JP Morgan 2025

Clinical stage biotechnology company discovering and developing allogeneic “off-the-shelf” gamma delta CAR T cell therapies for autoimmune diseases and cancer. Lead candidate ADI-001, a B-cell depleting off-the-shelf gamma-delta CAR T cell therapy, is being evaluated in a Phase 1 study in four autoimmune indications including lupus nephritis (LN), systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and anti-neutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis (AAV), idiopathic inflammatory myopathy (IIM), and stiff person syndrome (SPS). Clinical update from ADI-001 trial in LN is expected in 1H25, and for other autoimmune diseases in 2H25. ADI-270, a highly differentiated armored off-the-shelf gamma delta CAR T cell therapy candidate targeting CD70-positive cancers, is being investigated in renal cell carcinoma (RCC) and preliminary clinical data planned in 1H25. Granted FDA Fast Track designation for ADI-001 in relapsed/refractory class III or class IV LN and for ADI-270 in metastatic/advanced clear cell RCC patients.

Biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on cancer and genetically validated targets in other disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors.

Clinical-stage biotechnology company dedicated to improving the lives of patients suffering from debilitating and life-threatening diseases through the discovery, development, and commercialization of therapies that degrade disease-causing proteins. Arvinas uses its proprietary PROTAC® Discovery Engine platform to engineer proteolysis targeting chimeras, or PROTAC® targeted protein degraders, that are designed to harness the body’s own natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins. In addition to its robust preclinical pipeline of PROTAC® protein degraders against validated and “undruggable” targets, the company is progressing ARV-471 for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer with topline from VERITAC-2 Phase 3 monotherapy clinical trial expected in 4Q24/1Q25.

Clinical-stage biotechnology company developing therapies to precisely engage specific immune cells to fight cancer and chronic viral infections utilizing a proprietary cis-targeting platform. Lead candidate AB248, an IL-2 molecule specifically targeted to CD8+ effector T cells, is currently in an ongoing Phase 1a/1b trial for the treatment of locally advanced or metastatic solid tumors. Initial pharmacokinetic and pharmacodynamic data from the ongoing Phase 1a/1b clinical trial support AB248’s proof of mechanism and activity with a highly differentiated clinical profile. Early data shows potent and selective CD8+ T cell activation without substantial changes to Treg and NK cell numbers and initial evidence of anti-tumor activity, with a generally well-tolerated safety profile. Broader portfolio includes AB821, an IND-ready CD8-targeted IL-21 immunotherapy, and early stage programs targeting CAR-T cells, myeloid cells and CD4+ T cells.

Late-stage biopharmaceutical company focused on discovering, developing and commercializing oral therapies to address the unmet medical needs of patients with serious viral infections. Lead candidate, bemnifosbuvir, a nucleotide polymerase inhibitor, is an oral, direct-acting antiviral drug candidate being evaluated in combination with ruzasvir, an oral NS5A inhibitor, in a Phase 2 trial in treatment-naïve, HCV-infected patients either without cirrhosis or with compensated cirrhosis. Patient enrollment for the trial has been completed and initial results are expected in 4Q24. Atea has built a proprietary nucleos(t)ide prodrug platform to develop novel product candidates to treat single stranded ribonucleic acid, or ssRNA, viruses, which are a prevalent cause of serious viral diseases.

Clinical-stage biopharmaceutical company focused on developing small molecules to restore neuronal health and slow neurodegeneration. Athira aims to alter the course of neurological diseases by advancing its pipeline of drug candidates that modulate the neurotrophic HGF system. Advancing ATH-1105, a next-generation, orally administered, orally-delivered, positive modulator of the neurotrophic HGF system that is in development as a treatment for ALS. ATH-1105 is in a Phase 1 first-in-human, dose escalation study in healthy volunteers trial completion expected by year-end 2024. In preclinical models of ALS, ATH-1105 has been shown to significantly increase survival, enhance motor and nerve function, reduce peripheral nerve demyelination and axon degeneration, and improve neurodegeneration and inflammation. The Phase 1 study is expected to complete by year end 2024 and commencement of dosing of ALS patients is expected in 2025.

