knee preservation and arthroplasty,” said Anil Ranawat, M.D., Chief, Hip and Knee Division of Sports Medicine Institute, Hospital for Special Surgery in New York. “With the FDA’s clearance of the MISHA Knee System, millions of people with symptomatic knee OA will now have an opportunity to achieve high levels of pain relief, enjoy the lifestyle and activities that are important to them, and preserve the option for a primary knee arthroplasty in the future. This system fills that void for both surgeons and patients.”

“Knee replacement is undoubtedly the gold standard for well-selected patients with severe OA, but for every patient I see that is ready for joint replacement, I see as many, if not more, who are not yet good arthroplasty candidates,” said William A. Jiranek, M.D., former President of the American Association of Hip and Knee Surgeons (AAHKS). “In my current role as Vice Chair for Practice Innovation at Duke University, I consider treatments for OA across the entire disease continuum, rather than focusing only on the end stage. As such, I was invited to provide an independent, non-investigator assessment of Moximed’s clinical study protocol, and I provided an independent, periodic review of the data during the study. I’m excited that the study met its superiority composite endpoint, which included effectiveness and safety components. This new joint-preserving treatment provides arthritis surgeons the ability to intervene earlier in the disease process to help patients not ready for a joint replacement.”

Moximed utilized the clinically established benefits of load reduction on diseased joints to design the MISHA Knee System. Implanted during an outpatient-compatible procedure, the MISHA Knee System demonstrated superiority over high tibial osteotomy (HTO), a well-established surgery with decades of demonstrated clinical results, in its recent pivotal clinical study.i

“This is a milestone event for knee OA sufferers, and it’s the result of unwavering clinical research and development that spans more than 10 years. We offer special thanks to our study patients and surgeon investigators who helped advance the understanding of this new treatment for OA. Also, we recognize the dedicated reviewers at FDA for completing their thorough benefit-risk assessment of our breakthrough technology. We’re thrilled to now be in a position to make the surgery available to patients,” said Anton Clifford, PhD, founder and CEO of Moximed. “We are committed to providing excellent medical education and customer service, supporting selection and treatment of indicated patients, and demonstrating scalability of our business as we introduce the MISHA Knee System to the U.S.”

Osteoarthritis (OA) is a common, debilitating condition, affecting the lives of over 32 million adults in the U.S, and projected to impact 70 million Americans by 2040. Knee OA develops when the joint’s natural shock absorbers, cartilage and meniscus, no longer cushion the joint from daily activities, leading to chronic pain and activity limitation. Many people with mild to moderate OA are otherwise healthy, in their prime working years, and have busy lives to live. For these patients, total knee replacement is a reluctant option, as it is an end-stage treatment for end-stage disease. OA patients without end-stage disease seek options that preserve their knee, activity level, and quality of life.

About Moximed

Moximed was founded in 2008 and is dedicated to helping people with mild to moderate osteoarthritis preserve their knee joints while living healthy, active lives. The company’s technology, the MISHA™ Knee System, is the result of over a decade of clinical research and development and is the first implantable shock absorber being developed for the treatment of medial compartment knee osteoarthritis (OA). With experienced medtech leadership and strong investor support, Moximed is poised to elevate the standard of care and quality of life for millions of pre-arthroplasty knee OA sufferers hindered by arthritic knee pain and function loss. Moximed is based in Fremont, California.

To learn more, visit www.moximed.com.

