Mustang Bio Reports Third Quarter 2020 Financial Results and Recent Corporate Highlights
November 06, 2020
WORCESTER, Mass., Nov. 06, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced financial results and recent corporate highlights for the third quarter ended September 30, 2020.
“Mustang had an eventful third quarter on the regulatory front, as the U.S. Food and Drug Administration (“FDA”) granted Rare Pediatric and Orphan Drug Designations to both of our gene therapy product candidates for the treatment of X-linked severe combined immunodeficiency (“XSCID”), MB-107, for newly diagnosed infants, and MB-207, for patients previously treated with hematopoietic stem cell transplantation (“HSCT”) and for whom re-treatment is indicated,” said Manuel Litchman, M.D., President and Chief Executive Officer of Mustang.
Dr. Litchman continued, “We are also pleased to report progress across our CAR T cell therapy programs during the third quarter and subsequent period. Most importantly, as recently reported, we have observed compelling efficacy without CAR T related toxicity in the first 4 non-Hodgkin lymphoma patients treated with MB-106, a CD20-targeted CAR T cell therapy, following a major revision in the cell manufacturing process at the Fred Hutch Cancer Research Center (“Fred Hutch”). We also dosed the first patient in a Phase 1/2 clinical trial of MB-102, a CD123-targeted CAR T cell therapy, under our own Investigational New Drug (“IND”) application for relapsed or refractory blastic plasmacytoid dendritic cell neoplasm, acute myeloid leukemia and high-risk myelodysplastic syndrome. Finally, we are encouraged by the significant response to MB-105, a prostate stem cell antigen (“PSCA”)-targeted CAR T therapy, reported by City of Hope in a 73-year-old patient with PSCA-positive metastatic castrate-resistant prostate cancer. This patient, who had failed eight prior therapies, experienced a 94 percent reduction in prostate-specific antigen, with a near complete reduction of measurable soft tissue metastasis by computerized tomography and improvement in bone metastases by magnetic resonance imaging. We look forward to achieving additional milestones in the coming months, as we expect to disclose additional promising clinical data from our MB-106 program at the American Society of Hematology (“ASH”) Annual Meeting next month. In addition, next quarter we expect to enroll the first patient on our pivotal MB-107 lentiviral gene therapy trial for newly diagnosed infants with XSCID and to file an IND for our pivotal MB-207 lentiviral gene therapy trial for previously transplanted patients with XSCID.”
Recent Corporate Highlights:
In August 2020, Mustang announced that the FDA granted Rare Pediatric Disease Designation to MB-107, a lentiviral gene therapy for the treatment of XSCID, also known as bubble boy disease, in newly diagnosed infants and to MB-207, a lentiviral gene therapy for the treatment of patients with XSCID who have been previously treated with HSCT and for whom re-treatment is indicated.
In September 2020, the FDA granted Orphan Drug Designation to MB-107 for the treatment of XSCID in newly diagnosed infants and to MB-207 for the treatment of patients with XSCID who have been previously treated with HSCT and for whom re-treatment is indicated.
In October 2020, Mustang announced that the first patient had been dosed in a company-sponsored, open label, multicenter Phase 1/2 clinical trial to evaluate the safety and efficacy of MB-102 (CD123-targeted CAR T cell therapy) in patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm (BPDCN), acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (hrMDS).
In October 2020, Mustang licensed LentiBOOST™ technology from SIRION Biotech GmbH for the development of MB-207.
In October 2020, City of Hope presented initial Phase 1 data on MB-105, a PSCA-targeted CAR T administered systemically to patients with PSCA-positive mCRPC, at the virtual 27th Annual Prostate Cancer Foundation Scientific Retreat.
Earlier this month, Mustang announced that interim Phase 1/2 data on MB-106, a CD20-targeted, autologous CAR T cell therapy for patients with relapsed or refractory B-cell non-Hodgkin lymphomas, were selected for a poster presentation at the 62nd ASH Annual Meeting. A link to the abstract can be found here.
As of September 30, 2020, the company’s cash, cash equivalents and restricted cash totaled $76.3 million, compared to $86.4 million as of June 30, 2020, and $62.4 million as of December 31, 2019.
Research and development expenses were $8.0 million for the third quarter of 2020. This compares to $7.3 million for the third quarter of 2019. Non-cash, stock-based compensation expenses included in research and development were $0.3 million for the third quarter of 2020, compared to $0.7 million for the third quarter of 2019.