Commercial stage biopharmaceutical company applying innovative solutions to the development of medications that address the challenges patients face with current treatment options. Received U.S. FDA approval for LUMRYZ™, the first and only once-at-bedtime oxybate for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy, on May 1, 2023. Also awarded seven years of Orphan Drug Exclusivity concurrent with approval in adults, based on FDA finding of clinical superiority of LUMRYZ relative to currently marketed twice-nightly oxybate products in that once-at-bedtime dosing of LUMRYZ offers a major contribution to patient care over those first-generation products. Launched LUMRYZ on June 5, 2023, and reported more than 1,900 patients on LUMRYZ as of June 30, 2024, and from launch through June 30, 2024, approximately 3,800 patients have enrolled in Avadel’s RYZUP™ patient support services to begin the process of getting LUMRYZ prescriptions fulfilled and shipped. Robust IP protection with 19 Orange Book listed patents with expiry out to early 2042.

Clinical-stage biopharmaceutical company focused on developing targeted therapies for serious liver disease. Potentially best in class lead asset efimosfermin (BOS-580) is a highly-engineered, long-acting, once monthly subcutaneous injection of human fibroblast growth factor 21 (FGF21) for the treatment of metabolic dysfunction associated steatohepatitis (MASH) and in Phase 1B and 2a has shown statistically significant efficacy across all key liver and selected metabolic biomarkers, after only 3 doses, along with an overall positive side effect profile. Expecting to announce Phase 2 data in F2/F3 patients in 4Q24, with Phase 2 Part C extension data, as well as Part D, non-invasive biomarkers and biopsy data in advanced fibrosis patients, expected in 2025.

Clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies that have the potential to provide a deep and durable, perhaps curative, treatment for patients with autoimmune diseases. The CABA™ platform encompasses two strategies: the CARTA (chimeric antigen receptor T cells for autoimmunity) strategy, with CABA-201, a 4-1BB-containing fully human CD19-CAR T, as the lead product candidate being evaluated in the RESET™ (REstoring SElf-Tolerance) clinical trials in myositis, systemic lupus erythematosus, systemic sclerosis, generalized myasthenia gravis and in the RESET-PV™ sub-study within the DesCAARTes™ clinical trial in pemphigus vulgaris, along with the CAART (chimeric autoantibody receptor T cells) strategy, with multiple clinical-stage candidates, including DSG3-CAART for mucosal pemphigus vulgaris and MuSK-CAART for MuSK-associated myasthenia gravis. The expanding CABA™ platform is designed to develop potentially curative therapies that offer deep and durable responses for patients with a broad range of autoimmune diseases. Cabaletta Bio’s headquarters and labs are located in Philadelphia, PA.

Biopharmaceutical company with a mission to develop medicines that meaningfully improve and extend the lives of patients facing deadly diseases. The Company’s most advanced clinical-stage development program, ONC201, is in Phase 3 development for H3 K27M-mutant glioma.   The Company is conducting Phase 1 dose escalation studies of ONC206 to evaluate safety and PK data.

Clinical stage, oncology-focused biopharmaceutical company pioneering a novel class of masked, conditionally activated biologics, powered by its PROBODY® platform, with a pipeline across multiple treatment modalities. CX-904, a T-cell-engaging bispecific antibody targeting to EGFRxCD3 and partnered with Amgen, demonstrated positive data and a favorable safety profile, along with encouraging initial signs of efficacy in advanced pancreatic cancer. CX-2051, an EpCAM-directed ADC with a Topo-1 payload, has potential applicability across multiple EpCAM-expressing epithelial cancers, including CRC, and is anticipated to have Phase 1a data in 1H25. CX-801, an interferon alpha-2b cytokine, with broad potential applicability in traditionally immune-oncology sensitive as well as insensitive tumors, anticipated to have Phase 1a data in 2H25. Strategic collaborations with leaders in oncology such as Amgen, Astellas, Bristol Myers Squibb, Regeneron and Moderna.

Clinical-stage biopharmaceutical company building a portfolio of innovative, first-in-class therapeutic candidates that aim to address a wide spectrum of hematologic diseases by targeting fundamental biological pathways of red blood cell biology, specifically heme biosynthesis and iron homeostasis. Lead therapeutic candidate, bitopertin, is a first-in-class oral inhibitor of GlyT1, which has the potential to be the first disease-modifying treatment for erythropoietic protoporphyria (EPP) and additional indications such as Diamond-Blackfan Anemia (DBA). Disc has two additional pipeline candidates focused on iron modulation – including DISC-0974, an anti-hemojuvelin (HJV) antibody targeting anemias in myelofibrosis and chronic kidney disease driven by high hepcidin, and DISC-3405 targeting Transmembrane Serine Protease 6 (TMPRSS6) for indications characterized by iron overload and excess red blood cells (polycythemia vera, myelodysplastic syndromes).

