Category Archives: Biotech Companies Infectious Diseases

Ocedurenone: A Novel Therapy for Uncontrolled Hypertension in Advanced Chronic Kidney Disease

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EMJ Nephrology interviewed two World-renowned experts in nephrology and cardiology, George Bakris and Faiez Zannad, regarding the challenges of treating uncontrolled hypertension (uHTN) in patients with advanced chronic kidney disease (CKD), a large unmet need for safe, effective therapies, and the potential of one therapy, Ocedurenone, to address this need.

Original article: EMJ-8.1-2023-1.pdf (emg-health.com)

Large Unmet Need

Patients with advanced CKD have a high burden of disease, compounded by serious comorbidities, most commonly hypertension. Controlling hypertension is vital in these patients to reduce the associated risks of morbidity and mortality, as well as reducing cardiorenal risk, but treatment options are limited due to safety concerns with the use of existing agents.

Current Options

Bakris:”The PATHWAY-2 trial showed that in people with resistant hypertension without advanced CKD, spironolactone produced a greater reduction in blood pressure than a β-blocker and an α-blocker. However, it presents safety risks to patients when administered.”

“Then there is eplerenone, given twice a day, while safe, has a much weaker effect on blood pressure reduction than spironolactone. That’s all we’ve got, so that’s what people use.”

Dr Bakris also mentioned two existing nonsteroidal MRAs, esaxerenone which is “approved exclusively in Japan, for which there are no placebo-controlled comparisons”, and Finerenone which “reduces blood pressure (placebo subtracted) variously from 3 mmHg (ARTS-DN, FIGARO-DKD, and FIDELIO-DKD studies) to approximately 8 mmHg (subset analysis from ARTS-DN study, eGFR of approximately 68, though office blood pressure from the same study showed around 3 mmHg reduction)”.

Future Option – Ocedurenone

Bakris:”Now, Ocedurenone, a non-steroidal MRA, shows clear promise in patients whose standard of care includes two, three, or more drugs. Ocedurenone is the only drug that has consistently steadied resistant hypertension in people with advanced CKD.”

In the first dedicated study of patients with uncontrolled hypertension and advanced CKD (BLOCK-CKD), Ocedurenone demonstrated efficacy in lowering blood pressure, together with a favorable safety profile. The experts were optimistic that it could provide a much-needed treatment option for these patients, with the added potential for reduction in the risk of cardiorenal outcomes.

“This study showed that Ocedurenone reduces SBP by 11 mmHg (placebo-subtracted), a reduction that could lead to potential approval. No other agent to date has steadied blood pressure as well in this group of patients. And the point is that these are the patients who need the most help.”

Zannad reinforced the importance of the hyperkalemia data for the clinical safety of Ocedurenone:”With Ocedurenone, we may for the first time have an MRA that is both safe and efficacious.”

Bakris added:”A Phase 3 study (CLARION-CKD) of Ocedurenone in a similar patient population is ongoing. I remain optimistic that the signal seen in the BLOCK-CKD study will be seen in this larger study.”

“I remain excited about Ocedurenone. Its tolerability and efficacy in lowering blood pressure in a very high-risk group will have a significant impact on morbidity.”

“Mineralocorticoid receptors are so ubiquitous which may allow for indications beyond CKD and cardiovascular conditions. This is likely just the beginning for Ocedurenone.”

About Ocedurenone (KBP-5074)

KBP Biosciences is a global, clinical-stage biotechnology company, headquartered in Princeton, NJ, focused on discovering, developing, and commercializing innovative small-molecule therapeutics for the treatment of serious cardiorenal and infectious diseases with large unmet medical needs.

Ocedurenone (KBP-5074) is a non-steroidal MRA discovered and developed by KBP Biosciences. At present, the Phase 3 clinical trial of the first indication for Ocedurenone, advanced CKD and uncontrolled hypertension, is underway.

About KBP Biosciences, please visit https://www.kbpbiosciences.com/.

Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

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Autophagy is a catabolic process which degrades a cellular own component through the lysosomal machinery. The lysosome, at the heart of the autophagy sytem is important in various processes (cancer, infection…).

Genoscience Pharma - new approaches disrupting cancer cell lysosomal functions

In Cancer, they are required in tumor cells for cellular adhesion, motility and signaling, exocytosis, angiogenesis and overall survival, growth, aggressiveness, metastatic potential and drug resistance. Because of their high metabolic rates, rapidly dividing and invasive cancer cells require increased new biomass production to survive. The lysosome is also important for adaptation to nutrient stress as it contains hydrolytic enzymes that play a major role in the degradation of intracellular macromolecules and catabolic (such as autophagy) and anabolic growth. This busy lysosomal behavior leads to alterations in lysosomal structure and function, which, paradoxically, renders cancer cells more sensitive to lysosomal destabilization. In addition, lysosomal enzyme activity is elevated in many tumors compared to adjacent normal tissue, and several reports suggest that lysosomes in tumor cells are more fragile than normal lysosomes. Therefore, lysosome seems to be a target of interest in the fight against cancers. Targeting lysosomes triggers apoptotic and lysosomal cell death pathways.

Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

In virology, It has been shown that autophagy is activated during virus and bacterial infection and that some viruses can use the autophagy system to facilitate their own replication . Some viruses, such as Coronaviruses are single stranded, positive sense RNA viruses, which induce the rearrangement of cellular membranes upon infection of a host cell. This provides the virus with a platform for the assembly of viral replication complexes, improving efficiency of RNA synthesis. Genoscience Pharma focuses on new approaches disrupting cancer cell lysosomal functions.

Genoscience Pharma was founded in 2001 by Pr Philippe Halfon, a world renowned medical expert on viral diseases, especially on Human Immunodeficient Virus (HIV) and Hepatitis C Virus (HCV). The company was initially focused on the development of anti-HCV agents. Two protease inhibitors were thus developed and out-licensed to BioLineRX. A new scientific direction was taken in 2012 after the discovery of a new chemical family: autophagy inhibitors. Following promising preliminary in vitro and in vivo results from these small molecules, including against cancer stem cells, Genoscience Pharma has decided to take the opportunity to make the difference in Oncology, especially in cancers where medical needs are still unmet. Now, Genoscience Pharma is a clinical stage biopharmaceutical company focused on translating novel scientific insights into medicines for patients with cancer.

Genoscience Pharma – new approaches disrupting cancer cell lysosomal functions

More info: https://www.genosciencepharma.com/

Keywords : lysosomal functions , cancer, oncology , Genoscience Pharma , lysosomes , infectious diseases  , virus , Covid19, leukemia , pancreatic cancer , hepatocarcinoma , small molecules

Biophytis – therapeutics to slow down the degenerative processes associated with aging

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Biophytis is a clinical-stage biotechnology company specialized in the development of therapeutics that are aimed at slowing the degenerative processes associated with aging and improving functional outcomes for patients suffering from age-related diseases, including severe respiratoy failure in patients suffering from COVID-19.

Biophytis - therapeutics to slow down the degenerative processes associated with aging

Biophytis SA, founded in 2006, develops drug candidates targeting diseases of aging. Using its technology and know-how, Biophytis has begun clinical development of innovative therapeutics to restore the muscular and visual functions in diseases with significant unmet medical needs. Specifically, the company is advancing two proprietary drug candidates into mid-stage clinical testing this year: Sarconeos (BIO101) to treat sarcopenic obesity and Macuneos (BIO201) to treat dry age-related macular degeneration (AMD). The business model of BIOPHYTIS is to ensure the conduct of the project until clinical activity in the patient is proven, then to license the technologies in order to continue the development in partnership with a pharmaceutical laboratory. The company was founded in partnership with researchers at the UPMC (Pierre and Marie Curie University) and collaborates with scientists at the Institute of Myology, and the Vision Institute

Its lead drug candidate, Sarconeos (BIO101), is an orally administered small molecule in development for the treatment of neuromuscular diseases, including sarcopenia and Duchenne muscular dystrophy (DMD). Our second drug candidate, Macuneos (BIO201), is an orally administered small molecule in development for the treatment of retinal diseases, including dry AMD and Stargardt disease.