Research and development expenses from license acquisitions totaled $0.3 million for the third quarter of 2020, compared to $0.7 million for the third quarter of 2019.
General and administrative expenses were $2.2 million for the third quarter of 2020. This compares to $2.0 million for the third quarter of 2019. Non-cash, stock-based compensation expenses included in general and administrative expenses were $0.3 million for the third quarter of 2020, compared to $0.4 million for the third quarter of 2019.
Net loss attributable to common stockholders was $13.0 million, or $0.23 per share, for the third quarter of 2020, compared to a net loss attributable to common stockholders of $10.2 million, or $0.25 per share, for the third quarter of 2019.
About Mustang Bio
Mustang Bio, Inc. is a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases. Mustang aims to acquire rights to these technologies by licensing or otherwise acquiring an ownership interest, to fund research and development, and to outlicense or bring the technologies to market. Mustang has partnered with top medical institutions to advance the development of CAR T therapies across multiple cancers, as well as a lentiviral gene therapy for XSCID. Mustang is registered under the Securities Exchange Act of 1934, as amended, and files periodic reports with the U.S. Securities and Exchange Commission (“SEC”). Mustang was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit www.mustangbio.com.
It still has a few strong buy ratings. There hasn't been any follow up regarding the Tramadol letter? I would of assumed we would of heard something by now but nothing, Hopefully this movement up today is a positive sign of things to come??
Not sure but looking at the chart, and it consolidating here a bit, any vood/ positive news could send it north, risk/reward starring you in the face right here. Jmo, but watching
Ok, my avg is 3.98 so not too far off, I'll wait and see how she does
Its back...im out. Too scary
No idea...my trade went through now I cant trade. Uhhh maybe oops
Is it halted? Cant trade
190 shares at 3.98 for starters, Boom!
What up? Its another Randall Kirk stock, I follow them all. They all seem to have nice moves here and there.
Fortress Biotech Announces Initiation of Phase 3 Studies of CAEL-101 in AL Amyloidosis
September 14, 2020
- Caelum Biosciences, a company founded by Fortress, is collaborating with Alexion Pharmaceuticals on the studies
- Phase 2 study met primary objective, supporting initiation of two parallel Phase 3 studies that will enroll ~370 AL amyloidosis patients
- Positive long-term Phase 1a/1b data presented at the International Symposium on Amyloidosis (ISA) 2020 demonstrate prolonged overall survival (78 percent at 37 months) and durable organ response
NEW YORK, Sept. 14, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative revenue-generating company focused on acquiring, developing and commercializing or monetizing promising biopharmaceutical products and product candidates cost-effectively, today announced that Caelum Biosciences, Inc. (“Caelum”), in collaboration with Alexion Pharmaceuticals, Inc. (“Alexion”), initiated the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101, a first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in AL amyloidosis.
In 2019, Caelum, a company founded by Fortress, entered into a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 CAEL-101 data. Fortress maintains a 40 percent ownership in Caelum’s issued and outstanding stock and is eligible to receive approximately 43 percent of the proceeds from an Alexion acquisition option exercise.
“We are pleased that CAEL-101 has progressed into Phase 3 development,” said Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer of Fortress. “CAEL-101 has the potential to fill the urgent need for new treatment options for patients with AL amyloidosis, as the data thus far suggest it may improve organ function and overall survival. The partnership between Caelum and Alexion represents an exciting opportunity to help patients and create value for our shareholders. We look forward to the continued late-stage development of this important therapy.”
The CARES clinical program includes two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease and one in patients with Mayo stage IIIb disease – and will collectively enroll approximately 370 patients globally. Enrollment is underway in both studies. The primary objective of the clinical program is to assess overall survival.
AL amyloidosis, also called primary amyloidosis, is a rare disease that occurs when an abnormal protein called amyloid builds up in organs and interferes with their normal function. It affects many organs, including the heart and kidneys, causes significant damage and impairs organ function. Median survival in patients with AL amyloidosis that has affected the heart is less than a year following diagnosis.