Clinical-stage oncology company focused on the discovery and development of selective cancer therapies to treat patients across a range of solid tumors. Currently evaluating EO-3021, an anti-Claudin 18.2 ADC, in a Phase 1 study in patients with advanced, unresectable or metastatic solid tumors likely to express Claudin 18.2, including gastric, gastroesophageal, pancreatic or esophageal cancers. In August 2024, announced promising initial data, demonstrating 42.8% confirmed ORR observed in Claudin 18.2-enriched subset of gastric and GEJ cancer. Advancing into dose expansion portion of ongoing Phase 1 study of EO-3021 and expect additional monotherapy data in 1H 2025. Secured clinical supply agreements to evaluate EO-3021 in combination with ramucirumab and dostarlimab with Lilly and GSK, respectively, and expect to initiate dosing in combination portion by year-end 2024. Also advancing a HER3-targeting ADC for patients with solid tumors that overexpress HER3; expect to nominate a development candidate before year-end.

Commercial-stage global biopharmaceutical company focused on the discovery, development and commercialization of innovative medicines to help improve outcomes for patients with or at risk for cardiovascular and cardiometabolic diseases. The Company developed and is commercializing the only U.S. Food and Drug Administration (FDA) approved oral, once-daily, non-statin medicines for both primary and secondary prevention patients who are at risk for cardiovascular disease and are struggling with elevated low-density lipoprotein cholesterol (LDL-C). These medications are supported by the nearly 14,000 patient CLEAR Cardiovascular Outcomes Trial demonstrating their ability to significantly reduced cardiovascular risk and the risk of heart attack and coronary revascularization. Esperion continues to evolve into a leading global biopharmaceutical company through commercial execution, international partnerships and collaborations and advancement of its pre-clinical pipeline.

EyePoint is committed to developing and commercializing innovative therapeutics to help improve the lives of patients with serious retinal diseases. EyePoint’s pipeline includes DURAVYU™ (f/k/a EYP-1901), an investigational sustained delivery treatment for VEGF-mediated retinal diseases combining vorolanib, a selective and patent-protected tyrosine kinase inhibitor with bioerodible Durasert E™. DURAVYU is currently in Phase 2 clinical trials as a maintenance treatment for wet age-related macular degeneration with first patient dosing in the Phase 3 trials expected in 2024. DURAVYU is also being studied in the Phase 2 VERONA trial for diabetic macular edema with topline data expected in Q1 2025. Additional pipeline programs include EYP-2301, a novel TIE 2 activator being studied as a potential new MOA for treating serious retinal diseases and complement inhibition for geographic atrophy. EyePoint ended Q2 2024 with ~$280 million of cash and equivalents with cash guidance through Phase 3 wet AMD topline data in 2026.

Clinical-stage biotechnology company developing novel vaccines for many of the world’s most threatening infectious diseases and therapies for solid tumor cancers. The Company’s lead clinical program is GEO-CM04S1, a next-generation COVID-19 vaccine for which GeoVax was recently awarded a BARDA-funded contract to sponsor a 10,000-participant Phase 2b clinical trial to evaluate the efficacy of GEO-CM04S1 versus an approved mRNA COVID-19 vaccine. In addition, GEO-CM04S1 is currently in three Phase 2 clinical trials, being evaluated as (1) a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, (2) a booster vaccine in patients with chronic lymphocytic leukemia (CLL) and (3) a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. Also, GeoVax is advancing GEO-MVA, a vaccine against Mpox/Smallpox through cGMP manufacturing in order to potentially assist in the WHO declared global health emergency.  In oncology the lead clinical program is evaluating a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, having recently completed in a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. GeoVax recently announced plans to initiate a Phase 2 trial of Gedeptin® in combination with an immune checkpoint inhibitor, with that study anticipated to initiate in mid-2025. GeoVax has a strong IP portfolio in support of its technologies and product candidates, holding worldwide rights for its technologies and products. The Company’s leadership team has driven significant value creation across multiple life science companies over the past several decades. GeoVax is headquartered in the metropolitan Atlanta, GA area.

Advanced clinical-stage private company developing Padeliporfin Vascular Targeted Photodynamic (VTP) therapy, delivering non-thermal laser light to locally activate Padeliporfin as a targeted treatment for unresectable solid tumors. Preliminary Ph. 3 data achieved CR rate of 77% with well-tolerated safety profile in Low-Grade Upper Tract Urinary Cancer (LG-UTUC); additional interim analysis data expected in 4Q24. Platform technology, with Ph. 1 trial in PDAC expected in 2H24 and additional studies planned for high grade UTUC and NSCLC. Long-standing collaboration with the Weizmann Institute of Science and the Memorial Sloan Kettering Cancer Center.

