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Biotech Companies with FDA Approvals in Pipeline for Neurodegenerative Diseases

This group focuses on small-cap biotech companies developing therapies for neurodegenerative diseases, with their pipeline candidates having achieved significant FDA regulatory milestones or clear paths to approval.

AVXL
Mkt Cap: 809.33M

Anavex Life Sciences (AVXL): A Promising Neurodegenerative Disease Innovator with a Strong Pipeline

Anavex Life Sciences (AVXL) is a clinical-stage biopharmaceutical company focused on developing innovative treatments for CNS disorders, including Alzheimer's disease. Its lead candidate, ANAVEX 2-73 (blarcamesine), has shown promising results in clinical trials for Alzheimer's disease and Rett syndrome, and the company submitted a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for ANAVEX 2-73 for the treatment of Alzheimer's disease in November 2024, indicating FDA approvals in the pipeline.

ANVS
Mkt Cap: 39.95M

Annovis Bio: Unpacking the Buntanetap Story and the Path Ahead (NYSE:ANVS)

Annovis Bio (ANVS) is a clinical-stage biotechnology company focused on neurodegenerative diseases, with its lead candidate, Buntanetap, an oral small molecule, showing promising results in Phase 3 Parkinson's disease and Phase 2/3 Alzheimer's disease studies. The company has aligned with the FDA on a development path for Alzheimer's disease, initiating a pivotal Phase 3 trial in February 2025, with the potential for an NDA filing based on 6-month symptomatic data, demonstrating FDA approvals in the pipeline.

INMB
Mkt Cap: 123.71M

INmune Bio: Awaiting the Alzheimer's Verdict That Could Redefine Neuroinflammation Therapy (INMB)

INmune Bio (INMB) is a clinical-stage biotech focused on harnessing the innate immune system, with its most significant near-term catalyst being the topline cognitive data readout from the Phase 2 MINDFuL trial of XPro in early Alzheimer's disease patients. The company anticipates an End-of-Phase 2 meeting with the FDA in Q4 2026 to plan a pivotal Phase 3 trial and expects to be eligible for Breakthrough Therapy designation in 2025, indicating FDA approvals in the pipeline.

CTNM
Mkt Cap: 106.33M

Contineum Therapeutics: Unpacking the Pipeline and Financial Runway Ahead of Key Data Readouts (CTNM)

Contineum Therapeutics (CTNM) is a clinical-stage biotech focused on neuroscience, inflammation, and immunology, with its lead wholly-owned asset, PIPE-791, advancing in Phase 1b trials for chronic pain and PET imaging. The partnered asset, PIPE-307, recently completed enrollment in its Phase 2 RRMS trial, with top-line data anticipated in H2 2025, demonstrating FDA approvals in the pipeline as it progresses through clinical development towards potential regulatory submissions.

Biotech Companies with FDA Approvals in Pipeline for Rare Diseases

This group highlights small-cap biotech companies developing therapies for various rare diseases, with their pipeline candidates having achieved significant FDA regulatory milestones or clear paths to approval.

CAPR
Mkt Cap: 509.64M

Capricor Therapeutics (CAPR): Pioneering Transformative Therapies for Rare Diseases

Capricor Therapeutics (CAPR) is a clinical-stage biotechnology company developing cell and exosome-based therapeutics for rare diseases, notably Duchenne muscular dystrophy (DMD). Its lead product candidate, deramiocel, received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) in 2016, and the FDA accepted the company's Biologics License Application (BLA) for deramiocel in 2024, granting it Priority Review, indicating FDA approvals in the pipeline.

BCRX
Mkt Cap: 1.96B

BioCryst Pharmaceuticals (BCRX): Navigating the Rare Disease Landscape with Innovation and Execution

BioCryst Pharmaceuticals (BCRX) is a global biotechnology company focused on rare diseases, with its oral, once-daily ORLADEYO for hereditary angioedema (HAE) already approved. The company is on track to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) this year to expand the ORLADEYO label to include children with HAE aged 2 to 11 years, demonstrating FDA approvals in the pipeline.

BNTC
Mkt Cap: 297.94M

Benitec Biopharma's Transformative "Silence and Replace" Technology Offers Hope for Genetic Disorder Patients

Benitec Biopharma (BNTC) is a clinical-stage biotechnology company pioneering gene therapy innovation with its proprietary 'Silence and Replace' platform. Its lead investigational product, BB-301, targets Oculopharyngeal Muscular Dystrophy (OPMD), a rare genetic disorder, and received clearance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1b/2a clinical trial in 2023, indicating FDA approvals in the pipeline.