About the CARES Phase 3 Clinical Program
The CARES clinical program consists of two parallel double-blind, randomized, event-driven global Phase 3 studies, which are evaluating the efficacy and safety of CAEL-101 in AL amyloidosis patients who are newly diagnosed and naïve to standard of care (SoC) treatment (cyclophosphamide-bortezomib-dexamethasone (CyBorD) chemotherapy). One study is enrolling approximately 260 patients with Mayo stage IIIa disease and one study is enrolling approximately 110 patients with Mayo stage IIIb disease. The studies will be conducted at approximately 70 sites across North America, the United Kingdom, Europe, Israel, Japan, and Australia.
In each study, participants are being randomized in a 2:1 ratio to receive either CAEL-101 plus SoC or placebo plus SoC once weekly for four weeks. This will be followed by a maintenance dose administered every two weeks until the last patient enrolled completes at least 50 weeks of treatment. Patients will continue follow-up visits every 12 weeks.
The primary study objectives are overall survival and the safety and tolerability of CAEL-101. Key secondary objectives will assess functional improvement in the six-minute walk test (6MWT), quality of life measures (Kansas City Cardiomyopathy Questionnaire Overall Score & Short Form 36 version 2 Physical Component Score) and cardiac improvement (Global Longitudinal Strain (GLS)).
Phase 2 Study Results
The Phase 2 open-label dose escalation study was conducted to investigate higher doses of CAEL-101 than had been evaluated in Phase 1 studies with a primary objective to identify the best dose to advance into Phase 3 development. The study evaluated the safety and tolerability of CAEL-101 in 13 AL amyloidosis patients at three study sites who received up to 1000 mg/m2 of CAEL-101 (two times the Phase 1 dose) administered in combination with SoC treatment. The study met its primary objectives, supporting the safety and tolerability of CAEL-101 and the selection of the 1000 mg/m2 dose for the Phase 3 study.
Phase 1a/1b Long-Term Follow-Up Results Presented at International Symposium on Amyloidosis 2020
As previously reported, the Phase 1a/1b study of CAEL-101 was the first clinical trial to demonstrate improvement in cardiac function via GLS after treatment with an amyloid fibril targeted therapy in AL amyloidosis patients with amyloid cardiac involvement. New long-term follow-up data from the Phase 1a/1b study will be presented at the virtual International Symposium on Amyloidosis (ISA), September 14 to 18, 2020, in the poster titled, “Long term follow-up of patients with AL amyloidosis treated on a phase 1 study of Anti-Amyloid Monoclonal Antibody CAEL-101” (Abstract #342, Divaya Bhutani, M.D., et. al, Columbia University Medical Center). These data demonstrate 78 percent survival (15/19) at a median follow-up of more than three years (37 months) in AL amyloidosis patients treated with CAEL-101 as well as durable organ response among evaluable patients, further supporting the advancement of CAEL-101 into Phase 3 development.
CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicines Agency as a therapy for patients with AL amyloidosis.
About AL Amyloidosis
AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.
AL amyloidosis is a rare disease but is the most common form of amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.
About Caelum Biosciences
Caelum Biosciences, Inc. (“Caelum”) is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum’s lead asset, CAEL-101, is a novel antibody for the treatment of patients with amyloid light chain (“AL”) amyloidosis. In 2019, Caelum entered a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company based on Phase 3 CAEL-101 data. Caelum was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit www.caelumbio.com.
About Fortress Biotech
Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company that was recently ranked number 10 in Deloitte’s 2019 Technology Fast 500™, an annual ranking of the fastest-growing North American companies in the technology, media, telecommunications, life sciences and energy tech sectors, based on percentage of fiscal year revenue growth over a three-year period. Fortress is focused on acquiring, developing and commercializing high-potential marketed and development-stage drugs and drug candidates. The company has five marketed prescription pharmaceutical products and over 25 programs in development at Fortress, at its majority-owned and majority-controlled partners and at partners it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is driven by a world-class business development team that is focused on leveraging its significant biopharmaceutical industry expertise to further expand the company’s portfolio of product opportunities. Fortress has established partnerships with some of the world’s leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including Alexion Pharmaceuticals, Inc., AstraZeneca, City of Hope, Fred Hutchinson Cancer Research Center, InvaGen Pharmaceuticals Inc. (a subsidiary of Cipla Limited), St. Jude Children’s Research Hospital and Nationwide Children’s Hospital. For more information, visit www.fortressbiotech.com.
And We Have Another Double. Frontier Bio (FBIO).
Frontier Bio (FBIO) $3.90. We Have Another Double!