About The MISHA™ Knee System

Reducing weight on painful osteoarthritic joints is known to reduce pain and improve function. The MISHA™ Knee System being developed is the first implantable shock absorber that reduces weight on the knee joint with every walking step, easing pain, preserving function, and possibly delaying joint replacement surgery. The implant is placed on the medial knee and moves with the natural joint, reducing about 30% of the peak force on the knee with every walking step.ii

i Diduch DR, Crawford DC, Ranawat AS, Victor J, Flanigan DC. Implantable Shock Absorber Provides Superior Pain Relief and Functional Improvement Compared With High Tibial Osteotomy in Patients with Mild-to-Moderate Medial Knee Osteoarthritis: A 2-Year Report. CARTILAGE. 2023;0(0). doi:10.1177/19476035231157335.
ii Morgan OJ, Hillstrom HJ, Ranawat A, Fragomen AT, Rozbruch SR, Hillstrom R. Effects of a Medial Knee Unloading Implant on Tibiofemoral Joint Mechanics During Walking. J Orthop Res. 2019;37(10):2149-2156. doi:10.1002/jor.24379.

MEDIA CONTACTS

Annika Parrish

Health+Commerce

Mar 29
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Eloxx Pharmaceuticals Announces Submission of Investigational New Drug (IND) Application for ZKN-013

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

 

  • ZKN-013 in development for treatment of recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB)
  • Additional IND filing for ZKN-013 for treatment of familial adenomatous polyposis (FAP) expected in first half of 2023
  • Recent preclinical results have provided additional support for ZKN-013 in RDEB and FAP

WATERTOWN, Mass., March 28, 2023 (GLOBE NEWSWIRE) — Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today announced the submission of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration for ZKN-013 for the treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene.

“This IND application is an important milestone towards providing a treatment option for patients with RDEB and JEB, as there are currently no approved disease-modifying treatments,” said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. “We look forward to advancing ZKN-013, our lead TURBO-ZMTM based molecule, into clinical development, as we believe it has the potential to have a positive impact on the treatment of these two painful and debilitating diseases.”

Recent preclinical results demonstrated read-through activity of ZKN-013 in multiple COL7 genotypes across multiple RDEB patient derived fibroblasts and keratinocytes. Read-through activity resulted in up to an 18-fold increase in full-length COL VII protein levels. Prolonged treatment with ZKN-013 further increased COL VII protein levels. Functionality of the restored full-length COL VII protein was confirmed. These results have been accepted for presentation at an upcoming medical conference.

An additional IND filing for ZKN-013 for the treatment of familial adenomatous polyposis (FAP) is planned in the first half of 2023. FAP, a rare inherited disease with no approved drug therapies, is characterized by proliferation of colon polyps. Eloxx is targeting a subset of patients that have nonsense mutations in the Adenomatous Polyposis Coli (APC) gene that is truncated in these patients.

In January 2023, Eloxx published positive results from a study in the APCMin (multiple intestinal neoplasia) model evaluating the potential of ZKN-013 to treat FAP. The APCMin mouse is a translationally validated model for drug development for FAP. In the APCMin model, treatment with ZKN-013 demonstrated a decrease in intestinal polyps and adenomas, resulting in increased survival. The publication also included in vitro and in vivo demonstrating that ZKN-013 inhibited the growth of human colon carcinoma cells with APC nonsense mutations, and promoted read through of premature stop codons in the APC gene, leading to functional restoration of full-length APC protein.

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is engaged in the science of ribosome modulation, leveraging its innovative TURBO-ZMTM 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. The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ELX-02 for the treatment of CF patients with nonsense mutations. In addition, ELX-02 has also been granted Orphan Drug Designation for the treatment of CF patients with nonsense mutations by the FDA and orphan medicinal product designation by the European Commission. ELX-02 is in clinical development, focusing on cystic fibrosis (US Trial NCT04135495, EU/IL Trial NCT04126473). Eloxx also has preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases, including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.

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

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 present and historical facts contained in this press release, including without limitation, the expected timing of trials of our product candidates and the potential of our product candidate to treat nonsense mutations 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 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 preclinical studies and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development; the impact of the global COVID-19 pandemic on our clinical trials, operations, vendors, suppliers, and employees; 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 financial in the future through product licensing, public or private equity or debt financing or otherwise; 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 quarter ended September 30, 2022, as any such factors may be updated from time to time in our other filings with the SEC, accessible on the SEC’s website atwww.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.

