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Celsion Corporation Receives FDA Fast Track Designation for GEN-1 in Advanced Ovarian Cancer

February 23, 2021

by administrator Drug Discovery News Regulatory News

Celsion Corporation (NASDAQ: CLSN), a clinical stage development company focused on DNA based immunotherapy and next generation vaccines, today announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for GEN-1, its DNA-mediated interleukin-12 (IL-12) immunotherapy currently in Phase II development for the treatment of advanced ovarian cancer. GEN-1 was designed using TheraPlas, Celsion’s proprietary, synthetic, non-viral nanoparticle delivery system platform.

Fast Track designation is intended to facilitate the development and expedite the regulatory review of drugs to treat serious conditions and fill an unmet medical need. According to the FDA, a Fast Track Drug must show some advantage over available therapy, including:

Showing superior effectiveness, effect on serious outcomes or improved effect on serious outcomes
Avoiding serious side effects of an available therapy
Decreasing a clinical significant toxicity of an available therapy that is common and causes discontinuation of treatment

“Fast Track designation is an important step in developing GEN-1 for advanced ovarian cancer. Presuming the encouraging data we are generating in early clinical studies continues, this designation supports an expedited path to market,” said Michael H. Tardugno, Celsion’s chairman, president and chief executive officer. “Fast Track allows for more frequent communication with the FDA to discuss development plans and clinical trial design. In addition, should criteria be met, Fast Track-designated drugs are eligible for rolling review, a process whereby the drug’s sponsor can separately submit sections of its New Drug Application to the FDA. They also are eligible for accelerated approval and priority review, under which drugs for serious conditions fulfilling an unmet medical need can be approved based on a surrogate endpoint. We are optimistic that GEN-1 represents a game-changer for women with advanced ovarian cancer who have limited treatment options.”

GEN-1 is the subject of Celsion’s Phase II OVATION 2 Study, which combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).

As Celsion has previously announced, it has shared with the FDA data from the Phase I portion of the Phase I/II OVATION 2 Study that showed successful tumor resections, with seven out of eight patients (88%) in the GEN-1 treatment arm having a complete tumor resection (R0), which indicates a microscopically margin-negative resection in which no gross or microscopic tumor remains in the tumor bed. The NACT-only treatment arm had an R0 resection rate of 50%.

To Read the Complete Article at GlobeNewswire, Click Here

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BioPharma Global is a mission-driven corporation, operating like a not-for-profit, dedicated to using our FDA and EMA regulatory expertise and knowledge of various therapeutic areas to help drug developers advance treatments for the disease communities with a high unmet medical need. If you are a drug developer seeking regulatory support for Orphan Drug designation, Fast Track designation, Breakthrough Therapy designation, other FDA/EMA expedited programs, type A, B (pre-IND, EOPs), or C meeting assistance, or IND filings, the BioPharma Global team can help. Contact us today to arrange a 30-minute introductory call.

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Rare Disease Highlight: Gaucher Disease

February 22, 2021

by administrator Rare Disease Highlight

By Isaiah Manoogian

Gaucher Disease (GD) is a rare disease caused by GBA1 gene mutations. The GBA1 gene encodes for the production of the glucocerebrosidase (GCase) enzyme, which is in the lysosome to digest large materials.¹ Due to mutations disrupting specific pathways in the body, macrophages are transformed into Gaucher cells. The Gaucher cells infiltrate the bone marrow, spleen, liver, and potentially other organs, causing the disease’s main symptoms observed in the three primary types of GD.² Type 1 GD (GD1) is the most common form, with signs and symptoms including, anemia and fatigue, decreased bone density, painful bone crises, and an enlarged liver and spleen. Type 2 GD (GD2) is characterized by GCase accumulating in the brain resulting in severe early life neurological symptoms, such as trouble swallowing. Trouble swallowing leads to significant breathing and feeding difficulties, and even death within the first few years of life. Type 3 GD (GD3) is similar to GD2, but the symptoms develop more gradual; seizures can be a large problem for those suffering GD3. The signs and symptoms of GD2 and GD3 also include those associated with GD1.³Interestingly, the literature reports GBA gene mutations are the most common genetic risk factor for Parkinson’s Disease (PD), establishing a strong association between the two.⁴

