Thiogenesis Therapeutics, Corp.

THX Pharma is a biopharmaceutical company that develops therapeutic solutions for rare neurological diseases such as Batten disease, Niemann-Pick type C, and Gaucher disease. The company utilizes antisense oligonucleotides and scientific research to create targeted drug candidates for patients with limited treatment options.

EgetisPharmaceuticalsSweden

65% match

Egetis Therapeutics is a specialty pharmaceutical company that develops and commercializes treatments for serious, rare diseases with high unmet medical need. Its lead product is Emcitate (tiratricol), a treatment for MCT8 deficiency. The company serves patients, caregivers, and healthcare professionals.

Eloxx Pharmaceuticals is a clinical-stage biopharmaceutical firm that specializes in developing therapeutic solutions through ribosome modulation. Its primary objective is to address rare and ultra-rare genetic diseases that arise from premature stop codons. The company's most advanced investigational compound, ELX-02, is currently progressing through Phase 2 clinical trials. This candidate is being evaluated for its potential to treat patients with cystic fibrosis and nephropathic cystinosis who possess diagnosed nonsense mutations. In addition to its lead program, Eloxx is actively pursuing several preclinical projects aimed at conditions like Alport syndrome, recessive dystrophic epidermolysis bullosa, junctional epidermolysis bullosa, and familial adenomatous polyposis. The company also maintains various earlier-stage discovery initiatives focused on oncology. Eloxx Pharmaceuticals was established in 2013 and is headquartered in Watertown, Massachusetts.

Catalyst Pharmaceuticals, Inc., founded in 2002 and based in Coral Gables, Florida, operates as a commercial-stage biopharmaceutical enterprise. This company is dedicated to discovering, developing, and marketing therapeutic solutions for individuals in the United States who are afflicted with uncommon, severe, and long-term neuromuscular and neurological disorders. Among its key offerings is Firdapse, an amifampridine phosphate tablet formulation approved for managing Lambert-Eaton Myasthenic Syndrome (LEMS). Catalyst also provides Ruzurgi, a specific treatment option for pediatric patients diagnosed with LEMS. Beyond its existing products, Catalyst is actively investigating additional applications for Firdapse, including its potential use in treating MuSK antibody positive myasthenia gravis, spinal muscular atrophy type 3, and hereditary neuropathy with liability to pressure palsies. The company engages in various strategic collaborations, such as licensing agreements with BioMarin Pharmaceutical Inc., and a joint development and commercialization agreement with Endo Ventures Limited concerning a generic version of Sabril tablets. Previously known as Catalyst Pharmaceutical Partners, Inc., the company adopted its current name in May 2015.

Curatis Holding AG specializes in the entire lifecycle of pharmaceutical products, including the procurement, advancement, and market release of medications specifically designed for the diagnosis, prevention, and management of rare and specialized medical conditions. The company's extensive product offerings span a broad array of therapeutic areas. These encompass treatments for antiplatelet conditions, metastatic hormone receptor-positive breast cancer, anti-arrhythmic disorders, nasal oil applications, Wilson's disease, antidotes for cyanide poisoning and digoxin toxicity, antihypotensive agents, therapies for bladder pain syndrome/interstitial cystitis, antibiotics, diagnostic tools for ophthalmology, antivirals, ophthalmic/antibiotic combinations, gentamycin collagen sponges, solutions for benign prostatic hyperplasia, immunomodulators, treatments for epilepsy and essential tremor, thyreostatics, therapies for hereditary tyrosinemia type 1, gastroenterological products, additional immunomodulators and thyreostatics, tranquilizers, Helicobacter pylori tests, phosphate binders, specialized nutritional support for dialysis patients, dermatological corticosteroids, antidotes for lead and mercury poisoning, corticostimulants, anti-fog agents, treatments for chorea associated with Huntington's disease, and antiepileptics. In addition to its commercialized portfolio, Curatis is actively engaged in developing new pharmaceutical compounds. Its pipeline includes C-PTBE-01, currently in Phase IIb clinical trials for treating peritumoral brain edema in pediatric patients; C-AM-01, undergoing Phase IIa trials for preventing severe migraine with aura; and C-MOH-01, also in Phase IIa for the treatment and prevention of medication overuse headache. Furthermore, KIN001 is in Phase I clinical trials, targeting rare inflammatory and fibrotic diseases. The company maintains its headquarters in Liestal, Switzerland.

Poxel0RA2 · PharmaceuticalsFrance

64% matchPublic

Poxel S.A. is a biopharmaceutical firm in the clinical development phase, dedicated to discovering innovative therapies for metabolic disorders, particularly type 2 diabetes and various liver conditions. Its primary asset, TWYMEEG (Imeglimin), is an oral medication that addresses mitochondrial dysfunction and has already received approval in Japan for managing type 2 diabetes. Poxel is also advancing PXL770, an activator of the adenosine monophosphate-activated protein kinase (AMPK) enzyme, currently in Phase 2a trials for various chronic metabolic diseases, notably liver ailments like non-alcoholic steatohepatitis (NASH). Furthermore, the company holds licensing agreements: one with Enyo Pharma S.A.S. for a farnesoid X receptor (FXR) agonist, now in a Phase 2a study for hepatitis B and NASH, and another with DeuteRx LLC for PXL065, a mitochondrial pyruvate carrier inhibitor undergoing Phase 1 evaluation specifically for NASH treatment. Established in 2009, Poxel S.A. operates from its headquarters in Lyon, France.