Contact

Investors
John Woolford john.woolford@westwicke.com 443.213.0506

Media
Laureen Cassidy laureen@outcomescg.com

SOURCE: Eloxx Pharmaceuticals, Inc.

Feb 16
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Aura Biosciences Announces Positive Interim Phase 2 Safety and Efficacy Data of Belzupacap Sarotalocan (Bel-sar) for the First-Line Treatment of Patients with Early-Stage Choroidal Melanoma with Suprachoroidal Administration

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

 

Aura Biosciences Announces Positive Interim Phase 2 Safety and Efficacy Data of Belzupacap Sarotalocan (Bel-sar) for the First-Line Treatment of Patients with Early-Stage Choroidal Melanoma with Suprachoroidal Administration

Boston, USA, February 16 2023 – Aura Biosciences, Inc. (“Aura”) (Nasdaq: AURA), a clinical-stage biotechnology company developing a novel class of virus-like drug conjugate (VDC) therapies for multiple oncology indications, today announced the presentation of positive interim Phase 2 safety and efficacy data of bel-sar with 9-10 months of follow up evaluating two key clinical endpoints: tumor control and visual acuity preservation using the suprachoroidal (SC) route of administration for the first-line treatment of patients with early-stage choroidal melanoma (indeterminate lesions and small choroidal melanoma (IL/CM)). The results were presented at the Macula Society 46th Annual Meeting held February 15-18, 2023, in Miami, FL.

“The data presented today with an average of nine months of follow up for patients treated with three cycles of therapy, show an excellent response to the therapy with 89-100% tumor control. In addition, the safety profile to date has been favorable with only one patient losing visual acuity and no treatment-related SAEs or significant AEs, which is encouraging given that the majority of these patients had tumors close to the fovea or optic disk and would have likely experienced severe and irreversible vision loss with the current standard of care with radiotherapy,” said Dr. Ivana Kim, Director of the Ocular Melanoma Center, Massachusetts Eye and Ear. “These latest results strongly support the potential of bel-sar to be used as a first line treatment option for patients with early-stage choroidal melanoma.”

“We are excited with the interim efficacy data of the Phase 2 study which strongly supports the assumptions for the success of the global Phase 3 trial,” said Dr. Cadmus Rich, Chief Medical Officer of Aura Biosciences. “Collectively, we believe these interim data provide strong confidence to support the launch of a global Phase 3 trial which is on track to begin enrollment this year.”

The presentation can be accessed on the Company’s website: link

Updated Safety and Efficacy Data from the Ongoing Phase 2 Trial with SC Administration

This Phase 2 trial (NCT04417530) is assessing the safety and preliminary efficacy of single- and multiple ascending-doses of bel-sar up to three cycles of treatment via SC administration for the first-line treatment of early-stage choroidal melanoma. A total of 20 adult patients have been enrolled in the trial including the single dose Cohorts 1-3 (n=6) and multiple dose escalation Cohorts 4-6 (n=14). Cohorts 5 and 6 received up to three cycles of therapy, which was considered the therapeutic regimen for evaluation. One patient in Cohort 5 (n=3) received two cycles of therapy and two patients in Cohort 5 received three cycles of therapy (40 μg/dose). All patients from Cohort 6 (n=8) were assigned to receive three cycles of therapy at the highest dose (80 μg/dose). One patient from Cohort 6, who discontinued after one cycle due to unrelated serious adverse events (SAEs), is not included. All patients in Cohorts 5 and 6 had active growth at study entry, as an enrichment strategy to evaluate preliminary efficacy. This group of patients with active growth treated at the therapeutic regimen of three cycles was evaluated for tumor growth rate, tumor control, and visual acuity preservation as the defined clinical endpoints to evaluate preliminary efficacy. The results, with an average of nine months of follow up in patients who received three cycles of therapy in Cohorts 5 and 6, and who match the criteria for the planned global Phase 3 trial, showed a statistically significant reduction in the tumor growth rate (-0.289 mm/yr, p = <0.0001) compared to each patient’s documented growth rate at study entry, and a 100% (8/8) tumor control rate. In addition, the visual acuity preservation rate was 88% (7/8) in these cohorts, with the majority of patients being at high-risk for vision loss with tumors close to fovea or optic disk. The overall tolerability profile of bel-sar was generally favorable, with no dose-limiting toxicities, treatment-related SAEs or significant AEs reported as of January 10, 2023. There was no posterior inflammation and only mild anterior inflammation (Grade 1) in 20% of the patients. Treatment-related AEs were predominantly mild and resolved without sequalae. We believe these interim results indicate that bel-sar may offer a targeted, vision preserving therapy for the first-line treatment of primary CM, where 80% of patients are diagnosed early and have no approved therapies to date.

