Pharming Group N.V., a biopharmaceutical company, develops and commercializes protein replacement therapies and precision medicines for the treatment of rare diseases in the United States, Europe, and internationally. The company offers RUCONEST, a recombinant C1 esterase inhibitor for the treatment of acute attacks in adult and adolescent patients with acute hereditary angioedema (HAE); and Joenja (leniolisib), an oral small molecule PI3K? inhibitor for the treatment of activated phosphoinositide 3-kinase delta syndrome. It also develops OTL-105, an investigational ex-vivo autologous hematopoietic stem cell gene therapy for the treatment of HAE. The company has a development collaboration and license agreement with Novartis; and a strategic collaboration agreement with Orchard Therapeutics plc for research, development, manufacturing, and commercialization of OTL-105. Pharming Group N.V. was incorporated in 1988 and is headquartered in Leiden, the Netherlands.

Eton Pharmaceuticals, Inc., a specialty pharmaceutical company, focuses on developing, acquiring, and commercializing pharmaceutical products for rare diseases. The company offers ALKINDI SPRINKLE, a replacement therapy for adrenocortical insufficiency in children under 17 years of age; Carglumic Acid for the treatment of acute and chronic hyperammonemia due to N-acetylglutamate Synthase deficiency; Betaine Anhydrous for the treatment of homocystinuria; and Nitisinone for the treatment of tyrosinemia type 1. It also provides Zeneo hydrocortisone autoinjector for the treatment of adrenal crisis. Eton Pharmaceuticals, Inc. was incorporated in 2017 and is based in Deer Park, Illinois.

Aquestive Therapeutics, Inc. operates as a pharmaceutical company in the United States and internationally. The company markets Sympazan, an oral soluble film formulation of clobazam for the treatment of lennox-gastaut syndrome; Suboxone, a sublingual film formulation of buprenorphine and naloxone for the treatment of opioid dependence; Zuplenz, an oral soluble film formulation of ondansetron for the treatment of nausea and vomiting associated with chemotherapy and post-operative recovery; and Azstarys, a once-daily product for the treatment of attention deficit hyperactivity disorder. Its proprietary product candidates comprise Libervant, a buccal soluble film formulation of diazepam for the treatment of seizures; KYNMOBI, a sublingual film formulation of apomorphine for the treatment of episodic off-periods in Parkinson's disease; and Exservan, an oral soluble film formulation of riluzole for the treatment of amyotrophic lateral sclerosis. The company's proprietary pipeline of complex molecule product includes AQST-108, a sublingual film formulation delivering systemic epinephrine for the treatment of conditions other than anaphylaxis; and Anaphylm, an epinephrine sublingual film for the emergency treatment of allergic reactions, including anaphylaxis. In addition, it develops Adrenaverse, an epinephrine prodrug platform. The company was incorporated in 2004 and is headquartered in Warren, New Jersey.

ProQR Therapeutics N.V., a biotechnology company, focuses on the discovery and development of novel therapeutic medicines. The company's products pipeline includes AX-0810 for cholestatic diseases targeting Na-taurocholate cotransporting polypeptide (NTCP); and AX-1412 for cardiovascular diseases (CVDs) targeting Beta-1,4-galactosyltransferase 1 (B4GALT1). It also develops various other early-stage research programs, including AX-1005 for undisclosed targets in CVDs; AX-2402, which focuses on Rett syndrome; AX-2911 for nonalcoholic steatohepatitis (NASH); AX-0601 for obesity and Type 2 diabetes; and AX-9115 for rare metabolic condition, as well as various other targets. In addition, the company develops Axiomer RNA base-editing platform technology. It has a license agreement with Radboud University Medical Center; Inserm Transfert SA; Ionis Pharmaceuticals, Inc.; Vico Therapeutics B.V.; University of Rochester; and Leiden University Medical Center, as well as license and research collaboration with Eli Lilly and Company for the discovery, development, and commercialization of potential new medicines for genetic disorders in the liver and nervous system. The company was incorporated in 2012 and is headquartered in Leiden, the Netherlands.

Journey Medical Corporation focuses on the development and commercialization of pharmaceutical products for the treatment of dermatological conditions in the United States. The company's marketed products include Qbrexza, a medicated cloth towelette for the treatment of primary axillary hyperhidrosis; Accutane, an oral isotretinoin drug to treat severe recalcitrant nodular acne; and Amzeeq, a topical formulation of minocycline for the treatment of inflammatory lesions of non-nodular moderate to severe acne vulgaris. It also offers Zilxi, a topical minocycline treatment for inflammatory lesions of rosacea; Exelderm cream and solution an antifungal intended for topical use; Targadox, an oral doxycycline drug for adjunctive therapy for severe acne; and Luxamend, a water-based emulsion formulated to provide a moist healing environment for superficial wounds; minor cuts or scrapes; dermal ulcers; donor sites; first- and second-degree burns, including sunburns; and radiation dermatitis. In addition, the company sells sulconazole nitrate cream and solution indicated for the treatment of tinea cruris, tinea corporis, and tinea versicolor; and doxycycline hyclate tablets, as an adjunctive therapy for severe acne. The company was formerly known as Coronado Dermatology, Inc. and changed its name to Journey Medical Corporation. Journey Medical Corporation was incorporated in 2014 and is headquartered in Scottsdale, Arizona. Journey Medical Corporation is a subsidiary of Fortress Biotech, Inc.

