PHARMA TECH

COUR Pharmaceuticals Receives FDA Fast Track Designation for CNP-104 for the Treatment of Primary Biliary Cholangitis

COUR Pharmaceuticals | January 11, 2022

COUR Pharmaceuticals, a biotechnology company developing novel immune-modifying nanoparticles  to treat immune disorders, today announced the U.S. Food and Drug Administration granted Fast Track Designation to the Company's investigational therapy CNP-104 for the treatment of Primary Biliary Cholangitis. CNP-104 aims to reprogram the immune system to address autoimmune causes of PBC.

"We are pleased to have received Fast Track Designation for CNP-104, which demonstrates the FDA recognizes the importance of this potential treatment for PBC and the need for improved of clinical outcomes for patients with this degenerative autoimmune disease, which currently has no treatment to stop progression of disease. Based on data from another COUR autoimmune trial with a CNP platform product, our Phase 2 PBC trial is designed to show that we can stop progression of PBC disease with improved liver function.  If successful in Phase 2, COUR would seek breakthrough therapy designation in PBC given that current therapies treat only symptoms."

 COUR CEO John Puisis

Fast Track Designation is an FDA process designed to facilitate the development and review of potential therapies that seek to fill an unmet medical need. The FDA created this designation to help get new therapies to patients earlier. A therapy that receives this designation is eligible for more frequent communication with the FDA and may be eligible for Accelerated Approval and Priority review, if relevant criteria are met. 

The FDA accepted COUR's investigational new drug application for CNP-201 in October 2021 and COUR is currently opening the first human dosing in a proof-of-concept study for CNP-104 in early 2022 to evaluate the safety, tolerability, pharmacodynamics and efficacy of the therapy in PBC patients. CNP-104 uses COUR's proprietary nanoparticle platform, a novel system which combines disease specific pathogenic antigens with state-of-the-art pharmaceutical nanoparticles that mimic normal removal of dead or dying liver cells from the body.

In November 2021, Ironwood Pharmaceuticals, Inc., a GI-focused healthcare company entered into a collaboration and license option agreement with COUR for the development and commercialization of CNP-104.  This agreement gives Ironwood an option to acquire an exclusive license to develop and commercialize, in the U.S., COUR's investigational therapy CNP-104 for PBC.  

About COUR Pharmaceuticals
COUR Pharmaceuticals is developing first-in-class therapies designed to reprogram the immune system to achieve antigen-specific tolerance for immune-mediated disease. COUR's platform of immune-modifying nanoparticles is designed to treat the root cause of immune disease, unlike traditional approaches, which only minimize symptoms using toxic immune suppression. Data from clinical and preclinical settings demonstrate the opportunity for the COUR nanoparticle platform to address a wide range of immune and inflammatory conditions. The underlying technology was acquired from Northwestern University and draws from more than 30 years of research by the laboratory of Stephen D. Miller, Ph.D., the Judy E. Guggenheim Research Professor of Microbiology-Immunology.

Spotlight

Leading Australian medicinal cannabis exporter, Little Green Pharma continues to expand making significant strides in lucrative global markets

Spotlight

Leading Australian medicinal cannabis exporter, Little Green Pharma continues to expand making significant strides in lucrative global markets

Related News

RESEARCH, PHARMACY MARKET

Impel Pharmaceuticals Prioritizes Trudhesa Commercial Opportunity

Impel Pharmaceuticals | February 23, 2023

On February 22, 2023, Impel Pharmaceuticals, a pharmaceutical firm that develops innovative treatments for diseases with high unmet medical needs, announced that it will execute an operational streamlining plan to reduce employee and non-employee expenses, primarily affecting the research and development division. The company intends to divert resources to benefit from the successful response of payors and prescribers to Trudhesa® (dihydroergotamine mesylate) nasal spray (0.725 mg per spray). Impel Pharmaceuticals will stop research and development activities on INP105, a drug to treat agitation and aggression in autism spectrum disorder. The reduction in expenses is expected to come from lower non-employee-related general and administrative and research and development expenses, as well as a reduction of 16 percent in headcount. Additionally, the company announced that its Chief Medical Officer, Stephen Shrewsbury, M.D., will leave the company on March 31, 2023. The company projects that these measures, along with others, will provide sufficient capital to fund operations through Q3 2023, allowing it to focus on maximizing the commercial opportunity and positive momentum of Trudhesa® in the huge and rapidly expanding acute migraine market. Trudhesa® has experienced a strong launch trajectory, with 58,424 prescriptions at the end of 2022, accounting for 4.3% of branded acute migraine prescriptions (nTRx) among prescribers after a full-year launch. Impel expanded its sales force by 50%, from 60 to 90 sales professionals, in July 2022 to capitalize on this positive momentum, resulting in a 58% increase in new quarterly prescriptions from Q2 to Q4 2022. Reimbursement of all shipments finished the year at over 60%, and refill rates remained high, with an average of 63% at the end of 2022. The company plans to increase its efforts to commercialize Trudhesa® while also exploring strategic and financing initiatives and opportunities to fully leverage the potential of its proprietary Precision Olfactory Delivery (POD®) technology and expertise with small and large molecules in various disease areas. About Impel Pharmaceuticals Impel Pharmaceuticals is a commercial-stage pharmaceutical firm that focuses on developing and providing treatments for patients suffering from diseases with high unmet needs. The company employs a novel approach to drug delivery by utilizing its proprietary Precision Olfactory Delivery (POD®) technology in conjunction with established therapeutics to develop and offer treatments. In September 2021, it received approval from the U.S. FDA for its first product, Trudhesa® nasal spray, which is used to treat acute migraine with or without aura in adults. Along with Trudhesa, the company continues to address patient needs through licensing and partnerships.