American BriVision $4.00. (ABVC). Wait, What? Yet Another Double.
Aphex BioCleanse (SNST) $1.80. Wait, What? Seeking FDA Approval on a Sanitizer.
The Alkaline Water Company $1.50. (WTER) vs Celcius Holdings $20. (CELH). Must Read.
Bavarian “Cure for Common Cold” Nordic $11.50 (BVNRY) up 10%. Has to Start Somewhere.
Video: What to Look for in Management.
Mustang Bio Reports Second Quarter 2020 Financial Results and Recent Corporate Highlights
August 10, 2020
WORCESTER, Mass., Aug. 10, 2020 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang”) (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, today announced financial results and recent corporate highlights for the second quarter ended June 30, 2020.
Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, “Mustang made progress on multiple fronts during the second quarter of 2020. In June, we raised gross proceeds of approximately $37.2 million in an underwritten public offering of common stock. On the clinical front, we continued to advance our robust pipeline of gene and CAR T cell therapy programs. Notably, we began enrolling patients in a Phase 1/2 clinical trial of MB-102 (CD123-targeted CAR T cell therapy) under our own Investigational New Drug (“IND”) application for relapsed or refractory blastic plasmacytoid dendritic cell neoplasm, acute myeloid leukemia and high-risk myelodysplastic syndrome. We look forward to continued progress throughout the second half of this year, as we expect to initiate our pivotal MB-107 and MB-207 lentiviral gene therapy programs for newly diagnosed infants and previously transplanted patients with X-linked severe combined immunodeficiency (“XSCID”) and to disclose additional data from our MB-106 CD20-targeted CAR T cell therapy program.”
Recent Corporate Highlights:
In June 2020, Mustang raised gross proceeds of approximately $37.2 million in an underwritten public offering of common stock, including the exercise of the underwriter’s option.
In May 2020, City of Hope presented two posters pertaining to MB-104, an innovative CS1 CAR T cell therapy, at the virtual 23rd Annual Meeting of the American Society of Gene & Cell Therapy (“ASGCT”).
Also in May 2020, Mustang submitted an IND application to the U.S. Food and Drug Administration (“FDA”) to initiate a multi-center Phase 2 clinical trial of MB-107 in newly diagnosed infants with XSCID who are under the age of two. The trial is expected to enroll 10 patients who, together with 15 patients enrolled in the current multi-center trial led by St. Jude Children’s Research Hospital, will be compared with 25 matched historical control patients who have undergone hematopoietic stem cell transplant (“HSCT”). The primary efficacy endpoint will be event-free survival. The initiation of this trial is currently on hold pending CMC clearance by the FDA, which is expected in early Q4 2020. Mustang is targeting topline data from this trial in the second half of 2022.
Mustang expects to file an IND in the fourth quarter of 2020 for a registrational multi-center Phase 2 clinical trial of its lentiviral gene therapy in previously transplanted XSCID patients. This product will be designated MB-207. Mustang anticipates enrolling 20 patients and comparing them to matched historical control patients who have undergone a second HSCT. The company is targeting topline data from this trial in the second half of 2022.
In the ongoing Phase 1 trial of MB-105, a PSCA-directed CAR T administered systemically to patients with PSCA-positive castration resistant prostate cancer, at City of Hope, the first patient to receive the therapy following a standard CAR T conditioning regimen experienced a significant reduction in his prostate-specific antigen (“PSA”) at day 28. This PSA response was associated with radiographic improvement of the patient’s metastatic disease.
In April 2020, Mustang announced that the European Medicines Agency (“EMA”) granted Advanced Therapy Medicinal Product (“ATMP”) classification to MB-107 for the treatment of XSCID.
As of June 30, 2020, the company’s cash, cash equivalents and restricted cash totaled $86.4 million, compared to $56.8 million as of March 31, 2020, and $62.4 million as of December 31, 2019.
Research and development expenses were $9.8 million for the second quarter of 2020. This compares to $6.8 million for the second quarter of 2019. Non-cash, stock-based compensation expenses included in research and development were $0.4 million for the second quarter of 2020, compared to $0.3 million for the second quarter of 2019.
Research and development expenses from license acquisitions totaled $1.3 million for the second quarter of 2020, compared to $0.2 million for the second quarter of 2019.