After the onset of the first clinical and laboratory signs, GD is typically diagnosed years later using a specific blood test. If GCase levels are low, genetic testing is performed to identify GBA mutations.^2,3 Currently, the main treatment options for GD are enzyme replacement therapy (ERT) and substrate reduction therapy (SRT). Patients receiving these treatments may still have poor outcomes due to the natural progression of the disease. Although new therapeutic developments of enzymes and substrate inhibitors have been made, research must proceed, and monitoring patients is of utmost importance in order to identify progression of GD.²

References

GBA gene – Genetics Home Reference – NIH. (2020). Retrieved July 8, 2020, from https://ghr.nlm.nih.gov/gene/GBA
Stirnemann, J. Ô., Belmatoug, N., Camou, F., Serratrice, C., Froissart, R., Caillaud, C., Levade, T., Astudillo, L., Serratrice, J., Brassier, A., Rose, C., De Villemeur, T. B., & Berger, M. G. (2017). A review of gaucher disease pathophysiology, clinical presentation and treatments. In International Journal of Molecular Sciences (Vol. 18, Issue 2, p. 441). MDPI AG. https://doi.org/10.3390/ijms18020441
Gaucher Disease – NORD (National Organization for Rare Disorders). (2018). Retrieved July 9, 2020, from https://rarediseases.org/rare-diseases/gaucher-disease/
Sidransky, E., Nalls, M. A., Aasly, J. O., Aharon-Peretz, J., Annesi, G., Barbosa, E. R., Bar-Shira, A., Berg, D., Bras, J., Brice, A., Chen, C.-M., Clark, L. N., Condroyer, C., De Marco, E. V., Dürr, A., Eblan, M. J., Fahn, S., Farrer, M. J., Fung, H.-C., … Ziegler, S. G. (2009). Multicenter Analysis of Glucocerebrosidase Mutations in Parkinson’s Disease. New England Journal of Medicine, 361(17), 1651–1661. https://doi.org/10.1056/NEJMoa0901281

Rare Disease Highlight: Duchenne Muscular Dystrophy

February 16, 2021

by administrator Rare Disease Highlight

By Rita Ejiofor

Duchenne Muscular Dystrophy (DMD) is a muscle disorder which causes muscle loss and weakness due to a mutation in the dystrophin gene that is responsible for keeping the muscle cells sturdy. This disease is named after the French neurologist, Guillaume Benjamin Amand Duchenne, who discovered the disease in the 1860’s ¹. This disease predominantly occurs in males affecting approximately 1:50,000 in the US, making its patient population rare ². 70% of DMD cases are due to genetic mutations that are passed down through family generations. Therefore, most males affected receive a mutated copy of the X chromosome from their mother ¹. Symptoms of DMD include: muscle weakness, calf enlargement, scoliosis, breathing issues etc ³. There is currently no cure for DMD. The current standard of therapy is the use of corticosteroids which helps to improve the muscle membrane. However, corticosteroid have unwanted side effects such as weight gain, weakened immune system, behavioral changes, and excessive hair growth. Other therapy options include physical therapy or assistive devices which can only help with symptom management. Thus, patients have very limited options and more therapy is needed ⁴.

References

Diseases – DMD – Causes/Inheritance. (2020, April 7). Muscular Dystrophy Association. https://www.mda.org/disease/duchenne-muscular-dystrophy/causes-inheritance
Yiu, E. M., & Kornberg, A. J. (2015). Duchenne muscular dystrophy. In Journal of Paediatrics and Child Health. https://doi.org/10.1111/jpc.12868
Suthar, R., & Sankhyan, N. (2018). Duchenne Muscular Dystrophy: A Practice Update. In Indian Journal of Pediatrics. https://doi.org/10.1007/s12098-017-2397-y
Reinig, A. M., Mirzaei, S., & Berlau, D. J. (2017). Advances in the Treatment of Duchenne Muscular Dystrophy: New and Emerging Pharmacotherapies. In Pharmacotherapy. https://doi.org/10.1002/phar.1909

European Commission Grants Vico Therapeutics Orphan Drug Designation for VO659, an Investigational Therapy for Spinocerebellar Ataxia

February 16, 2021

by administrator Drug Discovery News Rare Disease News Regulatory News

Vico Therapeutics, a Leiden, the Netherlands, based biotech company focusing on the development of RNA modulating therapies for rare neurological disorders, today announced that the European Commission (EC) has granted orphan drug designation for VO659, Vico’s investigational antisense oligonucleotide therapy for the treatment of Spinocerebellar Ataxia (SCA). The orphan designation was based on a positive opinion from the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP). Vico previously received orphan drug designation for VO659 in Huntington Disease.