Read More

BUSINESS INSIGHTS, PHARMA TECH

KSQ Announces ONO’s Acquisition of Multiple Research-stage Oncology Programs

KSQ Therapeutics | January 30, 2023

KSQ Therapeutics, a leading clinical-stage biotechnology company developing therapies to treat cancer and autoimmune diseases, recently announced that Ono Pharmaceutical (ONO) has acquired multiple research-stage DNA damage response (DDR) programs. The programs were identified using KSQ's CRISPRomics platform technology. All of the programs are new and have the potential to be first-in-class therapies. ONO's Discovery & Research Executive Director/ Senior Executive Officer Toichi Takino commented, "ONO appreciates the ability of KSQ's CRISPRomics platform technology to discover novel therapeutic targets with high selectivity for cancer cells." He added, "Through this acquisition agreement, we expect that the programs acquired from KSQ will lead to the creation of innovative medicines that will help treat cancer patients." (Source – Business Wire) KSQ's Chief Executive Officer Qasim Rizvi commented, "This acquisition of multiple research-stage oncology programs by ONO is further validation of the power of our platform to identify novel oncology targets and develop potential first-in-class programs." He added, "This is an important transaction for KSQ, enabling us to focus on our other programs. We look forward to watching ONO's progress as they advance these programs toward clinical studies." (Source – Business Wire) As per the terms of the agreement, ONO will pay KSQ a double-digit million upfront payment, prospective near-term milestone payments with potential long-term value in the triple-digit millions, and net sales-based royalties. About KSQ Therapeutics Founded in 2015, KSQ Therapeutics is a clinical-stage firm using its CRISPRomics® discovery platform to systematically decode the genome and identify optimal gene targets for oncology and autoimmune disease. It utilizes the platform for innovative drug development across diverse therapeutic domains and is actively creating a proprietary pipeline of tumor- and immune-focused drug candidates. Headquartered in Cambridge, Massachusetts, KSQ was formed by functional genomics thought leaders and CRISPR screening technology pioneers.

Read More

BUSINESS INSIGHTS, PHARMACY MARKET

Caris Life Sciences and Xencor Expand Target Discovery Collaboration

Caris Life Sciences and Xencor | January 06, 2023

Caris Life Sciences®, a molecular science and technology company, and Xencor, Inc., a clinical-stage biopharmaceutical company, have announced that they will work more closely together to research, develop, and sell new bispecific and multispecific XmAb® antibodies. This expanded relationship will boost the number of targets and cancer types that Caris will investigate with Caris Discovery, a proprietary multi-omics discovery engine platform. The foundation for drug candidate development on Xencor's XmAb platform will be novel targets and their accompanying validation packages. Under the terms of the new contract, Xencor will receive worldwide commercial rights to study, develop, and market products aimed at up to three targets discovered through collaboration. In addition, Caris will get an upfront payment and may receive up to $187 million in commercial milestone payments and development. The company will also be eligible for royalties on the net sales of products commercialized as a result of the agreement, as well as future rights for molecular profiling and companion diagnostics. Caris Discovery leverages the collective force of its unmatched platform by combining the data generated from the primary patient tissues' molecular interrogation using ADAPT™, which integrates the company's extensive catalog of molecular and clinical data. About Caris Life Sciences Caris Life Sciences® is a Texas-based molecular science firm revolutionizing healthcare. Its market-leading molecular profiling products analyze DNA, RNA, and proteins to produce a molecular blueprint. This plan shows how patients, doctors, and researchers can use the information to improve outcomes and save lives. The company also uses cutting-edge algorithms for artificial intelligence (AI) and machine learning (ML) to improve precision medicine. About Xencor Xencor develops modified antibodies and cytokines for cancer and autoimmune illnesses. More than 20 XmAb® candidates are in clinical development while three are sold by partners. The company’s XmAb technology modifies protein structures to create new therapeutic pathways.

Read More