RCKT
Mkt Cap: 272.58M

Rocket Pharmaceuticals: Gene Therapy Ambition Meets Clinical Reality ($RCKT)

Rocket Pharmaceuticals (RCKT) is a late-stage biotechnology company developing gene therapies for rare and devastating diseases. The company has initiated regulatory filings, including BLA submissions for RP-L201 (LAD-I) and RP-L102 (Fanconi Anemia), and the FDA accepted the BLA for RP-L201 with priority review in September 2023, indicating FDA approvals in the pipeline.

LQDA
Mkt Cap: 1.12B

Liquidia Corporation (NASDAQ:LQDA): A Promising Horizon in Rare Cardiopulmonary Treatments

Liquidia Corporation (LQDA) is a biopharmaceutical company developing therapies for rare cardiopulmonary diseases, with its lead product candidate, YUTREPIA (treprostinil) inhalation powder, receiving tentative approval from the FDA in August 2024 for the treatment of both PAH and PH-ILD. This milestone positions the company for potential final approval and commercialization, indicating FDA approvals in the pipeline.

Biotech Companies with FDA Approvals in Pipeline for Oncology

This group includes small-cap biotech companies focused on developing novel cancer therapies, with their pipeline candidates having achieved significant FDA regulatory milestones or clear paths to approval.

PYXS
Mkt Cap: 68.14M

Pyxis Oncology (PYXS): Building a Promising Pipeline to Defeat Difficult-to-Treat Cancers

Pyxis Oncology (PYXS) is a clinical-stage biopharmaceutical company developing next-generation therapeutics to target challenging cancers. Its lead ADC candidate, PYX-201, is currently being evaluated in a Phase 1 dose-escalation trial, and the company recently announced a clinical trial collaboration agreement with Merck to evaluate the combination of PYX-201 and KEYTRUDA, indicating FDA approvals in the pipeline as it progresses through clinical development.

BCAX
Mkt Cap: 475.01M

Bicara Therapeutics: Unpacking the Bifunctional Strategy and FORTIFI-HN01 Momentum (BCAX)

Bicara Therapeutics (BCAX) is a clinical-stage biopharmaceutical company pioneering first-in-class bifunctional antibodies for solid tumors, with its lead candidate ficerafusp alfa. Bicara has initiated its pivotal FORTIFI-HN01 Phase 2/3 trial for ficerafusp alfa in combination with pembrolizumab in 1L R/M HNSCC, representing a critical step towards potential regulatory approval and indicating FDA approvals in the pipeline.

CTMX
Mkt Cap: 354.47M

CytomX Therapeutics: Unmasking Value in Oncology with Promising Early Data (CTMX)

CytomX Therapeutics (CTMX) is pioneering a novel class of conditionally activated biologics using its proprietary PROBODY platform for oncology. The company recently reported positive interim Phase 1a data for its lead wholly-owned program, CX-2051, an EpCAM-directed PROBODY ADC, and plans for Phase 2 initiation in the first half of 2026, indicating FDA approvals in the pipeline.

JANX
Mkt Cap: 1.37B

Janux Therapeutics: Unlocking Tumor-Activated Immunotherapy with Promising Early Data (NASDAQ:JANX)

Janux Therapeutics (JANX) is a clinical-stage biopharmaceutical company leveraging its proprietary TRACTr and TRACIr platforms to develop tumor-activated immunotherapies. Early clinical data for lead candidates JANX007 and JANX008 demonstrate encouraging anti-tumor activity and favorable safety profiles, supporting progression to expansion studies, indicating FDA approvals in the pipeline as they advance towards later-stage trials.

TYRA
Mkt Cap: 483.92M

TYRA Biosciences: Precision, Pipeline, and the Promise of FGFR Targeting ($TYRA)

TYRA Biosciences (TYRA) is a clinical-stage biotech leveraging its proprietary SNÅP platform to develop highly selective Fibroblast Growth Factor Receptor (FGFR) inhibitors for oncology and skeletal conditions. The lead program, TYRA-300, is advancing into three Phase 2 studies in Q2 2025, building on promising interim Phase 1/2 data, indicating FDA approvals in the pipeline as it progresses through clinical development.

SNDX
Mkt Cap: 777.87M

Syndax Pharmaceuticals: Navigating the Oncology Landscape with Innovative Therapies

Syndax Pharmaceuticals (SNDX) is a commercial-stage biopharmaceutical company with recent FDA approvals for Revuforj (revumenib) and Niktimvo (axatilimab-csfr). The company is now focused on expanding the label for Revuforj, with plans to submit a supplemental new drug application (sNDA) in the second quarter of 2025 for the treatment of R/R acute myeloid leukemia (AML) with a nucleophosmin 1 (NPM1) mutation, indicating FDA approvals in the pipeline.

