Confidential & Proprietary · Genflare, Inc.
Target Account Universe · Full Combined View · Q2 2025
Target Account Universe · Full Combined View · Q2 2025
Commercially active specialty pharma companies segmented by commercial maturity stage — the dimension that drives Genflare's go-to-market motion and SIGNAL product mapping.
| # | Company Ticker |
2024 Revenue Actual / Est. |
Market Cap Approx. 2025 |
HQ | CEO | In-Market Products | Treatment Area | Patient Pop. (US Est.) | Employees | Links |
|---|---|---|---|---|---|---|---|---|---|---|
| T1·01 | Alkermes ALKS |
~$1.50B 2024 guidance mid |
~$4.0B |
Dublin, IE Waltham, MA (US ops) |
Richard Pops Chairman & CEO |
Vivitrol® — OUD / AUDAristada® — SchizophreniaLybalvi® — Schizophrenia / Bipolar IVumerity® — Multiple Sclerosis |
PsychiatryNeuroscienceAddictionMS |
~38M AUD/OUD · Schizophrenia · MS · Bipolar |
~1,200 | ↗ Website ↗ Annual Report |
| T1·02 | Lantheus Holdings LNTH |
~$1.45B 2024 actual |
~$3.0B |
N. Billerica, MA | Brian Markison President & CEO |
PYLARIFY® — Prostate Cancer ImagingDEFINITY® — Cardiac ImagingAzedra® — Rare Adrenal Tumors |
Oncology DxNuclear MedicineCardiology |
~18M Prostate Ca · Cardiac imaging |
~700 | ↗ Website ↗ Annual Report |
| T1·03 | ACADIA Pharma ACAD |
~$870M 2024 actual |
~$2.0B |
San Diego, CA | Steve Davis President & CEO |
Nuplazid® — Parkinson's Disease PsychosisDaybue™ — Rett Syndrome |
PsychiatryNeurologyRare Disease |
~510K PDP ~500K · Rett ~8K US |
~600 | ↗ Website ↗ Annual Report |
| T1·04 | PTC Therapeutics PTCT |
~$800M 2024 mid guidance |
~$2.5B |
S. Plainfield, NJ | Matthew Klein, MD President & CEO |
Emflaza® — Duchenne MDTranslarna™ — Duchenne (ex-US)Kebilidi® — AADC Deficiency |
Rare DiseaseNeuromuscularGene Therapy |
~16K Duchenne ~15K US · AADC ~1K |
~1,000 | ↗ Website ↗ Annual Report |
| T1·05 | Harmony Biosciences HRMY |
~$730M 2024 est. |
~$1.2B |
Plymouth Meeting, PA | Jeffrey Dayno, MD President & CEO |
Wakix® — Narcolepsy (EDS & Cataplexy) |
Sleep MedicineRare NeurologyCNS |
~3.4M Narcolepsy ~200K · EDS ~3M |
~550 | ↗ Website ↗ Annual Report |
| T1·06 | Pacira BioSciences PCRX |
~$710M 2024 actual |
~$1.2B |
Parsippany, NJ | Frank D. Lee CEO |
EXPAREL® — Surgical Pain Mgmtiovera°® — Perioperative Pain |
Non-Opioid PainSurgical RecoveryMSK |
~50M ~50M US surgical procedures/yr |
~1,000 | ↗ Website ↗ Annual Report |
| T1·07 | Supernus Pharma SUPN |
~$700M 2024 est. |
~$1.5B |
Rockville, MD | Jack Khattar President & CEO |
Qelbree® — ADHDOxtellar XR® — EpilepsyTrokendi XR® — Epilepsy / Migraine |
NeurologyADHDEpilepsy |
~14M ADHD ~10M · Epilepsy ~3.4M |
~700 | ↗ Website ↗ Annual Report |
| T1·08 | Collegium Pharma COLL |
~$680M 2024 actual |
~$1.7B |
Stoughton, MA | Michael Heffernan Chairman & Interim CEO |
Xtampza® ER — Chronic PainJornay PM® — ADHDNucynta® — Chronic Pain |
PainADHDCNS |
~60M Chronic pain ~50M · ADHD ~10M |
~600 | ↗ Website ↗ Annual Report |
| T1·09 | Corcept Therapeutics CORT |
~$665M 2024 actual; '25 $900–950M |
~$3.5B |
Redwood City, CA | Joseph K. Belanoff, MD CEO & Co-Founder |
Korlym® — Cushing's Syndrome Hyperglycemia |
EndocrinologyRare MetabolicOncology |
~80K Cushing's syndrome ~70–80K US |
~600 | ↗ Website ↗ Annual Report |
| T1·10 | Amphastar Pharma AMPH |
~$620M 2024 actual |
~$2.0B |
Rancho Cucamonga, CA | Jack Zhang, PhD CEO & Co-Founder |