General and administrative expenses were $3.0 million for the second quarter of 2020. This compares to $3.2 million for the second quarter of 2019. Non-cash, stock-based compensation expenses included in general and administrative expenses were $1.5 million for the second quarter of 2020, compared to $1.6 million for the second quarter of 2019.
Net loss attributable to common stockholders was $14.6 million, or $0.32 per share, for the second quarter of 2020, compared to a net loss attributable to common stockholders of $10.4 million, or $0.29 per share, for the second quarter of 2019.
Fortress Biotech Announces Positive Opinion on Orphan Drug Designation Received from the European Medicines Agency for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease
Rolling submission of New Drug Application to the FDA for CUTX-101 on track to begin in the fourth quarter of 2020
NEW YORK, July 31, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”), an innovative biopharmaceutical company focused on acquiring, developing and commercializing high-potential marketed pharmaceutical products and development-stage pharmaceutical product candidates, today announced that the European Medicines Agency (“EMA”) Committee for Orphan Medicinal Products issued a positive opinion on Cyprium Therapeutics’ (“Cyprium”) application for Orphan Drug Designation for Copper Histidinate, also referred to as CUTX-101, a potential treatment for Menkes disease. Menkes disease is an often lethal, if untreated, X-linked recessive disorder of copper metabolism caused by mutations in ATP7A, an evolutionarily conserved copper-transporting ATPase. The U.S. Food and Drug Administration (“FDA”) previously granted Orphan Drug, Fast Track and Rare Pediatric Disease Designations to CUTX-101 for the treatment of Menkes disease.
Lung S. Yam, M.D., Ph.D., President and Chief Executive Officer of Cyprium, said, “The positive opinion for Orphan Drug Designation from the EMA is an important milestone in bringing a much-needed potential therapy to patients with Menkes disease, a devastating pediatric disease with limited treatment options. We look forward to working closely with the EMA and continuing to progress CUTX-101 for children in need. To this end, we remain on track to begin a rolling submission of a New Drug Application to the FDA for CUTX-101 in the U.S. in the fourth quarter of this year.”
Orphan Drug Designation in the European Union (“EU”) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products. To qualify, an investigational medicine must be intended to treat a seriously debilitating or life-threatening condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient non-clinical or clinical data to suggest the investigational medicine may produce clinically relevant outcomes. EMA orphan drug designation provides companies with certain benefits and incentives, including clinical protocol assistance, differentiated evaluation procedures for Health Technology Assessments in certain countries, access to a centralized marketing authorization procedure valid in all EU member states, reduced regulatory fees and 10 years of market exclusivity.
CambPharma Solutions (CY) Limited submitted the Orphan Drug Designation application on behalf of Cyprium, as its agent in the EU.
About Menkes Disease and Related Copper Metabolism Disorders
Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 males, but could potentially be as high as 1 in 8,664 live male births, higher than previously recognized. Biochemically, Menkes patients have low levels of copper in their blood and brain, as well as abnormal levels of certain neurochemicals. Definitive diagnosis is typically made by sequencing the ATP7A gene. The condition is characterized by distinctive clinical features, including sparse and depigmented hair (“kinky hair”), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, and failure to thrive. Mortality is high in untreated Menkes disease, with many patients dying before the age of three. Milder versions of ATP7A mutations are associated with other conditions, including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants.
About CUTX-101 (Copper Histidinate)
CUTX-101 is in clinical development to treat patients with Menkes disease by replenishing Copper Histidinate, restoring copper homeostasis, and maintaining serum copper levels in the normal age appropriate range. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under current good manufacturing practice (“cGMP”) that is intended to improve tolerability due to physiological pH and to bypass the oral absorption of copper, which is impaired in patients with Menkes disease. In a Phase 1/2 clinical trial conducted by Stephen G. Kaler, M.D., M.P.H., at the National Institutes of Health (“NIH”), early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. A Phase 3 trial of CUTX-101 in patients with Menkes disease also led by Dr. Kaler has completed enrollment. A Cyprium-sponsored expanded access protocol for Menkes disease patients is ongoing.