“Spinocerebellar Ataxias make up a great part of the so called polyglutamine disorders, debilitating and progressive diseases, leading to significant impairment of mobility, speech, and multiple other daily activities of patients suffering from this condition. So far, there is no treatment which can halt the progression of these diseases,” said Rupert Sandbrink M.D. Ph.D., Chief Medical Officer at Vico.

“Our investigational RNA modulating therapy is designed to lower the mutant protein levels causing these neurodegenerative diseases. We’re pleased to receive orphan drug designation, which recognizes both the unmet medical need for patients living with spinocerebellar ataxias, and the potential of our approach.”

Qualified for orphan drug designation are drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions that impact fewer than 5 in 10,000 patients in the European Union. Orphan drug designation gives developing companies certain benefits, including clinical protocol assistance, reduced regulatory fees, research grants and 10 years of market exclusivity following regulatory approval.

About SCA
Spinocerebellar ataxia’s (SCA) are a group of rare, progressive hereditary genetic disorders that affects the cerebellum, brain stem and spinal cord. More than 30 types of SCAs have been identified to date (SCA1–SCA36), and the most common SCAs (types 1, 2, 3, 6 and 7) are caused by translated CAG trinucleotide repeat expansions that encode elongated polyglutamine (polyQ) stretches in the respective disease proteins. Presence of the elongated polyQ stretch confers pathogenic properties to the resulting protein through a dominant gain-of-function mechanism, resulting in degeneration of specific neuronal subpopulations that differ between the different SCA types. For SCA1 and SCA3 disease manifestation includes ataxia of gait, stance, and limbs, dysarthria, and oculomotor abnormalities. To date, there are no disease-modifying therapies.

To Read the Complete Article at B3C Newswire, Click Here

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BrainQ gets FDA Breakthrough status for its device for reducing disability following stroke

February 16, 2021

by administrator Drug Discovery News Regulatory News

The Breakthrough Device designation, which comes after new trial data, is expected to expedite the device’s path to market.

BrainQ, the Israeli start-up with a therapeutic solution to reduce disability following stroke, announced today that the United States Food and Drug Administration (FDA) has designated its AI-powered technology as a Breakthrough Device. The designation, which was based on BrainQ’s latest randomized controlled clinical trial results for ischemic stroke patients, provides BrainQ with the opportunity to work closely with the FDA to expedite development plans and premarket clearance. Breakthrough status also gives BrainQ access to the new Medicare Coverage of Innovative Technology (MCIT) pathway, allowing for Medicare coverage to be provided concurrently with FDA market authorization.

Stroke is one of the leading causes of disability, affecting 800,000 people every year in the United States alone. In the days and weeks following stroke, the brain attempts to repair damaged neural pathways and develop new ones to restore function, but often with limited success. This results in chronic disability for about 50-70% of survivors.

BrainQ is developing an AI-powered electromagnetic field therapy that aims to enhance recovery and reduce disability after neurological damage caused by stroke. The therapy is based on biological insights retrieved from brainwaves, using proprietary machine learning algorithms that translate into a frequency-tuned low intensity electromagnetic field. BrainQ’s therapy is delivered via a cloud-connected wearable device, the non-invasive BQ System, and is designed for scalable and portable treatment, with the flexibility to be accessed from home.

“We’re excited that the FDA has granted BrainQ a Breakthrough Device Designation,” said BrainQ CEO and Co-founder Yotam Drechsler. “Stroke is a debilitating condition with limited recovery options, creating a huge unmet need in the US. Covid-19 has only made things worse by limiting patients’ access to treatment facilities. FDA Breakthrough Designation is an important milestone in our mission to reduce disability for these patients and treat them in the comfort of their homes.”

