BUSINESS INSIGHTS

Tris Pharma Announces Development of a Very Low Sodium and Once-Nightly Oxybate Formulation

Tris Pharma, Inc. | July 28, 2022 | Read time : 02:00 min

Tris Pharma
Tris Pharma, Inc. a fully integrated pharmaceutical company with a robust portfolio of CNS products announced that it has successfully developed an oxybate formulation for the treatment of cataplexy or excessive daytime sleepiness n patients 7 years of age and older with narcolepsy that has the potential to be significantly advantageous to existing approved therapies, such as Jazz Pharmaceuticals' blockbuster XYWAV® and XYREM® franchise. The Tris oxybate formulation is believed to provide an advantage over approved therapies by utilizing Tris's proprietary LiquiXR technology platform and our newly developed RaftWorks design to provide a unique oxybate formulation offering both once-nightly dosing, and very low sodium content. The Tris oxybate formulation is investigational and not yet approved by any regulatory agency.

Both XYWAV® and XYREM® require twice a night dosing – once at bedtime and again 2.5 to 4 hours later for the second dose. Further, XYREM contains an express warning based on its high sodium content and associated risk to patients with heart failure, hypertension or impaired renal function. The Tris oxybate formulation seeks to improve on existing therapies by providing convenient once per night dosing, and significantly reduced sodium content such that it will contain less than 100 mg of sodium per 9.0 gm of maximum sodium oxybate equivalent dose. Based on the significant potential advantage the Tris oxybate formulation could provide over existing available therapies, Tris is pursuing an Orphan Drug Designation for its unique oxybate formulation.

In a human clinical study, Tris established about 100% bioavailability for one dose of its formulation against two doses of XYREM at 4.5 gm each, equivalent to the 9.0 gm maximum dose of sodium oxybate. If approved, the Tris product will provide significant advantages in that patients will not have to wake up in the middle of the night to take a second dose and will have all the benefits of a very low sodium product.

The Tris oxybate product achieved its clinical objectives by combining two distinct proprietary Tris technologies: the LiquiXR platform, and the newly developed RaftWorks design. Working together, these unique formulation platforms provide an interpenetrating network raft in-situ, in which sodium-free drug particles are entrapped in a raft that allows for extended release of the active ingredient. Many drugs, such as oxybate, are poorly absorbed because they have a narrow window of absorption within the human gastro-intestinal tract. The RaftWorks approach permits a slow and extended release of oxybate from a floating raft in-situ, thereby facilitating absorption of the active ingredient within the targeted area in the gastro-intestinal tract. Further, the raft disintegrates at a predetermined time due to the trigger mechanism that is built into the formulation.

"This unique formulation design by Tris scientists potentially achieves drug delivery capabilities that have never previously been achieved within our industry. We are looking to build on this approach and develop extended-release formulations for other drugs that are limited by their narrow window of absorption."

Ketan Mehta, Founder and Chief Executive Officer of Tris

About Narcolepsy
Narcolepsy, a chronic and disabling neurological disorder that affects the stability of sleep and wakefulness, is characterized primarily by excessive daytime sleepiness, with or without episodic loss of muscle tone usually triggered by strong emotions during wakefulness (cataplexy) 1-4. Additional symptoms can vary by person and may include disrupted nighttime sleep, and REM-sleep phenomena (sleep paralysis, hallucinations). The exact number of individuals with narcolepsy in the United States is unknown. Several studies have estimated the prevalence of narcolepsy in the United States to be approximately 1 (range: 0.6 to 1.6) in 2,000 people 2,4-6. There is currently no cure for narcolepsy; treatment of symptoms includes medications and lifestyle modifications 1.

About Tris Pharma
Tris is a fully integrated, innovation-driven CNS company that provides a differentiated approach to target unmet medical needs, including the application of novel technologies designed to enhance patient benefits across therapeutic categories. Tris's CNS portfolio includes treatments for pain; addiction; spasticity in multiple sclerosis, cerebral palsy; narcolepsy; and ADHD. 

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Pfizer Completes Acquisition of Biohaven Pharmaceuticals

