Fortress Biotech and Cyprium Therapeutics Announce Positive Clinical Data for CUTX-101, Copper Histidinate, Presented at 2021 American Academy of Pediatrics National Conference & Exhibition


NEW YORK and SOLANA BEACH, Calif., Oct. 13, 2021 /PRNewswire/ — Cyprium Therapeutics, Inc. (“Cyprium”), a Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) accomplice firm, with help from its licensing accomplice Sentynl Therapeutics, Inc. (“Sentynl”), a completely owned subsidiary of Cadila Healthcare Restricted (“Zydus”), at the moment introduced optimistic outcomes from an efficacy and security evaluation of knowledge built-in from two accomplished pivotal research in sufferers with Menkes illness handled with CUTX-101, copper histidinate (CuHis). In each pre-specified major and secondary efficacy analyses, remedy with CUTX-101 demonstrated a considerably higher median general survival (OS) in comparison with untreated historic management sufferers. These knowledge shall be offered as a digital poster on the 2021 American Academy of Pediatrics Nationwide Convention & Exhibition. Extra info on the poster is listed beneath:

Digital Poster Title: Copper Histidinate Remedy for Menkes Illness (Kinky Hair Syndrome)
Presentation Date: Friday, October 08, 2021
Session: (VH1410) Part on Advances in Therapeutics and Expertise Program
Authors: Stephen G. Kaler, M.D., M.P.H., Shama Munim, M.S., Michael Chen, Ph.D., Robert Niecestro, Ph.D., Lung S. Yam, M.D., Ph.D.

“There’s a important unmet want for an authorized remedy for sufferers with Menkes illness. These optimistic knowledge exhibit the potential of CUTX-101 to be an efficient remedy for sufferers with this devastating illness. We stay up for working with the U.S. Meals and Drug Administration (“FDA”) to start our rolling submission of a brand new drug software (“NDA”) for CUTX-101 within the fourth quarter of this yr,” mentioned Lung S. Yam, M.D., Ph.D., President and Chief Government Officer of Cyprium.

In two accomplished open-label, single-arm, single-site research, 129 sufferers with Menkes illness have been handled with CUTX-101 (1450 mcg CUTX-101, equal to 250 mcg elemental copper) administered subcutaneously twice each day till 12 months of age, and as soon as each day thereafter, for a complete length of as much as three years.  Sixty-six sufferers born after 1999 and with extreme loss-of-function ATP7A mutations from these two research have been mixed and categorized into an Early Remedy cohort (CuHis-ET; remedy initiated inside 4 weeks of start, corrected for prematurity, n=31) and a Late Remedy cohort (CuHis-LT; remedy initiated after 4 weeks of start, n=35). A historic management cohort of 18 Menkes illness sufferers who had not been handled with CUTX-101 have been enrolled (together with 18 in historic control-early remedy cohort (HC-ET); 17 of whom have been additionally included in historic control-late remedy (HC-LT)). Efficacy of CUTX-101 was assessed by evaluating CuHis-ET to untreated HC-ET, and CuHis-LT to untreated HC-LT, utilizing OS as the first and secondary efficacy endpoints, respectively.

The first efficacy endpoint evaluating CuHis-ET to HC-ET and the secondary efficacy endpoint evaluating CuHis-LT to HC-LT have been each met. General, a 79% discount in threat of loss of life was noticed in CuHis-ET sufferers in contrast with HC-ET sufferers and median OS was 177.1 and 16.1 months, respectively with a hazard ratio (HR) of (95% CI) = 0.208 (0.094, 0.463) p

Medical profit was higher for sufferers who have been handled inside 4 weeks of start with CUTX-101, emphasizing the significance of early identification, together with new child screening and immediate initiation of remedy. A new child screening take a look at for Menkes illness is at the moment in growth.

CUTX-101 was proven to be nicely tolerated. In CuHis-ET and CuHis-LT cohorts, the most typical remedy emergent adversarial occasions have been pneumonia (30.3%), seizures (21.2%), dehydration (18.2%), failure to thrive (16.7%), and respiratory misery (15.2%) and no affected person discontinued resulting from an adversarial occasion thought-about associated to remedy.

Stephen G. Kaler, M.D., M.P.H., a physician-scientist within the Heart for Gene Remedy within the Abigail Wexner Analysis Institute at Nationwide Kids’s Hospital, is Principal Investigator of the medical research and can be a professor of Pediatrics and Genetics at The Ohio State College Faculty of Medication.