BAQSIMI® — Severe HypoglycemiaPrimatene® Mist — AsthmaEnoxaparin — DVT/PE Prevention |
EndocrinologyRespiratoryHematology |
~30M T1D · Asthma · DVT/PE |
~2,200 | ↗ Website ↗ Annual Report |
| T1·11 | ANI Pharmaceuticals ANIP |
~$600M 2024 actual |
~$1.3B |
Baudette, MN | Nikhil Lalwani President & CEO |
Cortrophin® Gel — Autoimmune / InflammatoryIluvien® — Diabetic Macular Edema |
Rare DiseaseRheumatologyNephrologyOphthalmology |
~750K ACTH-eligible · DME |
~700 | ↗ Website ↗ Annual Report |
| T1·12 | Ultragenyx Pharma RARE |
~$560M 2024 actual |
~$2.5B |
Novato, CA | Emil D. Kakkis, MD, PhD President & CEO |
Crysvita® — XLH / TIODojolvi® — LC-FAODEvkeeza® — HoFH |
Ultra-Rare DiseaseMetabolicBone Disease |
~95K XLH · LC-FAOD · HoFH |
~800 | ↗ Website ↗ Annual Report |
| T1·13 | Ionis Pharmaceuticals IONS |
~$900M 2024 actual |
~$5.0B |
Carlsbad, CA | Brett Monia, PhD President & CEO |
Wainua® — hATTR AmyloidosisTegsedi® — hATTR PolyneuropathySpinraza® — SMA (w/ Biogen) |
Rare DiseaseCardiovascularNeurologyRNA Tx |
~170K SMA · hATTR · Rare CV |
~600 | ↗ Website ↗ Annual Report |
| # | Company Ticker |
2024 Revenue Actual / Est. |
Market Cap Approx. 2025 |
HQ | CEO | In-Market Products | Treatment Area | Patient Pop. (US Est.) | Employees | Links |
|---|---|---|---|---|---|---|---|---|---|---|
| T2·01 | Catalyst Pharma CPRX |
~$492M 2024 actual |
~$2.3B |
Coral Gables, FL | Richard J. Daly President & CEO |
Firdapse® — Lambert-Eaton Myasthenic SyndromeAgamree® — Duchenne MD (launched 2023) |
Rare NeuromuscularNeurology |
~18K LEMS ~3K · Duchenne ~15K US |
~320 | ↗ Website ↗ Annual Report |
| T2·02 | TG Therapeutics TGTX |
~$660M 2024 est. |
~$3.0B |
New York, NY | Michael Weiss Exec Chairman & CEO |
Briumvi® — Relapsing MS (launched Jan 2023) |
Multiple SclerosisImmunology |
~1.0M Relapsing MS ~900K–1M US |
~200 | ↗ Website ↗ Annual Report |
| T2·03 | Blueprint Medicines BPMC |
~$660M 2024 est. |
~$4.0B |
Cambridge, MA | Kate Haviland President & CEO |
Ayvakit® — Systemic Mastocytosis / GIST |
OncologyRare / Mast Cell |
~50K Systemic mastocytosis ~32K · GIST ~15K/yr |
~750 | ↗ Website ↗ Annual Report |
| T2·04 | BioCryst Pharma BCRX |
~$451M 2024 actual; '25 $560–575M |
~$2.0B |
Durham, NC | Jon P. Stonehouse President & CEO |
Orladeyo® — Hereditary Angioedema |
Rare ImmunologyHAE |
~25K HAE ~25K US |
~500 | ↗ Website ↗ Annual Report |
| T2·05 | Axsome Therapeutics AXSM |
~$386M 2024 actual (+88% YoY) |
~$3.0B |
New York, NY | Herriot Tabuteau, MD CEO & Co-Founder |
Auvelity® — Major Depressive DisorderSunosi® — Narcolepsy / OSA-EDSSymbravo™ — Acute Migraine (2025) |
PsychiatryCNSMigraineSleep |
~60M MDD · OSA-EDS · Migraine · Narcolepsy |
~300 | ↗ Website ↗ Annual Report |
| T2·06 | Krystal Biotech KRYS |
~$300M 2024 est.; strong ramp |
~$4.5B |
Pittsburgh, PA | Krish S. Krishnan President & CEO |
Vyjuvek® — Dystrophic Epidermolysis Bullosa (launched 2023) |
Rare GeneticDermatologyGene Therapy |
~3,500 DEB (dystrophic EB) ~3,500 US |
~200 | ↗ Website ↗ Annual Report |
| T2·07 | Sage Therapeutics SAGE |
~$470M 2024 est. incl. collaboration |
~$1.0B |
Cambridge, MA | Barry Greene President & CEO |
Zurzuvae™ — MDD / PPD (co-promote w/ Biogen)Zulresso® — Postpartum Depression (IV) |
PsychiatryNeuroscienceWomen's Health |
~22M MDD ~21M US · PPD ~600K new/yr |
~450 | ↗ Website ↗ Annual Report |