To Read the Complete Article at PR Newswire, Click Here

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Forge Biologics Receives FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations for FBX-101 Gene Therapy for Patients with Krabbe Disease

February 16, 2021

by administrator Drug Discovery News Rare Disease News Regulatory News

– The FDA has granted Fast Track Designation, Rare Pediatric Disease Designation, and Orphan Drug Designation to FBX-101 for treatment of patients with Krabbe disease

– FBX-101 is a first-in-human gene therapy utilizing an adeno-associated virus (AAV) to deliver a functioning copy of the GALC gene intraveniously to cells in the central nervous system (CNS) and peripheral organs

– FBX-101 GMP manufacturing is completed and clinical trial enrollment is active

Forge Biologics Inc., a fully integrated clinical stage gene therapy manufacturing and development company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease (RPD) designations to FBX-101 for the treatment of patients with Krabbe disease. Forge is now actively recruiting patients for enrollment in the RESKUE phase 1/2 clinical trial of FBX-101, a novel, first-in-human AAV gene therapy for the disease. FBX-101 is the first intravenious gene therapy program for patients with Krabbe disease and marks a major step forward in building out the company’s hybrid model as a gene therapy manufacturing and development engine.

“FDA’s decision to grant these designations to our first-in-human investigational gene therapy highlights the urgency of developing a treatment for Krabbe patients,” said Timothy J. Miller, Ph.D., CEO, President and Co-Founder of Forge Biologics. “Krabbe is a devastating disease, and it is imperative to develop treatment options like FBX-101 that may address all manifestations of the disease.”

Fast Track Designation is given when the FDA determines that a drug demonstrates the potential to address unmet medical needs for a serious or life-threatening disease or condition. This designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions, and may also allow for priority or rolling review of a company’s Biologics License Application (BLA).

The FDA grants Orphan Drug designation to drugs and biological products intended for the treatment of patients with rare diseases that affect fewer than 200,000 people in the United States. RPD designation is granted by the FDA to encourage treatments for serious or life-threatening diseases primarily affecting children 18 years of age and younger and fewer than 200,000 people in the United States. On December 27, 2020, the Rare Pediatric Disease Priority Review Voucher Program was extended by Congress after it was scheduled to sunset in 2020. Under the newly extended RPD program, if FBX-101 is approved by the FDA, Forge Biologics will qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.

To Read the Complete Article at PR Newswire, Click Here

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Rare Disease Highlight: Vanishing Bone Disease

February 8, 2021

by administrator Rare Disease Highlight

By Isaiah Manoogian

Vanishing Bone Disease (VBD) is an ultra-rare disease, which only about 300 cases have been reported in the literature. However, many cases can go undiagnosed or misdiagnosed, indicating VBD is more prevalent than what is in current literature. VBD is characterized by the increase of vascular structures, such as blood or lymphatic vessels, and osteolysis. Osteolysis is the active process in which osteoclasts (a type of bone cell) absorb and break down bone.¹ The cause of VBD is unknown, possibly due to its ultra-rarity, however few theories exist. Specifically, elevated levels of a certain protein involved in the osteolysis process, malformation of blood and lymphatic vessels, and genetic mutations may play a role in this orphan disease.^2-5 Since VBD is classified as Type IV osteolysis, the bone degradation can occur in any type of bone.⁶ This results in either gradual development of dull aches and weakness, or rapid pain and swelling.¹ Although the VBD prognosis is unpredictable, it is rarely fatal, however, spinal and organ involvement is associated with high morbidity and mortality.^7,8 One of the possible serious complications is lymphatic fluid accumulation (chylothorax), which greatly impedes patients’ breathing ability, possibly resulting in death.⁹

Although no current standard of care exists to treat VBD, three major available options for treatment are bisphosphonates, radiation, and surgery.¹⁰ Bisphosphonates have been successful, due to their antiosteolytic activity, while moderate doses of radiation and surgery are preferred for individuals with large symptomatic lesions and established disabling limitations.^8,11,12 Resection of lesions, and reconstruction with bone grafts and/or prostheses are common surgical procedures.¹³ Despite the available treatment options for VBD, further research is required for the development of more effective interventions.