A leading GI-focused healthcare company and pioneers in the development of LINZESS® (linaclotide), U.S. branded prescription market leader for patients with IBS-C or CIC, which continues to deliver meaningful prescription demand growth in its 12th year. Advancing apraglutide, a next-generation, synthetic GLP-2 analog for SBS-IF with the potential to be standard of care; expect to complete rolling NDA submission in 1Q 2025.

Late clinical-stage biopharmaceutical company developing LNZ100, a preservative-free, single-use, once-daily aceclidine based eye drop, for patients with presbyopia, a condition characterized by inevitable loss of near vision impacting an estimated 128 million people in the United States. In registrational Phase 3 CLARITY study, LNZ100 demonstrated strong tolerability as well as safety profile and showed near vision improvement and statistically significant three-lines or greater improvement in Best Corrected Distance Visual Acuity (BCDVA) at near, without losing one line or more in distance visual acuity. Submitted a New Drug Application (NDA) to the FDA for the treatment of presbyopia with potential for commercial launch as early as 2H2025.

Clinical-stage biopharmaceutical company founded to confront the global brain disease crisis by taking a fundamentally different approach to the way treatments for brain diseases are developed. Current pipeline consists of seven clinical and preclinical neuroscience programs that target novel mechanisms of action for a broad range of underserved neuropsychiatric disorders and neurodegenerative diseases. This work is supported by an integrated suite of translational, clinical, and computational tools to generate insights that can enable precision medicine approaches.

Late-stage biopharmaceutical company developing obicetrapib, an oral, low-dose and once-daily cholesteryl ester transfer protein (CETP) inhibitor, as the preferred LDL-C lowering therapy to be used in patients at risk of CVD for whom existing therapies are not sufficiently effective or well-tolerated. Recently announced topline results of the first pivotal phase 3 trial, BROOKLYN evaluating the effect of obicetrapib on LDL-C levels in patients with HeFH as an adjunct to maximally tolerated lipid-lowering therapy, and currently conducting three additional pivotal Phase 3 clinical trials of obicetrapib: BROADWAY, evaluating the effect of obicetrapib on top of maximally tolerated lipid-lowering therapy in patients with HeFH and/or established atherosclerotic cardiovascular disease; TANDEM, evaluating the FDC of obicetrapib plus ezetimibe in adult patients with HeFH and/or ASCVD or multiple risk factors for ASCVD; and PREVAIL, a CVOT in patients with a history of ASCVD with inadequately controlled LDL-C despite treatment with maximally tolerated lipid-modifying therapies. Recently initiated REMBRANDT, a Phase 3 clinical trial evaluating the FDC of obicetrapib and ezetimibe against placebo on coronary atherosclerotic plaque characteristics in adults with or at high-risk for ASCVD. Full data from BROOKLYN expected at an upcoming medical conference; data from BROADWAY expected in the fourth quarter of 2024; data from TANDEM expected in the first quarter of 2025; and data from PREVAIL expected in 2026.

Biotechnology company leveraging the extraordinary adaptive biology of bats to unlock new insights and accelerate the discovery and development of innovative therapies for humans, with an initial focus on immunology & inflammation and cardiometabolism. Paratus' proprietary platform integrates cell biology, genomics and informatics alongside its unique expertise in bat biology data and comparative biology to compare bats' evolved patterns of disease resistance to humans' patterns of disease development in order to address significant challenges across a spectrum of therapeutic areas. Lead ASC candidate advancing towards DC nomination with IND in late 2026. Backed by top-tier investor syndicate, including Polaris Partners, ARCH Venture Partners, ClavystBio, EcoR1 Capital, Leaps by Bayer, and Alexandria Venture Investments.

Clinical stage biotechnology company focused on generating and developing novel antibody therapeutics selectively targeting toxic misfolded proteins in neurodegenerative diseases such as Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and multiple system atrophy (MSA). Lead candidate, PMN310, is humanized monoclonal antibody designed to selectively bind toxic forms of amyloid beta. ProMIS reported successful top-line data from its first-in-human Phase 1a clinical trial, which showed that PMN310 was safe and well tolerated. The Company expects to initiate a Phase 1b clinical trial in Alzheimer's disease later this year. In July 2024, the Company announced a private placement financing of up to $122.7 million to support its novel antibody therapeutics for AD, ALS and MSA.