| T2·08 | Travere Therapeutics TVTX |
~$400M 2024 est.; growing |
~$1.0B |
San Diego, CA | Eric Dube, PhD President & CEO |
Filspari® (sparsentan) — IgA Nephropathy (launched 2023)Thiola® EC — Cystinuria |
Rare RenalNephrology |
~200K IgAN ~150K US · Cystinuria ~20K |
~450 | ↗ Website ↗ Annual Report |
| T2·09 | Agios Pharma AGIO |
~$300M 2024 est. |
~$2.0B |
Cambridge, MA | Brian Goff President & CEO |
Pyrukynd® (mitapivat) — Pyruvate Kinase Deficiency / Thalassemia |
Rare HematologyHemolytic Anemia |
~50K PKD ~3K US · Thalassemia ~1K US |
~350 | ↗ Website ↗ Annual Report |
| T2·10 | Insmed INSM |
~$500M 2024 est.; brensocatib launch |
~$10B Above range; watch list |
Bridgewater, NJ | Will Lewis President & CEO |
ARIKAYCE® — MAC Lung DiseaseBrensocatib — Bronchiectasis (launch 2025) |
Rare PulmonaryRespiratoryInfectious Disease |
~400K MAC lung ~75K US · Bronchiectasis ~300K |
~1,000 | ↗ Website ↗ Annual Report |
| T2·11 | Rhythm Pharma RYTM |
~$170M 2024 est.; rapid growth |
~$1.5B |
Boston, MA | David Meeker, MD Chairman & CEO |
Imcivree® (setmelanotide) — Rare Genetic Obesity |
Rare MetabolicRare ObesityEndocrinology |
~50K MC4R pathway obesity disorders ~50K US eligible |
~300 | ↗ Website ↗ Annual Report |
| T2·12 | Arcutis Biotherapeutics ARQT |
~$200M 2024 est. |
~$800M |
Westlake Village, CA | Frank Watanabe President & CEO |
Zoryve® cream (roflumilast) — Plaque Psoriasis / Seborrheic DermatitisZoryve® foam — Scalp & Body Psoriasis |
DermatologyImmunology |
~10M Plaque psoriasis ~8M US · Seb derm ~15M |
~350 | ↗ Website ↗ Annual Report |
| T2·13 | Halozyme Therapeutics HALO |
~$800M 2024 est.; royalty-driven |
~$5.0B At upper mktcap bound |
San Diego, CA | Helen Torley, MB ChB President & CEO |
ENHANZE® Technology Platform — SC drug delivery (licensed to pharma partners) |
Drug Delivery PlatformOncologyImmunology |
N/A Royalty/platform model — indirect patient exposure |
~350 | ↗ Website ↗ Annual Report |
| # | Company Ticker |
2024 Revenue Actual / Est. |
Market Cap Approx. 2025 |
HQ | CEO | In-Market Products | Treatment Area | Patient Pop. (US Est.) | Employees | Links |
|---|---|---|---|---|---|---|---|---|---|---|
| T3·01 | Verona Pharma VRNA |
~$100M 2024 est.; launched mid-2024 |
~$3.0B |
Raleigh, NC | David Zaccardelli President & CEO |
Ohtuvayre® (ensifentrine) — COPD Maintenance (launched Jun 2024) |
RespiratoryCOPD |
~15M COPD ~15M US; first new class in 20 yrs |
~300 | ↗ Website ↗ Annual Report |
| T3·02 | Day One Biopharm. DAWN |
~$120M 2024 est.; launched Apr 2024 |
~$1.5B |
Brisbane, CA | Jeremy Bender, PhD President & CEO |
Ojemda® (tovorafenib) — Pediatric Low-Grade Glioma (launched Apr 2024) |
Pediatric OncologyCNS Tumors |
~4,000 Peds low-grade glioma ~4K new cases/yr US |
~200 | ↗ Website ↗ Annual Report |
| T3·03 | Geron Corporation GERN |
~$100M 2024 est.; launched Jun 2024 |
~$1.5B |
Foster City, CA | Andrew Grethlein President & CEO |
Rytelo® (imetelstat) — Myelodysplastic Syndrome (launched Jun 2024) |
Hematologic OncologyMDS |
~170K MDS ~170K US; ESA-relapsed/refractory |
~250 | ↗ Website ↗ Annual Report |
| T3·04 | Syndax Pharma SNDX |
~$100M 2024 est.; launched Dec 2023 |
~$1.0B |
Waltham, MA | Michael Metzger President & CEO |
Revuforj® (revumenib) — Relapsed/Refractory Leukemia |
Hematologic OncologyLeukemia |
~10K KMT2A-r or NPM1-mutant leukemia ~10K US |