References

Gorham-Stout Disease – NORD (National Organization for Rare Disorders). (2019). Retrieved June 12, 2020, from https://rarediseases.org/rare-diseases/gorham-stout-disease/
GORHAM, L. W., & STOUT, A. P. (1955). Massive osteolysis (acute spontaneous absorption of bone, phantom bone, disappearing bone); its relation to hemangiomatosis. The Journal of Bone and Joint Surgery. American Volume, 37–A(5), 985–1004. http://www.ncbi.nlm.nih.gov/pubmed/13263344
Devlin, R. D., Bone, H. G., & Roodman, G. D. (1996). Interleukin-6: a potential mediator of the massive osteolysis in patients with Gorham-Stout disease. The Journal of Clinical Endocrinology & Metabolism, 81(5), 1893–1897. https://doi.org/10.1210/jcem.81.5.8626854
Dellinger, M. T., Garg, N., & Olsen, B. R. (2014). Viewpoints on vessels and vanishing bones in Gorham-Stout disease. In Bone (Vol. 63, pp. 47–52). Elsevier Inc. https://doi.org/10.1016/j.bone.2014.02.011
Dickson, G. R., Mollan, R. A. B., & Carr, K. E. (1987). Cytochemical localization of alkaline and acid phosphatase in human vanishing bone disease. Histochemistry, 87(6), 569–572. https://doi.org/10.1007/BF00492472
Ganau, M., Prasad, V., Ligarotti, G. K. I., Syrmos, N. C., & Ellamushi, H. (2018). An overview of the neurosurgical implications, pathophysiology, diagnosis and recent treatment strategies for grade IV idiopathic osteolysis, also known as Gorham-Stout or phantom bone disease. In Hellenic Journal of Nuclear Medicine (Vol. 21, Issue 3, pp. 198–201). P.Ziti and Co. https://doi.org/10.1967/s002449910905
Bruch-Gerharz, D., Gerharz, C. D., Stege, H., Krutmann, J., Pohl, M., Koester, R., & Ruzicka, T. (2007). Cutaneous lymphatic malformations in disappearing bone (Gorham-Stout) disease: A novel clue to the pathogenesis of a rare syndrome. Journal of the American Academy of Dermatology, 56(2 SUPPL.), S21–S25. https://doi.org/10.1016/j.jaad.2006.01.063
Patel, D. V. (2005). Gorham’s disease or massive osteolysis. In Clinical medicine & research (Vol. 3, Issue 2, pp. 65–74). Marshfield Clinic. https://doi.org/10.3121/cmr.3.2.65
Tie, M. L. H., Poland, G. A., & Rosenow, E. C. (1994). Chylothorax in Gorham’s syndrome: A common complication of a rare disease. Chest, 105(1), 208–213. https://doi.org/10.1378/chest.105.1.208
Hu, P., Yuan, X. G., Hu, X. Y., Shen, F. R., & Wang, J. A. (2013). Gorham-Stout syndrome in mainland China: A case series of 67 patients and review of the literature. Journal of Zhejiang University: Science B, 14(8), 729–735. https://doi.org/10.1631/jzus.B1200308
Dunbar, S. F., Rosenberg, A., Mankin, H., Rosenthal, D., & Suit, H. D. (1993). Gorham’s massive osteolysis: The role of radiation therapy and a review of the literature. International Journal of Radiation Oncology, Biology, Physics, 26(3), 491–497. https://doi.org/10.1016/0360-3016(93)90968-2
Hammer, F., Kenn, W., Wesselmann, U., Hofbauer, L. C., Delling, G., Allolio, B., & Arlt, W. (2004). Gorham-Stout Disease-Stabilization During Bisphosphonate Treatment. Journal of Bone and Mineral Research, 20(2), 350–353. https://doi.org/10.1359/JBMR.041113
Kiran, D. N., & Anupama, A. (2011). Vanishing bone disease: A review. Journal of Oral and Maxillofacial Surgery, 69(1), 199–203. https://doi.org/10.1016/j.joms.2010.05.088

FDA grants breakthrough designation to Roche Elecsys GDF-15 assay

February 8, 2021

by administrator Drug Discovery News Regulatory News

The US Food and Drug Administration (FDA) has granted Breakthrough Device Designation to Roche’s Elecsys GDF-15 assay as a companion diagnostic (CDx) in cancer treatment.

The in vitro diagnostic immunoassay is designed for measuring Growth Differentiation Factor-15 (GDF-15) in cachectic adult patients with solid tumours for treatment with Pfizer’s investigational drug PF-06946860.

Cachexia is a metabolic disorder and comorbidity that occurs with various chronic diseases such as cancer, heart failure, chronic obstructive pulmonary disease (COPD), and chronic kidney disease (CKD).

It manifests as marked involuntary body weight loss, muscle atrophy, and reduced appetite, advancing to functional impairment and high death risk.

Cachexia is a common complication of cancer, and affects more than half of cancer patients globally.

As elevated GDF-15 is linked to cachexia in cancer patients, successful cachexia treatment can potentially lead to better cancer treatment.