About Aura Biosciences

Aura Biosciences, Inc. is a clinical-stage biotechnology company developing virus-like drug conjugates (VDCs), a novel class of therapies, for the treatment of multiple oncology indications. Aura’s lead VDC candidate, belzupacap sarotalocan (bel-sar; AU-011), consists of a virus-like particle conjugated with an anti-cancer agent. Bel-sar is designed to selectively target and destroy cancer cells and activate the immune system with the potential to create long-lasting anti-tumor immunity. Bel-sar is currently in development for ocular cancers, and Aura has initiated activities for the global Phase 3 trial evaluating first-line treatment of early-stage choroidal melanoma, a vision- and life-threatening form of eye cancer where standard of care with radiotherapy leaves patients with severe comorbidities, including major vision loss. Aura plans to pursue development of bel-sar across its ocular oncology franchise including for the treatment of patients with choroidal metastasis. In addition, leveraging Aura’s technology platform, Aura is developing bel-sar more broadly across multiple cancers, including in patients with non-muscle invasive bladder cancer (NMIBC). Aura is headquartered in Boston, MA.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, and other federal securities laws. Any statements that are not statements of historical fact may be deemed to be forward looking statements. Words such as “may,” “will,” “could”, “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions that can be used to identify forward-looking statements. These forward looking statements include express or implied statements regarding Aura’s future expectations, plans and prospects, including, without limitation, statements regarding the therapeutic potential of bel-sar for the treatment of cancers including choroidal melanoma, non-muscle invasive bladder cancer and choroidal metastases; any express or implied statements regarding the Company’s expectations for the Phase 2 and Phase 3 clinical trials of bel-sar for early-stage choroidal melanoma; and Aura’s expectations regarding the estimated patient populations and related market opportunities for bel-sar.

The forward-looking statements in this press release are neither promises nor guarantees, and investors should not place undue reliance on these forward-looking statements because they involve known and unknown risks, uncertainties, and other factors, many of which are beyond Aura’s control and which could cause actual results to differ materially from those expressed or implied by these forward-looking statements, including, without limitation, an improved quality of life of patients after treatment with bel-sar; a potential paradigm shift in the approach to the treatment of choroidal melanoma; the urgent need for a vision preserving targeted therapy; the potential of bel-sar compared to the existing standard of care for patients with choroidal melanoma; uncertainties inherent in clinical trials and in the availability and timing of data from ongoing clinical trials; the expected timing for submissions for regulatory approval or review by governmental authorities; the risk that the results of Aura’s clinical trials may not be predictive of future results in connection with future clinical trials; the risk that interim data from ongoing clinical trials may not be predictive of final data from completed clinical trials; whether Aura will receive regulatory approvals to conduct trials or to market products; whether Aura’s cash resources will be sufficient to fund its foreseeable and unforeseeable operating expenses and capital expenditure requirements; risks, assumptions and uncertainties regarding the impact of the continuing COVID-19 pandemic on Aura’s business, operations, strategy, goals and anticipated timelines; Aura’s ongoing and planned pre-clinical activities; and Aura’s ability to initiate, enroll, conduct or complete ongoing and planned clinical trials. These risks, uncertainties, and other factors include those risks and uncertainties described under the heading “Risk Factors” in Aura’s most recent Annual Report on Form 10-K and Quarterly Report on Form 10-Q filed with the U.S. Securities and Exchange Commission (SEC) and in subsequent filings made by Aura with the SEC, which are available on the SEC’s website at www.sec.gov. Except as required by law, Aura disclaims any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise. These forward-looking statements are based on Aura’s current expectations and speak only as of the date hereof and no representations or warranties (express or implied) are made about the accuracy of any such forward-looking statements.