Clinical-stage biotechnology company translating scientific advances into transformative therapies for patients with devastating rare diseases. Rallybio has built a broad pipeline of promising product candidates aimed at addressing diseases with unmet medical need in areas of maternal fetal health, complement dysregulation, hematology, and metabolic disorders. Rallybio is advancing two clinical stage programs: RLYB212, an anti-HPA-1a antibody for the prevention of fetal and neonatal alloimmune thrombocytopenia (FNAIT) and RLYB116, an inhibitor of complement component 5 (C5), with the potential to treat several diseases of complement dysregulation. Additional preclinical programs include RLYB332, a long-acting MTP-2 inhibitor for the treatment of diseases of iron overload, an ENPP1 Inhibitor for the treatment of Hypophosphatasia (HPP), and RLYB114, a C5 inhibitor for ophthalmic use.

Clinical-stage genetic medicines company using precision delivery to power the next wave of mRNA and gene correction therapeutics. ReCode’s Selective Organ Targeting (SORT) lipid nanoparticle (LNP) platform enables highly precise and targeted delivery of genetic medicines directly to the organs and cells implicated in disease, enabling improved efficacy and potency. Lead programs include RCT2100 for the treatment of the 10-13 percent of cystic fibrosis patients who do not respond to currently approved CFTR modulators, as well as RCT1100 for the treatment of primary ciliary dyskinesia caused by pathogenic mutations in the DNAI1 gene. ReCode is expanding its pipeline to develop potential therapies for other rare and common genetic diseases including musculoskeletal, central nervous system, liver and infectious disease indications.

Clinical-stage biotechnology company developing novel small molecule therapies designed to reprogram RNA processing and treat disease. The company’s REMaster™ technology platform facilitates RNA processing pattern identification, leveraging these learnings to modulate gene expression. Remix's innovative therapeutic approach has the potential to alter the way genes are read from the genome, to correct, enhance, or eliminate the gene message, thereby addressing disease drivers at their origin.

Clinical-stage biopharmaceutical company pioneering engineered macrophage cell therapy for inflammatory organ diseases. Proprietary platform engineers pro-regenerative macrophages for optimized safety, persistence and efficacy. Lead candidate RTX001 for decompensated liver cirrhosis has demonstrated more potent anti-fibrotic and anti-inflammatory effects relative to non-engineered macrophages in both in vitro and in vivo preclinical studies. RTX001 clinical development program features FIH Phase 1/2 EMERALD study, on track to initiate in 2024 following UK MHRA CTA approval, and ongoing OPAL natural history study. Platform expansion provides opportunities to explore ex-hepatic indications where engineered macrophages have therapeutic potential independently and through strategic collaborations. Resolution is built on foundational science from the University of Edinburgh and is led by CEO Dr. Amir Hefni alongside a world-class team of experienced cell therapy leaders.

Clinical-stage biopharmaceutical company developing a potentially best-in-class, next-generation psychedelic therapy for the treatment of underserved mental health disorders. Lead candidate, RE104, the only prodrug of 4-OH-DiPT in development, is designed to deliver fast-acting therapeutic benefits with a short duration of psychoactive effects. RE104 is currently being evaluated in the ongoing RECONNECT Phase 2 clinical trial, a multicenter, randomized, double-blind, active dose-controlled study in moderate-to-severe postpartum depression (PPD) patients; topline results from study are expected in mid-2025. Reunion also plans to evaluate RE104 in Adjustment Disorder in Cancer and Other Illnesses (ADCO), and expects to initiate a Phase 2 clinical trial in 2025. In May 2024, closed a $103 million Series A financing co-led by MPM BioImpact and Novo Holdings, extending cash runway through 2026, beyond data readouts for both the RECONNECT Phase 2 trial and the planned Phase 2 trial in ADCO.

Biopharmaceutical company committed to our mission of pioneering solutions to deliver life-changing brain health medicines, so every person can thrive. Sage developed the only two FDA-approved treatments indicated for postpartum depression and is advancing a robust pipeline to target unmet needs in brain health.