About Cyprium Therapeutics
Cyprium Therapeutics, Inc. (“Cyprium”) is focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders. In March 2017, Cyprium entered into a Cooperative Research and Development Agreement (“CRADA”) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (“NICHD”), part of the NIH, to advance the clinical development of CUTX-101 (Copper Histidinate injection) for the treatment of Menkes disease. In addition, Cyprium and NICHD entered into a worldwide, exclusive license agreement to develop and commercialize adeno-associated virus (AAV)-based gene therapy, called AAV-ATP7A, to deliver working copies of the copper transporter that is defective in Menkes patients, and to be used in combination with CUTX-101. CUTX-101 was granted FDA Fast Track and Rare Pediatric Disease Designations, and both CUTX-101 and AAV-ATP7A have received FDA Orphan Drug Designation previously. Cyprium was founded by Fortress Biotech, Inc. (Nasdaq: FBIO) and is based in New York City. For more information, visit www.cypriumtx.com.
Avenue Therapeutics Announces Presentation of IV Tramadol E-Posters
Download as PDF
April 23, 2020
NEW YORK, April 23, 2020 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (NASDAQ: ATXI) (“Avenue”), a company focused on the development of intravenous (“IV”) tramadol for the U.S. market, today announced that two e-posters highlighting efficacy and safety results from its Phase 3 program are available for online viewing from the cancelled Annual Regional Anesthesiology and Acute Pain Medicine Meeting hosted by the American Society of Regional Anesthesia and Pain Medicine (“ASRA”). The meeting was originally scheduled to take place April 23-25, 2020 in San Francisco, CA and was cancelled due to COVID-19 concerns.
The e-poster (816) titled “Intravenous Tramadol is Effective in Management of Postoperative Pain Following Abdominoplasty: A 3-arm Randomized Controlled Trial” presents data from the Phase 3 abdominoplasty study and can be found here.
This Phase 3, multicenter, double-blind, placebo and active controlled trial evaluated the efficacy and safety of IV tramadol in 370 patients following abdominoplasty surgery. Patients were randomized to a postoperative regimen of IV tramadol 50 mg, placebo or IV morphine 4 mg. The primary endpoint of the study assessed the analgesic efficacy of IV tramadol compared to placebo as measured by SPID24 (sum of pain intensity differences through 24 hours post first dose). The key secondary endpoints included SPID48 (sum of pain intensity differences through 48 hours post first dose), total consumption of rescue medicine and Patient Global Assessment. IV tramadol 50 mg was statistically significantly superior to placebo for the primary efficacy endpoint and all three key secondary efficacy endpoints. In addition, IV tramadol demonstrated a similar efficacy profile to that of IV morphine 4 mg, a standard-of-care active comparator in this study. Topline results from this study were announced in June 2019.
The e-poster (1001) titled “IV tramadol – A New Treatment Option for Management of Post-Operative Pain: A Safety Trial Including Various Types of Surgery”, presents data from the Phase 3 safety study and can be found here.
The Phase 3 safety study was a single-arm open label study that enrolled patients undergoing a range of surgical procedures including both orthopedic and soft tissue surgeries. IV tramadol 50 mg was administered at Hours 0, 2, 4, and every 4 hours thereafter for up to 7 days of treatment. Patients were allowed to use non-opioid medications per treating physicians’ discretion if additional pain relief was needed. While efficacy was not a primary objective of the study, patient satisfaction with treatment was measured with Patient Global Assessment.
IV tramadol 50 mg was well tolerated in this real-world trial, with only 4% of patients discontinuing for adverse events. The most commonly observed adverse events were nausea and vomiting, which is consistent with known tramadol pharmacology. At the end of treatment, 95% of patients reported that study medication was good, very good, or excellent for controlling pain. No patients discontinued the study due to a lack of efficacy.
“The results of these two studies indicate that IV tramadol may become a useful option in patients where exposure to conventional opioids is not desired,” said Harold Minkowitz, M.D., an anesthesiologist at Memorial Hermann Memorial City Medical Center in Houston, TX. “IV tramadol, with its dual mechanisms of action, may fill a gap between IV non-opioid medicine and conventional opioids. The availability of IV tramadol as an alternative to conventional opioid analgesics should be a valuable option for U.S. clinicians who treat pain in the hospital setting.”
About Avenue Therapeutics
Avenue Therapeutics is a specialty pharmaceutical company whose mission is to develop IV tramadol, a potential alternative that could reduce the use of conventional opioids, for patients suffering from acute pain in the U.S. Avenue is headquartered in New York City and was founded by Fortress Biotech, Inc. (NASDAQ: FBIO). For more information, visit www.avenuetx.com.