Pfizer and Biohaven Pharmaceuticals | October 04, 2022

Pfizer Inc. announced the completion of its acquisition of Biohaven Pharmaceutical Holding Company Ltd., the maker of NURTEC® ODT an innovative migraine therapy approved for both acute treatment and prevention of episodic migraine in adults. The acquisition brings to Pfizer a portfolio of promising calcitonin gene-related peptide receptor antagonists including Rimegepant Approved in the United States under the trade name NURTEC® ODT, in adults for both the acute treatment of migraine with or without aura and the preventive treatment of episodic migraine Approved in the European Union under the trade name VYDURA® for both the acute treatment of migraine with or without aura in adults and the preventive treatment of episodic migraine in adults who have at least 4 migraine attacks per month Zavegepant New Drug Application for intranasal spray for the acute treatment of migraine under U.S. Food and Drug Administration review, with a Prescription Drug User Fee Act goal date in 1Q 2023 "We are proud to build on Pfizer’s legacy of delivering breakthrough medicines for patients living with complex pain disorders. The success of NURTEC® ODT coupled with Biohaven’s CGRP pipeline will strengthen Pfizer’s innovative Internal Medicine pipeline through 2030, and beyond. Combined with Pfizer’s global reach, this acquisition increases our potential to bring new treatment options to patients with migraine – a disease which affects over 1 billion people worldwide. Aamir Malik, Executive Vice President, Chief Business Innovation Officer, Pfizer Pfizer acquired all of the outstanding shares of Biohaven not already owned by Pfizer for $148.50 per share in cash, for a total transaction consideration of approximately $11.6 billion. As a result of the acquisition, Biohaven became a wholly-owned subsidiary of Pfizer. Effective immediately prior to the closing of the acquisition, Biohaven completed the spin-off of Biohaven Ltd. distributing Biohaven Ltd.’s shares to Biohaven’s shareholders. Biohaven Ltd., a new company that retained Biohaven’s non-CGRP development stage pipeline compounds, holds the Kv7 ion channel activators, glutamate modulation, and myostatin inhibition platforms, preclinical product candidates, and certain corporate infrastructure assets excluded from the Pfizer acquisition. Pfizer, a Biohaven shareholder, received a pro rata portion of Biohaven Ltd.’s shares in the distribution and owns approximately 3% of Biohaven Ltd. Biohaven Ltd. will continue to trade on the New York Stock Exchange under the ticker “BHVN”. About Migraine Worldwide, more than one billion people suffer from migraine, which predominately affects women.i Findings from the 2019 Global Burden of Disease study indicate that migraine is one of the worlds leading causes of disability.ii Migraine is characterized by debilitating attacks lasting four to 72 hours with multiple symptoms, including pulsating headaches of moderate to severe pain intensity that can be associated with nausea or vomiting, and/or sensitivity to sound and sensitivity to light. About Rimegepant Rimegepant targets a key component of migraine by reversibly blocking CGRP receptors, thereby inhibiting the biologic cascade that results in a migraine attack. Rimegepant was approved by the U.S. Food and Drug Administration (FDA) under the trade name Nurtec® ODT for the acute treatment of migraine in adults in February 2020 and for the preventive treatment of episodic migraine in adults in May 2021. Nurtec® ODT is taken orally as needed, up to once daily for acute treatment, and every other day for preventive treatment. The maximum dose in a 24 hour period is 75 mg. About Zavegepant Zavegepant is a third generation, high affinity, selective and structurally unique, small molecule CGRP receptor antagonist from the NOJECTION® Migraine Platform and the only CGRP receptor antagonist in clinical development with both intranasal and oral formulations. The FDA has accepted for review a New Drug Application (NDA) for zavegepant nasal spray, with a PDUFA date in 1Q 2023. About Pfizer: Breakthroughs That Change Patients’ Lives At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us.

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TC BioPharm Begins Dosing Phase 2B Clinical Study Evaluating its Lead Compound, OmnImmune®, in Patients with Acute Myeloid Leukemia

TC BioPharm | November 23, 2022

TC Biopharm a clinical stage biotechnology company developing platform allogeneic gamma-delta T cell therapies for cancer today announced the dosing of its first three patients within its Phase 2b clinical trial of OmnImmune®, an allogeneic unmodified cell therapy focused on treating Acute Myeloid Leukemia. The initial 5 patients in the trial are deemed a "safety cohort", spaced two weeks apart with safety review by an oversight board to confirm no drug related toxicity issues, subsequent to 5 patients being dosed the study will advance to open enrollment. This safety cohort is in line with TCBP's step-wise clinical trial advancement, moving from donor matching in the Phase 1b to a universal donor model with no HLA matching of donor to patient. "The launch of our Phase 2B trial is a key milestone in the development of our lead therapeutic, OmnImmune®, for patients with AML and for TC BioPharm's emerging pipeline of 'off-the-shelf' gamma-delta T cell therapies. This study design includes a 5 patient safety cohort prior to open enrollment, we expect to complete the safety cohort before the end of 2022. The next step in the study is a 19 patient interim review, which will allow TCBP to review dosing and increase dosing to a higher level should our team deem it necessary for efficacy, or we can elect to maintain our current dosing level of 7x10^7 or 700 million cells per dose. We look forward to moving ahead with our Phase 2b trial with a target for open enrollment in January 2023, as well as our efforts to expand our clinical efforts in the US in the first half of 2023." Bryan Kobel, CEO of TC BioPharm TC BioPharm's Phase 2B trial, dubbed ACHIEVE, will enroll adults diagnosed with AML who have either relapsed or are refractory to prior treatments as well as a cohort for patients with myelodysplastic syndromes conditions that can occur when the blood-forming cells in the bone marrow become abnormal. The trial is expected to enroll approximately 37 patients. About TC BioPharm PLC TC BioPharm is a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of gamma-delta T cell therapies for the treatment of cancer with human efficacy data in acute myeloid leukemia. Gamma-delta T cells are naturally occurring immune cells that embody properties of both the innate and adaptive immune systems and can intrinsically differentiate between healthy and diseased tissue. TC BioPharm uses an allogeneic approach in both unmodified and CAR modified gamma-delta T cells to effectively identify, target and eradicate both liquid and solid tumors in cancer. TC BioPharm is the leader in developing gamma-delta T cell therapies, and the first company to conduct phase II/pivotal clinical studies in oncology. The Company is conducting two investigator-initiated clinical trials for its unmodified gamma-delta T cell product line - Phase 2b/3 pivotal trial for OmnImmune® in treatment of acute myeloid leukemia using the Company's proprietary allogenic CryoTC technology to provide frozen product to clinics worldwide. TC BioPharm also maintains a robust pipeline for future indications in solid tumors as well as a significant IP/patent portfolio in the use of CARs with gamma-delta T cells and owns our manufacturing facility to maintain cost and product quality controls.