FHTX
Mkt Cap: 269.79M

Foghorn Therapeutics Inc. (FHTX): Pioneering a New Class of Precision Medicines

Foghorn Therapeutics Inc. (FHTX) is a clinical-stage biotechnology company pioneering a new class of medicines to treat serious diseases by correcting abnormal gene expression, with an initial focus on precision oncology therapies. Its lead candidate, FHD-909, is a first-in-class oral selective SMARCA2 (BRM) inhibitor being evaluated in a Phase 1 clinical trial for SMARCA4 (BRG1) mutated cancers, indicating FDA approvals in the pipeline as it progresses through clinical development.

XNCR
Mkt Cap: 579.33M

Xencor: Engineering Antibodies for Growth and Navigating the Clinical Gauntlet (NASDAQ:XNCR)

Xencor (XNCR) leverages its proprietary XmAb® protein engineering platform to develop differentiated antibody and cytokine therapeutics, with a strategic focus on bispecific T-cell engagers for solid tumors. The company is advancing several wholly-owned programs in Phase 1 clinical studies, indicating FDA approvals in the pipeline as they progress towards later-stage trials and potential regulatory submissions.

TSBX
Mkt Cap: 8.16M

Turnstone Biologics: A Strategic Pivot in Pursuit of Stockholder Value (TSBX)

Turnstone Biologics (TSBX) is a clinical-stage biotechnology company revolutionizing solid tumor treatment with a focus on developing a differentiated approach to tumor-infiltrating lymphocyte (TIL) therapy. Its lead product candidate, TIDAL-01, is progressing through three ongoing Phase 1 clinical trials, with positive initial data reported, indicating FDA approvals in the pipeline as it advances through clinical development.

Biotech Companies with FDA Approvals in Pipeline for Metabolic and Inflammatory Diseases

This group features small-cap biotech companies developing therapies for metabolic and inflammatory diseases, with their pipeline candidates having achieved significant FDA regulatory milestones or clear paths to approval.

MBX
Mkt Cap: 359.31M

MBX Biosciences: Precision Peptides Poised for Catalysts with a Strong Cash Runway (NASDAQ:MBX)

MBX Biosciences (MBX) is a clinical-stage biopharmaceutical company leveraging its proprietary Precision Endocrine Peptide (PEP™) platform to develop novel, long-acting therapies for endocrine and metabolic disorders. The company is advancing a pipeline of three lead candidates, with an IND submission anticipated in Q2 2025 for MBX 4291 for obesity, indicating FDA approvals in the pipeline as it progresses through clinical development.

VTVT
Mkt Cap: 46.29M

vTv Therapeutics Inc. (NASDAQ:VTVT): A Small Biotech with a Big Pipeline of Innovative Therapies

vTv Therapeutics Inc. (VTVT) is a clinical-stage biopharmaceutical company focused on developing novel small molecule drug candidates to treat metabolic and inflammatory diseases. Its lead program, cadisegliatin, for type 1 diabetes (T1D), was granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) in 2021, indicating FDA approvals in the pipeline despite a recent clinical hold.

Biotech Companies with FDA Approvals in Pipeline for Gene Editing and Cell Therapies

This group includes small-cap biotech companies focused on advanced gene editing and cell therapies, with their pipeline candidates having achieved significant FDA regulatory milestones or clear paths to approval.

FDMT
Mkt Cap: 177.89M

4D Molecular Therapeutics: Pioneering Genetic Medicines for Ophthalmology and Pulmonology

4D Molecular Therapeutics (FDMT) is a late-stage biotechnology company focused on genetic medicines, with its lead product candidate, 4D-150, for wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME). The company is on track to initiate the pivotal 4FRONT Phase 3 program in 2025, and has aligned with the FDA on a path forward for a single Phase 3 trial for 4D-150 in DME, indicating FDA approvals in the pipeline.

DTIL
Mkt Cap: 47.68M

Precision BioSciences: ARCUS Platform Drives Pipeline Acceleration Amidst Financial Headwinds (DTIL)

Precision BioSciences (DTIL) is a clinical-stage gene editing company focused on developing in vivo therapies using its proprietary ARCUS platform. Its lead wholly-owned program, PBGENE-HBV, for chronic Hepatitis B, received FDA Fast Track designation in April 2025, and the company plans to accelerate PBGENE-DMD for Duchenne muscular dystrophy towards an IND/CTA filing in 2025, indicating FDA approvals in the pipeline.