~250 | ↗ Website ↗ Annual Report |
| T3·05 | Phathom Pharma PHAT |
~$60M 2024 est.; very early launch |
~$400M Below range; growth case |
Florham Park, NJ | Terrie Curran President & CEO |
Voquezna® (vonoprazan) — GERD / H. pylori (launched 2023) |
GastroenterologyGI |
~80M GERD ~60M US · H. pylori ~20M |
~300 | ↗ Website ↗ Annual Report |
| T3·06 | Protagonist Therapeutics PTGX |
~$50M 2024 est.; NDA stage |
~$2.0B |
Newark, CA | Dinesh Patel, PhD President & CEO |
Rusfertide — Polycythemia Vera (NDA submitted 2024)Imeciclomab — ITP (Phase 3) |
HematologyRare Blood Disorders |
~200K PV ~200K US |
~200 | ↗ Website ↗ Annual Report |
| T3·07 | Kiniksa Pharmaceuticals KNSA |
~$200M 2024 est. |
~$800M |
Lexington, MA | Sanj K. Patel Chairman & CEO |
Arcalyst® (rilonacept) — Recurrent Pericarditis |
Rare InflammatoryCardiovascularImmunology |
~40K Recurrent pericarditis ~40K US |
~200 | ↗ Website ↗ Annual Report |
| T3·08 | Emergent BioSolutions EBS |
~$900M 2024 est.; restructuring |
~$500M Low end; distressed mktcap |
Gaithersburg, MD | Joe Papa CEO |
NARCAN® Nasal Spray — Opioid Overdose ReversalCYFENDUS® — Anthrax MCMACAM2000® — Smallpox Vaccine |
Public HealthMedical CountermeasuresAddiction |
~100M Opioid OD risk ~6M · Narcan broad OTC access |
~1,700 | ↗ Website ↗ Annual Report |
| T3·09 | Eagle Pharmaceuticals EGRX |
~$220M 2024 est. |
~$400M Below range; niche commercial org |
Woodcliff Lake, NJ | Scott Tarriff CEO |
Ryanodex® — Malignant Hyperthermia / Exertional Heat StrokeBelrapzo® — Hematologic MalignanciesPemfexy® — NSCLC / Mesothelioma |
OncologyHospital SpecialtyCritical Care |
~30K Niche hospital oncology and rare conditions |
~100 | ↗ Website ↗ Annual Report |
| T3·10 | Exelixis EXEL |
~$1.8B Above rev ceiling; oncology org relevant |
~$8.0B Above mktcap range; adjacent |
Alameda, CA | Michael M. Morrissey, PhD President & CEO |
Cabometyx® (cabozantinib) — RCC / HCC / DTC / NETCometriq® — MTC |
OncologyMulti-Tumor TKI |
~80K RCC · HCC · DTC · NET new cases/yr |
~1,000 | ↗ Website ↗ Annual Report |
| T3·11 | BioMarin Pharmaceutical BMRN |
~$2.7B Above criteria; rare disease org highly relevant |
~$14B Above range; adjacent account |
San Rafael, CA | Alexander Hardy President & CEO |
VOXZOGO® — AchondroplasiaNaglazyme® — MPS VIVimizim® — MPS IVAPalynziq® — PKU |
Ultra-Rare DiseaseEnzyme ReplacementSkeletal Conditions |
~30K Multiple ultra-rare genetic conditions |
~3,200 | ↗ Website ↗ Annual Report |
Revenue and market cap figures are estimates based on 2024 annual guidance, earnings releases, and public filings as of Q1–Q2 2025. Market caps are approximate and subject to fluctuation. Patient population estimates reflect combined addressable US populations across all approved indications and may include overlapping populations. Tier assignment reflects commercial maturity at time of research — companies may move between tiers as launches progress or new products reach market. Tier 3 exceptions (Exelixis, BioMarin, Insmed, Halozyme) fall outside one or both core criteria but are included due to highly relevant commercial organization profiles; revenue/mktcap flags noted inline. This list will be updated quarterly as new specialty approvals reach commercial stage. Universe calibration: ~50 new specialty FDA approvals annually replenish this list on a rolling basis.