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PHARMACY MARKET

CASI PHARMACEUTICALS ANNOUNCES EXIT OF JUVENTAS INVESTMENT TO STRENGTHEN BALANCE SHEET

CASI Pharmaceuticals, Inc. | September 26, 2022

CASI Pharmaceuticals, Inc. a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products announced that CASI Biopharmaceuticals Co., Ltd, a subsidiary of CASI Pharmaceuticals, Inc. entered into an Equity Transfer Agreement with Shenzhen Jiadao Gongcheng Equity Investment Fund, LLP pursuant to which CASI Wuxi agreed to transfer its equity interest in Juventas Biotechnology Co., Ltd. amounting to 12.0098% total Juventas equity to Jiadao Gongcheng for RMB 240.87 million. The Equity Transfer Agreement states there will be two even payment installments from Jiadao Gongcheng; one payment to be made after the Equity Transfer Agreement is signed and the second payment to be made after the completion of the equity transaction. "We are confident in CNCT-19, and we expect Juventas to submit for NDA to the NMPA in 2022 for the B-ALL indication. CASI and Juventas will continue to maintain a working partnership, focusing on product launch and co-marketing for CNCT-19." Wei-Wu He, Ph.D., CASI's Chairman and Chief Executive Officer Dr. He continued, "The completion of the equity transfer transaction will strengthen CASI's balance sheet by providing CASI with sufficient cash to operate until, at least, the end of 2023. This transaction will allow CASI to advance the development of our other pipeline projects while augmenting the efforts of our sales and marketing team." About CNCT-19 CNCT19 targets CD19, a B-cell surface protein widely expressed during all phases of B-cell development and a validated target for B-cell driven hematological malignancies. CD19- targeted CAR constructs from several different institutions have demonstrated consistently high antitumor efficacy in children and adults with relapsed B-cell acute lymphoblastic leukemia chronic lymphocytic leukemia and B-cell non-Hodgkin lymphoma. CD19 antigen is the most frequently used target in the CAR-T cell therapy clinical trials for hematological malignancies such as leukemia and lymphoma. Juventas is responsible for the development of CNCT19. CASI and Juventas will co-commercialize CNCT19 under the direction of the program's joint steering committee. About CASI Pharmaceuticals CASI Pharmaceuticals, Inc. is a U.S. biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products in China, the United States, and throughout the world. The Company is focused on acquiring, developing, and commercializing products that augment its hematology oncology therapeutic focus as well as other areas of unmet medical need. The Company intends to execute its plan to become a leader by launching medicines in the Greater China market, leveraging the Company's China-based regulatory and commercial competencies and its global drug development expertise. The Company's operations in China are conducted through its wholly-owned subsidiary, CASI Pharmaceuticals Co., Ltd., located in Beijing, China. The Company has built a commercial team of more than 100 hematology and oncology sales and marketing specialists based in China. About Juventas Juventas is a biopharmaceutical company headquartered in China dedicated to the development and commercialization of cell therapies globally. Utilizing innovative and integrated technology platforms, the company has developed a diverse pipeline of cellular immunotherapies for treatment of hematological malignancies, solid tumors, and other non-oncological conditions both in China and globally. At present, the company is conducting two pivotal clinical trials of CNCT19 for treating adult r/r-B-ALL and r/r-B-NHL in China. CNCT19 has the potential to become the first launched domestically developed CD19 CAR-T therapy in China and the first CAR-T product for the treatment of adult R/R B-ALL in China.

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