 

Contacts:

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

Argot Partners
Matthew DeYoung
aura@argotpartners.com

Source: Aura Biosciences, Inc.

Jan 13
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Pheno Therapeutics Announces Worldwide License From UCB to a Novel Program for Neurodegenerative Disease

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

 

Preclinical program with potential for disease modifying therapeutic

Edinburgh, UK January 11 2023 – Pheno Therapeutics Limited., a biotechnology company focused on the discovery and development of small molecule therapeutics that promote remyelination for the treatment of neurological diseases such as multiple sclerosis, announced today it has entered into an exclusive worldwide license agreement with UCB. The agreement grants Pheno Therapeutics access and rights to develop, manufacture and commercialize a preclinical-stage program of novel small molecules designed to promote remyelination.

“Pheno Therapeutics utilized its proprietary human phenotypic screening platform to discover novel and tractable therapeutic targets to modulate oligodendrocyte biology and promote remyelination. This license allows us to accelerate a promising drug target towards clinical development,” said Fraser Murray, PhD., Chief Executive Officer of Pheno Therapeutics. “Together with our human neurological drug development expertise, there is significant opportunity for Pheno Therapeutics to fast-track this program towards clinical proof-of-concept studies and potentially deliver transformational drugs for the treatment of  demyelination diseases.”

“License agreements like this are a demonstration of the value UCB scientists and our partners are creating through strong research productivity, and we are confident that Pheno Therapeutics, with its expertise in phenotypic screening, stem cell technology and myelin biology, will develop this preclinical program  to its full potential,” said Dhaval Patel, Executive Vice President and UCB’s Chief Scientific Officer.

Pheno Therapeutics will make a one-time upfront payment and will be responsible for development, manufacturing and global commercialization. UCB will receive milestones and tiered royalties on net sales. Further financial details of the agreement were not disclosed.

About the Program:

The preclinical program is a novel small molecule program designed to selectively modulate the activity of an undisclosed target that is preferentially expressed on human oligodendrocytes and is known to play a critical role in the biological pathway and process of myelination.  The therapeutic program is being developed by Pheno Therapeutics under an exclusive worldwide license from UCB.

About Pheno Therapeutics:

Pheno Therapeutics is a discovery and development focused biotechnology company developing small molecule therapeutics that boost the function of human oligodendrocytes and their natural ability to promote remyelination for the treatment of neurological diseases with high unmet medical need such as multiple sclerosis (MS).  MS is a devastating chronic disease with significant individual and societal impact that often manifests in young adults and is associated with a wide range of neurological symptoms which can progress to total physical and cognitive disability.  Pheno Therapeutics is seeking to promote remyelination and reverse the critical demyelination aspect of MS. MS demyelination occurs when the immune system attacks and damages the myelin sheaths that insulate and nourish axons and nerve fibres in the central nervous system, leading to multifocal demyelination, axonal injury and neurodegeneration. For more information on Pheno Therapeutics, please visit https://www.phenotherapeutics.com.

 

Contacts:

Fraser Murray, PhD.
Chief Executive Officer
Pheno Therapeutics
Fraser.Murray@PhenoTherapeutics.com

Katie Odgaard
Account Director
Zyme Communications
Katie.odgaard@zymecommunications.com

+44 (0)7787 502 947