Quince Therapeutics, Inc., a biopharmaceutical company, focuses on acquiring, developing, and commercializing therapeutics for patients with debilitating and rare diseases. The company's lead asset candidature comprises EryDex for the treatment of rare pediatric neurodegenerative disease, including A-T, an inherited autosomal recessive neurodegenerative and immunodeficiency disorder caused by mutations in ATM gene. Its AIDE technology platform, a drug/device combination platform that uses an automated process to encapsulate a drug into a patient's own red blood cells, as well as consists of an automated equipment the RCL, a sterile single-use consumable treatment kit comprising EryKit, Syringe Kit, drugs, and process solutions. The company was formerly known as Cortexyme, Inc. and changed its name to Quince Therapeutics, Inc. in August 2022. Quince Therapeutics, Inc. was incorporated in 2012 and is headquartered in South San Francisco, California.

Fortress Biotech, Inc., a biopharmaceutical company, develops dermatology, pharmaceutical, and biotechnology products in the United States. The company markets dermatology products, such as Qbrexza a medicated cloth towelette for the treatment of primary axillary hyperhidrosis; Accutane capsules for severe recalcitrant nodular acne; Amzeeq for severe acne vulgaris; Zilxi, a topical foam; Exelderm cream and solution for topical use; Targadox an oral doxycycline drug for adjunctive therapy for severe acne; Luxamend; sulconazole nitrate cream and solution for tinea cruris and tinea corporis; and doxycycline hyclate tablet. It also develops late stage product candidates, such as intravenous Tramadol for the treatment of post-operative acute pain; CUTX-101, an injection for the treatment of Menkes disease; Cosibelimab for metastatic cancers; Olafertinib for the treatment of patients with EGFR mutation-positive NSCLC; CAEL-101 for the treatment of amyloid light chain amyloidosis; Triplex vaccine for cytomegalovirus; and DFD-29 for the treatment of rosacea. The company's early stage product candidates include Dotinurad for gout; MB-106 for B-cell non-hodgkin lymphoma; MB-101 for glioblastoma; MB-108 for recurrent GBM; MB-109 for refractory glioblastoma; AJ201, an androgen receptor degradation enhancer; BAER-101, a positive allosteric modulator; MB-117 for newly diagnosed x-linked severe combined immunodeficiency; and MB217 for previously transplanted; and MB-110 for RAG1 severe combined immunodeficiency. Its preclinical product candidates comprise Mayo Clinic In Vivo CAR T Platform Technology; AAV-ATP7A gene therapy; AVTS-001 gene therapy; CK-103 BET inhibitor; CEVA-D and CEVA-102; CK-302, an anti-GITR; CK-303, an anti-CAIX; and oligonucleotide platform. The company was formerly known as Coronado Biosciences, Inc. and changed its name to Fortress Biotech, Inc. in April 2015. Fortress Biotech, Inc. was incorporated in 2006 and is based in Bay Harbor Islands, Florida.

Hoth Therapeutics, Inc., a clinical-stage biopharmaceutical company, focuses on developing therapies for unmet medical needs. It develops HT-001, a topical formulation in Phase II clinical trial for the treatment of patients with rash and skin disorders associated with initial and repeat courses of tyrosine kinase epidermal growth factor receptor inhibitor therapy; HT-KIT to treat mast-cell derived cancers and anaphylaxis; HT-TBI to treat traumatic brain injury and ischemic stroke; HT-ALZ for the treatment and/or prevention of Alzheimer's or other neuroinflammatory diseases; HT-004 for treatment of asthma and allergies using inhalational administration; and HT-003 for the treatment of acne and psoriasis, as well as inflammatory bowel diseases. The company is also developing BioLexa Platform, a proprietary, patented, drug compound platform in Phase I clinical trial for the treatment of eczema; and HT-005 for treating patients with lupus. The company has license agreements with the George Washington University; Isoprene Pharmaceuticals, Inc.; Virginia Commonwealth University; the North Carolina State University; Chelexa BioSciences, Inc. and the University of Cincinnati; Zylö Therapeutics, Inc.; and Voltron Therapeutics, Inc. It also has a research collaboration agreement with Weill Cornell Medicine to develop HT-003. Hoth Therapeutics, Inc. was incorporated in 2017 and is headquartered in New York, New York.