“We’re grateful to the sufferers and their households who’ve participated within the medical research through the years.  We’d additionally prefer to thank Dr. Kaler for his ongoing efforts and dedication to enhancing therapies for this devastating pediatric illness. We commend the Cyprium group who has labored diligently to advance the CUTX-101 program and can stay steadfast and centered on bringing this remedy to sufferers,” mentioned Dr. Yam.

Cyprium has partnered with Sentynl Therapeutics, Inc., a U.S.-based specialty pharmaceutical firm owned by the Zydus Group, to convey CUTX-101 to market. Cyprium will retain growth duty of CUTX-101 by way of approval of the NDA by the FDA, and Sentynl shall be answerable for commercialization of CUTX-101 in addition to progressing new child screening actions.

“We’re inspired by the medical knowledge offered on the 2021 American Academy of Pediatrics Nationwide Convention & Exhibition and are excited to proceed to help Cyprium’s efforts to advance CUTX-101 by way of NDA approval as we parallel important efforts in Illness Consciousness training and New child Screening. These developments help the group’s ongoing endeavor to handle unmet healthcare wants,” mentioned Matthew Heck, President and Chief Government Officer of Sentynl.

About Menkes Illness 
Menkes illness is a uncommon X-linked recessive pediatric illness brought on by gene mutations of copper transporter ATP7A. The minimal start prevalence for Menkes illness is believed to be 1 in 34,810 reside male births, and probably as excessive as 1 in 8,664 reside male births, primarily based on current genome-based ascertainment (Kaler SG, Ferreira CR, Yam LS. Estimated start prevalence of Menkes illness and ATP7A-related issues primarily based on the Genome Aggregation Database (gnomAD). Molecular Genetics and Metabolism Stories 2020 June 5;24:100602). The situation is characterised by distinctive medical options, together with sparse and depigmented hair (“kinky hair”), connective tissue issues, and extreme neurological signs reminiscent of seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Mortality is excessive in untreated Menkes illness, with many sufferers dying earlier than the age of three years outdated. Milder variations of ATP7A mutations are related to different circumstances, together with Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Presently, there is no such thing as a FDA-approved remedy for Menkes illness and its variants.

About CUTX-101 (Copper Histidinate)
CUTX-101 is in medical growth to deal with sufferers with Menkes illness. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured underneath present good manufacturing observe (“cGMP”) and physiological pH. In a Section 1/2 medical trial performed by Stephen G. Kaler, M.D., M.P.H., on the Nationwide Institutes of Well being (“NIH”), early remedy of sufferers with Menkes illness with CUTX-101 led to an enchancment in neurodevelopmental outcomes and survival. In August 2020, Cyprium reported optimistic topline medical efficacy outcomes for CUTX-101, demonstrating statistically important enchancment in general survival for Menkes illness topics who obtained early remedy (ET) with CUTX-101, in comparison with an untreated historic management (HC) cohort, with an almost 80% discount within the threat of loss of life. A Cyprium-sponsored expanded access protocol for sufferers with Menkes illness is ongoing at a number of U.S. medical facilities.

About Cyprium Therapeutics
Cyprium Therapeutics, Inc. (“Cyprium”) is targeted on the event of novel therapies for the remedy of Menkes illness and associated copper metabolism issues. In March 2017, Cyprium entered right into a Cooperative Analysis and Growth Settlement (“CRADA”) with the Eunice Kennedy Shriver Nationwide Institute of Little one Well being and Human Growth (“NICHD”), a part of the NIH, to advance the medical growth of CUTX-101 (Copper Histidinate injection) for the remedy of Menkes illness. As well as, Cyprium and NICHD entered right into a worldwide, unique license settlement to develop and commercialize adeno-associated virus (AAV)-based gene remedy, known as AAV-ATP7A, to ship working copies of the copper transporter that’s faulty in sufferers with Menkes illness, and for use together with CUTX-101. CUTX-101 was granted FDA Breakthrough Remedy, Quick Observe and Uncommon Pediatric Illness Designations, and each CUTX-101 and AAV-ATP7A have obtained FDA Orphan Drug Designation beforehand. Moreover, the European Medicines Company beforehand granted Orphan Drug Designation to CUTX-101. Cyprium was based by Fortress Biotech, Inc. (Nasdaq: FBIO) and relies in New York Metropolis. For extra info, go to www.cypriumtx.com.