Clinical-stage biotechnology company developing AAV-based gene therapies to treat debilitating chronic conditions. Spur’s lead asset, FLT201, is an AAV gene therapy candidate that leverages Spur’s proprietary and potent AAVS3 capsid to deliver GCase85, a rationally engineered longer-acting version of the GCase enzyme missing in Gaucher patients, to stop disease progression and reduce symptoms in Gaucher disease. FLT201 is currently being investigated in a Phase 1/2 trial in Gaucher disease Type 1, with data to date demonstrating favorable safety and tolerability as well as early signals of clinical improvements. The Company expects to initiate a Phase 3 registrational trial in 2025. The Company is also advancing a Phase 1/2 clinical trial for asset SBT101 in adrenomyeloneuropathy (AMN) and preclinical programs in GBA1-linked Parkinson’s disease and severe subset of chronic heart failure.

Late-stage biopharma company developing novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction. Stealth Biotherapeutic’s lead product candidate, elamipretide, is under review for Barth syndrome and is in late-stage development for primary mitochondrial myopathy and for dry age-related macular degeneration (AMD). The company is also evaluating a topical ophthalmic formulation of our second-generation clinical-stage candidate, bevemipretide (SBT-272), for dry age-related macular degeneration, and has a deep pipeline of novel compounds under evaluation for rare neurological and myopathic diseases.

Clinical-stage company committed to developing new standards of care for the frontline treatment of patients with hematologic malignancies. Driven by the motivation to help patients with blood disorders that have largely eluded other targeted approaches, Syros is developing tamibarotene, an oral selective RARα agonist in frontline patients with higher-risk myelodysplastic syndrome (HR-MDS) with RARA gene overexpression. Ongoing Phase 3 global clinical trial, SELECT-MDS-1, with pivotal data expected by mid-4Q24.

Commercial stage biopharmaceutical company focused on developing therapeutic candidates for highly prevalent diseases with limited treatment options, beginning with eyecare indications. XDEMVY® (lotilaner ophthalmic solution) 0.25% , the first and only U.S. FDA approved prescription treatment for Demodex blepharitis, has continually shown strong quarter-over-quarter sales and revenue growth since launch in August 2023. Well positioned to continue delivering net product sales growth with a projected peak sales potential of >$1B and gross-to-net discounts with robust payer coverage including commercial and Medicare payers, expanded sales force and a planned new direct-to-consumer TV campaign. Advancing clinical development of other pipeline programs and remain on-track to engage with the FDA by year end of 2024 on TP-03, a topical ophthalmic formulation of lotilaner for Meibomian Gland Disease (MGD); TP-04, a topical gel formulation of lotilaner for the treatment of papulopustular rosacea; and TP-05, a lotilaner oral tablet for the prevention of Lyme disease.

Clinical-stage biopharmaceutical company developing a portfolio of orally delivered small-molecule product candidates to address serious diseases, including oncology and obesity. Terns’ pipeline contains three clinical stage development programs including an allosteric BCR-ABL inhibitor, a small-molecule GLP-1 receptor agonist, a THR-β agonist, and a preclinical GIPR modulator discovery effort, prioritizing a GIPR antagonist nomination candidate.

Clinical-stage biopharmaceutical company focused on the discovery and development of transformative medicines for the treatment of serious diseases in areas of high unmet medical need. The Company’s pipeline consists of a family of small molecule compounds called SMTPs (Stachybotrys Microspora Triprenyl Phenols) derived from a fungus. TMS’ lead program, TMS-007 (JX10), has demonstrated efficacy and safety in its Phase 2a study for the treatment of acute ischemic stroke. The Company’s robust pipeline also includes programs in resistant or uncontrolled hypertension, acute kidney injury, and spinal cord injury. TMS continues to explore new pipeline products by leveraging its established partnerships with leading academic institutions in Japan, with the intention to build a bridge between academic discoveries and the global pharmaceutical market.

Clinical-stage cell therapy company leveraging its global presence and core non-viral platform to democratize CAR-T therapy for patients worldwide. Headquartered in San Mateo, California with R&D, manufacturing and clinical operations hubs across Europe and Asia, TriArm is executing on its mission to enable better, safer and more accessible CAR-T products through a four-pronged approach centered around the company’s (1) Fast-in-Time™ (FIT™) non-viral platform capable of CAR-T manufacture in two days, (2) straightforwardly differentiated programs aimed at clinically- and/or commercially-validated targets, (3) rapid and efficient clinical translation and de-risking process via investigator-initiated trials (IITs), and (4) focus on delivering real benefit to CAR-T eligible patients currently marginalized by manufacturability issues and prohibitive cost tied to conventional CAR-T programs.