Biophytis also has preclinical efficacy data for Sarconeos (BIO101) in Spinal Muscular Atrophy (SMA), a rare neuromuscular disease. Spinal Muscular Atrophy is a rare progressive neurodegenerative disease that robs an individual of the ability to walk, eat and breathe. SMA is the leading genetic cause of death in infants. Symptoms may appear in the first 6 months of life (type 1, the most severe and common), in infancy (types 2 and 3) or in adulthood (type 4, the least common form). SMA affects 1 in 11,000 births in the United States each year, and about 1 in 50 Americans is a genetic carrier.

More info : www.biophytis.com

Keywords for Biophytis :

neuromuscular diseases, dry age-related macular degeneration, AMD, ophthalmology, infectious diseases, COVID19, Asthma, COPD, small molecule, sarcopenia, Stargardt disease, Duchenne muscular dystrophy , DMD, preclinical stage pharma company, clinical stage pharma company , Spinal Muscular Atrophy , rare diseases , aging

Bavarian Nordic – poxviral-based vaccine

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Bavarian Nordic is a fully integrated biotechnology company focused on the development, manufacturing and commercialization of cancer immunotherapies and vaccines for infectious diseases.

Bavarian Nordic – poxviral-based vaccine

Bavarian Nordic has built its foundation around two poxviral-based vaccine platform technologies, Modified Vaccinia Ankara – Bavarian Nordic (MVA-BN) and Vaccinia-Fowlpox-TRICOM (VF-TRICOM) that have the potential to support a broad product pipeline in both infectious diseases and cancer immunotherapies. Both technologies can be manufactured at commercial scale at Bavarian Nordic’s own facility, which was recently expanded to accommodate the production of multiple products. Bavarian Nordic focuses on developing product candidates to address cancer and infectious diseases by leveraging several core strengths:

  • Extensive R&D expertise in vaccines and immunotherapy
  • Broadly applicable platform technology
  • Multiple clinical stage development programs, including two Phase 3 trials
  • Flexible, commercial scale manufacturing facility
  • Significant financial resources resulting from IMVAMUNE supply contracts and government-funded R&D programs

Through a long-standing collaboration with the U.S. Government, Bavarian Nordic has developed a portfolio of biological countermeasures, including the non-replicating smallpox vaccine, IMVAMUNE, which is stockpiled for emergency use by the U.S. and other governments. The vaccine is approved in the EU (under the trade name IMVANEX) and in Canada. Bavarian Nordic and its partner Janssen are pioneering the development of an Ebola vaccine, which has been fast-tracked by authorities in response to the current situation in West Africa. Additionally, in collaboration with the National Cancer Institute, Bavarian Nordic has developed a portfolio of active cancer immunotherapies based on its versatile pox-virus based technologies, including PROSTVAC, which is currently in Phase 3 clinical development for the treatment of advanced prostate cancer. The company has partnered with Bristol-Myers Squibb for the potential commercialization of PROSTVAC.

Bavarian Nordic is a biotech company headquartered in Denmark, Germany and USA. Bavarian Nordic’s shares are listed on Nasdaq Copenhagen under the symbol BAVA (Reuters: BAVA.CO, Bloomberg: BAVA.DC). The company has a sponsored Level 1 ADR program listed in the US (OTC) under the symbol BVNRY.

More about Bavarian Nordic : www.bavarian-nordic.com

Bavarian Nordic – poxviral-based vaccine – poxviral – vaccine – PROSTVAC – IMVANEX – IMVAMUNE

Bavarian Nordic – poxviral-based vaccine – poxviral – vaccine – PROSTVAC – IMVANEX – IMVAMUNE