Roche Diagnostics CEO Thomas Schinecker said: “We are pleased to partner with Pfizer to address this unmet medical need in oncology through strong companion diagnostics.

“The FDA BDD grant for the Elecsys GDF-15 assay shows the importance of these strong partnerships.

To Read the Complete Article at Medical Device Network, Click Here

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Elevar Therapeutics Announces Orphan Drug Designation for Rivoceranib (Apatinib) for the Treatment of Adenoid Cystic Carcinoma (ACC)

February 8, 2021

by administrator Drug Discovery News Rare Disease News Regulatory News

Elevar Therapeutics, Inc. (“Elevar”), a fully integrated biopharmaceutical company built on the promise of elevating treatment experiences and outcomes for patients who have limited or inadequate therapeutic options, today announced that the U.S. Food and Drug Administration (FDA) has granted rivoceranib (apatinib) with orphan drug designation for the treatment of adenoid cystic carcinoma (ACC), a rare form of cancer that most commonly develops in the salivary glands or other regions of the head and neck. Approximately 1,200 new cases of ACC are diagnosed each year in the United States.

The FDA’s Office of Orphan Drug Products grants orphan status to medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the U.S.

“We are pleased by the FDA’s recognition of the critical need to develop treatments for adenoid cystic carcinoma (ACC) given the progressive nature of the disease,” said Alex Kim, chief executive officer of Elevar. “The orphan drug designation provides further momentum for the rivoceranib development program, which we are committed to advancing as quickly as possible for patients in need.”

ACC is considered a slow growing but relentless cancer that is characterized by nerve invasion and metastases. Although good local control is usually achieved by resection of the primary tumor and adjuvant radiation therapy, more than half of patients eventually have recurrent and/or metastatic disease. Currently, no curative treatments are available for these patients which underscores the need for effective new therapies.

“With no approved treatments for adenoid cystic carcinoma (ACC), a significant unmet need remains for therapies that slow or stop this relentless disease,” said Mark Gelder, M.D., chief medical officer of Elevar. “The orphan drug designation from the FDA reinforces the urgency of Elevar’s work with rivoceranib in ACC and our commitment to improving treatment experiences and outcomes for patients who have limited or inadequate therapeutic options.”

To Read the Complete Article at PR Newswire, Click Here

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Theratechnologies’ Lead Peptide Drug Conjugate TH1902 Receives FDA Fast Track Designation for the Treatment of Sortilin-expressing Cancers

February 8, 2021

by administrator Drug Discovery News Regulatory News

Theratechnologies Inc. (Theratechnologies) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, is pleased to announce that the United States Food and Drug Administration (FDA) has granted fast track designation to TH1902 as a single agent for the treatment of patients with sortilin positive recurrent advanced solid tumors that are refractory to standard therapy.

“Receiving fast track designation for TH1902 at this early stage of development is a significant recognition for our SORT1+ Technology™ and further supports the future development of TH1902. The designation, which applies to all solid tumours expressing sortilin, also highlights the broad applicability and immense medical need for innovative, targeted, and potentially more effective and better-tolerated therapies for cancer,” said Dr. Christian Marsolais, Senior Vice President and Chief Medical Officer, Theratechnologies.

Phase 1 clinical trial of TH1902
Theratechnologies announced on January 7, 2021 that it had received a “Study May Proceed” letter from the FDA for the Phase 1 clinical trial of TH1902.

The proposed Phase 1 trial design includes a dose escalation study to evaluate the safety, pharmacokinetics, maximum tolerated dose (MTD) and preliminary anti-tumor activity of TH1902 administered once every three weeks in patients with advanced solid tumors refractory to available anti-cancer therapies. Once the MTD is determined, it is planned that a total of 40 additional patients will be enrolled to evaluate the potential anti-tumor activity of TH1902 in patients with endometrial, ovarian, colorectal, pancreatic and triple negative breast cancers where it has been estimated that the sortilin receptor is expressed in 40 to 90% of cases. The Phase 1 trial is expected to be initiated in the second quarter of calendar year 2021 and is designed to identify a recommended dose for Phase 2 development.

Funda Meric-Bernstam, M.D., Chair of the Department of Investigational Cancer Therapeutics at The University of Texas MD Anderson Cancer Center is the Lead Principal Investigator of the Phase 1 trial for TH1902. The detailed study protocol is available at ClinicalTrials.gov under the identifier number: NCT04706962.

To Read the Complete Article at GlobeNewswire, Click Here

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