About Fortress Biotech
Fortress Biotech, Inc. (“Fortress”) is an revolutionary biopharmaceutical firm that was ranked in Deloitte’s 2019 and 2020 Expertise Quick 500™, annual rankings of the fastest-growing North American corporations within the know-how, media, telecommunications, life sciences and power tech sectors, primarily based on percentages of fiscal yr income development over three-year intervals. Fortress is targeted on buying, growing and commercializing high-potential marketed and development-stage medicine and drug candidates. The corporate has seven marketed prescription pharmaceutical merchandise and over 25 packages in growth at Fortress, at its majority-owned and majority-controlled companions and at companions it based and during which it holds important minority possession positions. Such product candidates span six large-market areas, together with oncology, uncommon illnesses and gene remedy, which permit it to create worth for shareholders. Fortress advances its diversified pipeline by way of a streamlined working construction that fosters environment friendly drug growth. The Fortress mannequin is pushed by a world-class enterprise growth group that’s centered on leveraging its important biopharmaceutical trade experience to additional broaden the corporate’s portfolio of product alternatives. Fortress has established partnerships with among the world’s main educational analysis establishments and biopharmaceutical corporations to maximise every alternative to its full potential, together with Alexion Prescribed drugs, Inc., AstraZeneca, Metropolis of Hope, Fred Hutchinson Most cancers Analysis Heart, St. Jude Kids’s Analysis Hospital, Nationwide Kids’s Hospital and Sentynl Therapeutics, Inc.  For extra info, go to www.fortressbiotech.com.

About Sentynl Therapeutics
Sentynl Therapeutics is a San Diego-based commercial-stage specialty pharma firm that focuses on buying, growing and launching distinctive merchandise. The corporate was acquired by the Zydus Group in 2017. Sentynl’s extremely skilled and educated administration group has beforehand constructed a number of profitable pharmaceutical corporations. With a concentrate on commercialization, Sentynl appears to supply efficient and nicely differentiated merchandise throughout a broad spectrum of therapeutic areas. Sentynl is dedicated to the very best moral requirements and compliance with all relevant legal guidelines, laws, and trade pointers.  For extra info, go to www.sentynl.com.

About Zydus
Zydus is an revolutionary, international pharmaceutical firm that discovers, develops, manufactures and markets a broad vary of healthcare therapies, together with small molecule medicine, biologic therapeutics and vaccines. The group employs over 23,000 individuals worldwide, together with 1,400 scientists engaged in R & D, and is devoted to creating more healthy communities globally. For extra info, go to www.zyduscadila.com.

Ahead-Trying Statements
This press launch could include “forward-looking statements” inside the that means of Part 27A of the Securities Act of 1933 and Part 21E of the Securities Trade Act of 1934, as amended. As used beneath and all through this press launch, the phrases “we”, “us” and “our” could confer with Fortress individually or along with a number of accomplice corporations, as dictated by context. Such statements embrace, however should not restricted to, any statements referring to our development technique and product growth packages and some other statements that aren’t historic information. Ahead-looking statements are primarily based on administration’s present expectations and are topic to dangers and uncertainties that would negatively have an effect on our enterprise, working outcomes, monetary situation and inventory value. Elements that would trigger precise outcomes to vary materially from these at the moment anticipated embrace: dangers referring to our development technique; our capacity to acquire, carry out underneath and keep financing and strategic agreements and relationships; dangers referring to the outcomes of analysis and growth actions; uncertainties referring to preclinical and medical testing; dangers referring to the timing of beginning and finishing medical trials; our dependence on third-party suppliers; dangers referring to the COVID-19 outbreak and its potential affect on our staff’ and consultants’ capacity to finish work in a well timed method and on our capacity to acquire extra financing on favorable phrases or in any respect;  our capacity to draw, combine and retain key personnel; the early stage of merchandise underneath growth; our want for substantial extra funds; authorities regulation; patent and mental property issues; competitors; in addition to different dangers described in our SEC filings. We expressly disclaim any obligation or enterprise to launch publicly any updates or revisions to any forward-looking statements contained herein to mirror any change in our expectations or any adjustments in occasions, circumstances or circumstances on which any such assertion relies, besides as could also be required by regulation, and we declare the safety of the protected harbor for forward-looking statements contained within the Non-public Securities Litigation Reform Act of 1995. The data contained herein is meant to be reviewed in its totality, and any stipulations, circumstances or provisos that apply to a given piece of knowledge in a single a part of this press launch must be learn as making use of mutatis mutandis to each different occasion of such info showing herein.

Firm Contacts:
Jaclyn Jaffe and William Begien
Fortress Biotech, Inc.
(781) 652-4500
ir@fortressbiotech.com

Lung Yam, M.D., Ph.D.
Cyprium Therapeutics, Inc.
ir@cypriumtx.com

Michael Hercz
Sentynl Therapeutics, Inc.
ir@sentynl.com

Media Relations Contact:
Tony Plohoros
6 Levels
(908) 591-2839
tplohoros@6degreespr.com

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SOURCE Sentynl Therapeutics



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