ADVM
Mkt Cap: 48.47M

Adverum Biotechnologies: Gene Therapy's Shot at Wet AMD, Fueled by Data, Facing Funding Headwinds (ADVM)

Adverum Biotechnologies (ADVM) is pioneering a one-time intravitreal gene therapy, Ixo-vec, for wet age-related macular degeneration (wet AMD). The company has initiated ARTEMIS, the first of two planned Phase 3 trials for Ixo-vec, and Ixo-vec has received FDA Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations, indicating FDA approvals in the pipeline.

CABA
Mkt Cap: 141.21M

Cabaletta Bio: Resetting Autoimmunity with CAR T, Poised for Pivotal Myositis Data (CABA)

Cabaletta Bio (CABA) is a clinical-stage biotechnology company pioneering engineered T cell therapies, primarily through its CD19-directed CAR T candidate, rese-cel, for autoimmune diseases. The company has received significant regulatory designations for rese-cel, including Fast Track, Orphan Drug, and RMAT designation for myositis, and plans to initiate two registrational cohorts for myositis in 2025, indicating FDA approvals in the pipeline.

FBLG
Mkt Cap: 23.65M

FibroBiologics: Unlocking Fibroblast Potential Amidst Funding Headwinds (NASDAQ:FBLG)

FibroBiologics (FBLG) is a clinical-stage biotechnology company pioneering fibroblast-based cell therapies for chronic diseases. Its pipeline includes lead candidates like CYWC628 for wound healing, which is entering Phase 1/2 in Australia in 2H 2025, and CYMS101 for MS, which completed Phase 1. The company plans to file an IND application for a Phase 1/2 trial for CYMS101 in the United States as funding allows, indicating FDA approvals in the pipeline.

Biotech Companies with FDA Approvals in Pipeline for Diverse Therapeutic Areas

This group includes small-cap biotech companies with pipeline candidates addressing a range of therapeutic areas, where their products have achieved significant FDA regulatory milestones or are on a clear path to approval.

ABVC
Mkt Cap: 43.98M

ABVC BioPharma, Inc. (NASDAQ:ABVC): Revolutionizing Healthcare with Innovative Botanical-Derived Treatments

ABVC BioPharma, Inc. (ABVC) is a clinical-stage biopharmaceutical company developing innovative therapeutic solutions in ophthalmology, central nervous system (CNS), and oncology/hematology. Its lead drug candidate, ABV-1504, has completed successful Phase II clinical trials for major depressive disorder (MDD), and the company is planning to out-license this new chemical entity (NCE) drug for further development and commercialization, indicating FDA approvals in the pipeline.

XBIT
Mkt Cap: 85.37M

XBiotech Inc. (XBIT): A Pioneering Biotech Navigating the Frontiers of True Human Antibodies

XBiotech Inc. (XBIT) is a pre-market biopharmaceutical company developing True Human monoclonal antibodies for various diseases, focusing on interleukin-1 alpha (IL-1α). The company has brought one anti-IL-1α candidate into clinical studies in oncology and rheumatology, and another into a Phase I study for a neurological indication, indicating FDA approvals in the pipeline as these candidates progress through clinical development.

INBX
Mkt Cap: 208.89M

Inhibrx Biosciences: Unpacking the Post-Spin-Off Pipeline and Technology Edge (NASDAQ:INBX)

Inhibrx Biosciences (INBX) has transformed into a focused, clinical-stage biopharmaceutical company centered on its proprietary protein engineering platforms and clinical candidates ozekibart (INBRX-109) and INBRX-106. Key clinical data readouts for both ozekibart (in chondrosarcoma, Ewing sarcoma, and colorectal cancer) and INBRX-106 (in NSCLC and HNSCC) are anticipated in the second half of 2025, indicating FDA approvals in the pipeline.

ABCL
Mkt Cap: 1.08B

AbCellera: Pivoting to Pipeline, Backed by Tech and Capital (ABCL)

AbCellera (ABCL) is strategically transitioning into a clinical-stage biotech, focusing on building a proprietary pipeline of antibody therapeutics. The first two wholly-owned programs, ABCL635 (targeting NK3R for VMS) and ABCL575 (an OX40 ligand antagonist), are on track for CTA filings in Q2 2025 and Phase 1 clinical trial starts in Q3 2025, indicating FDA approvals in the pipeline as they enter clinical development.

IBIO
Mkt Cap: 12.25M

IBIO's AI-Powered Transformation: Unpacking the Engine and Pipeline Potential (NASDAQ:IBIO)

IBIO (IBIO) has completed a strategic transformation to focus entirely on becoming an AI-enabled preclinical biotechnology company developing precision antibody therapies. The company is actively building a preclinical pipeline, notably in obesity/cardiometabolic diseases and immuno-oncology, leveraging its AI Drug Discovery Platform to identify and optimize antibodies, indicating FDA approvals in the pipeline as these candidates advance towards clinical trials.

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