Venture-backed Phase 2 clinical development-stage company focused on developing breakthrough, disease-modifying medicines to treat kidney diseases. Walden is applying novel, multi-disciplinary approaches that directly target the kidney to prevent damage, slow disease progression, and restore kidney function. Walden’s programs address novel targets for therapeutic intervention, directly targeting two cell types critical for kidney function: podocytes and proximal tubular cells. Dysfunction of these cells are critical hallmarks of the majority of kidney diseases. Walden’s clinical-stage program is a humanized monoclonal antibody that inhibits suPAR, a pro-inflammatory mediator that causes podocyte dysfunction and kidney disease. Walden’s second most advanced program is a small molecule that is designed to restore the function of dynamin, an enzyme responsible for the maintenance of the cytoskeletal architecture and function of podocytes and proximal tubule cells. All of Walden’s programs offer the promise to deliver disease-modifying, breakthrough therapies that are readily combinable with the standard of care to transform the treatment of kidney disease.

Clinical-stage biopharmaceutical company engineering conditionally activated cytokines for cancer and other serious diseases. Clinical INDUKINE™ molecules have demonstrated platform proof of concept. WTX-124 (IL-2) shows best-in-class potential in ongoing Ph. 1/1b study: well-tolerated as monotherapy in outpatient setting with clinical activity, with multiple expansion arms now open; strong combination profile observed in dose escalation. WTX-330 (IL-12) induced clinically relevant monotherapy activity at exposures historically toxic; opened expansion arms in advanced or metastatic solid tumors. Updated data from WTX-124 and WTX-330 expected in 4Q 2024. Additional research-stage programs extend platform to a broader range of targets (IL-21, IL-18, IL-10) and indications (inflammation) for future opportunities; cash runway into at least 1Q26.

Clinical-stage biopharmaceutical company advancing a broad a pipeline for cancer and autoimmune diseases, leveraging its XmAb® protein engineering technologies. Wholly owned programs targeting solid tumors include bispecific T cell engagers: ‘819 (ENPP3 x CD3) in ccRCC, ‘808 (B7-H3 x CD28) and ‘541 (CLDN6 x CD3) in ovarian/CLDN6+. Recently added autoimmune pipeline and 4 programs: plamotamab (CD20 x CD3) for RA, XmAb657 (CD19 x CD3), and two targeting TL1A for IBD+. Multiple near-term catalysts, with initiations and clinical readouts expected across the portfolio in 2025. Legacy of partnerships generated >20 XmAb-based candidates in clinical development and 3 marketed products; select partners include Janssen, Amgen, Genentech and more. Company is well-capitalized with runway guided through 2027.

Clinical-stage genetic medicines company focused on redefining the course of neurodegenerative diseases. Lead candidate, PBFT02, is an AAV1 gene therapy that aims to elevate progranulin levels to restore lysosomal function and slow disease progression. PBFT02 is initially targeting genetic forms of frontotemporal dementia (FTD-GRN and FTD-C9orf72) in the ongoing upliFT-D global Phase 1/2 clinical study. Interim data from FTD-GRN patients suggests PBFT02 could be a best-in-class, one-time progranulin-raising therapy. Preclinical models support the potential of PBFT02 to correct TDP-43 pathology, a hallmark of multiple neurodegenerative diseases, including ALS and Alzheimer’s Disease. The company is also advancing a preclinical program for Huntington’s disease in partnership with GEMMA Biotherapeutics, led by Dr. Jim Wilson. Strong cash position, with runway to end of 2Q26.

Emerging biotechnology company pioneering the next generation of RNAi therapies with a unique central nervous system (CNS) pipeline built on a proprietary, three stranded RNA platform named CASi (Conditionally Activated siRNA).  CASi molecules have excellent biodistribution, potency and durability, can be cell-specific and have broad applicability across a range of diseases. Multiple programs are advancing towards the clinic, with one development candidate recently declared, targeting a large market, neurodegenerative indication, with IND filing targeted for next year. A second development candidate will be declared in quick succession in the next quarter. Switch was established on technologies from Caltech, Harvard and City of Hope, launching in March 2023 with $52M, supported by corporate and institutional VCs.

Focused on the development of emavusertib, a potentially first-to-market, orally available small molecule IRAK4 inhibitor. Demonstrated safety and single-agent activity as well as synergy with BTKi, HMA and BCL2i. Broad opportunity in NHL, AML and solid tumors, where it is being evaluated in three company sponsored Phase 1/2 clinical studies in NHL and AML/MDS as well several investigator-sponsored studies in solid tumors. Near term milestones include data in patients with R/R PCNSL, patients with R/R FLT3m AML and initial safety data for patients with triplet (azacitidine/venetoclax/emavusertib) combination in frontline AML.

Clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for patients with cancer and autoimmune diseases, has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Company’s immuno-oncology and autoimmune pipeline includes iPSC-derived natural killer (NK) cell and T-cell product candidates, which are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple therapeutic mechanisms to patients.

Clinical-stage biopharmaceutical company pioneering the discovery and development of a new generation of immuno-oncology therapeutics. Phase 3 trial underway for belrestotug, a highly potent, FcγR-engaging anti-TIGIT antibody, in collaboration with GSK for NSCLC, with additional late-stage trials ongoing including a Phase 2 trial in HNSCC. Wholly owned program inupadenant, an adenosine A2A receptor antagonist, is currently in a randomized Phase 2 trial in NSCLC patients in combination with chemotherapy. EOS-984, a first-in-class small molecule targeting a novel mechanism in the adenosine pathway, is currently treating patients with advanced solid tumors in a Phase 1 trial. Strong cash balance of ~$714M as of June 30, 2024, expected to provide runway into 2027.

Clinical-stage allogeneic cell therapy and genetic medicines company advancing differentiated non-viral treatments for patients with cancer and rare diseases. Validating big pharma relationships, including cell therapy-focused strategic collaborations with Roche and Astellas. Three clinical stage allogeneic CAR-T programs include Roche-partnered P-BCMA-ALLO1 for multiple myeloma and a dual CAR program, P-CD19CD20-ALLO1, for B-cell malignancies and wholly owned P-MUC1C-ALLO1 in solid tumors. Interim clinical data for P-BCMA-ALLO1 presented at IMS in September 2024 demonstrated high response rates, was well tolerated and available on demand, supporting recently initiated Phase 1b trial and the Company's belief that T stem cell (TSCM)-rich allogeneic CAR-Ts have the potential to address unmet needs in multiple myeloma safely, effectively and reliably. P-BCMA-ALLO1 has received ODD for MM and RMAT designation for adult patients with RRMM from FDA. Also advancing multiple preclinical non-viral genetic medicines programs, including P-FVIII-101 for Hemophilia A and P-KLKB1-101 for hereditary angioedema (HAE). Data updates across pipeline anticipated in Q4 2024.

Leading biotechnology company deploying proprietary Prime Editing platform to develop a new class of differentiated one-time curative genetic therapies for patients. Currently progressing a diversified portfolio of high-value investigational therapeutic programs organized around core areas of focus: hematology, immunology and oncology, liver and lung. Company recently announced a strategic license and broad collaboration agreement with Bristol Myers Squibb to develop ex vivo T cell products, and is on track to announce first-in-human clinical data from its Phase 1/2 clinical trial of PM359 in CGD in 2025 and to continue to advance it’s Wilson’s disease program towards IND.

Clinical-stage company developing transformational treatments for patients with serious metabolic diseases marked by high unmet medical need, including MASH. Akero's lead product candidate, EFX, is currently being evaluated in the ongoing SYMMETRY study, a 96-week Phase 2b clinical trial in patients with compensated cirrhosis due to MASH (F4 fibrosis), as well as three ongoing Phase 3 clinical trials in patients with pre-cirrhotic MASH or compensated cirrhosis due to MASH: SYNCHRONY Histology, SYNCHRONY Real-World, and SYNCHRONY Outcomes. The SYNCHRONY program builds on the results of two Phase 2b clinical trials, the HARMONY study in patients with pre-cirrhotic MASH (F2-F3) and the SYMMETRY study in patients with compensated cirrhosis due to MASH (F4) in which patients have been treated for up to 96 weeks.

Clinical-stage genetic medicines company focused on redefining the course of neurodegenerative diseases. Lead candidate, PBFT02, is an AAV1 gene therapy that aims to elevate progranulin levels to restore lysosomal function and slow disease progression. PBFT02 is initially targeting genetic forms of frontotemporal dementia (FTD-GRN and FTD-C9orf72) in the ongoing upliFT-D global Phase 1/2 clinical study. Interim data from FTD-GRN patients suggests PBFT02 could be a best-in-class, one-time progranulin-raising therapy. Preclinical models support the potential of PBFT02 to correct TDP-43 pathology, a hallmark of multiple neurodegenerative diseases, including ALS and Alzheimer’s Disease. The company is also advancing a preclinical program for Huntington’s disease in partnership with GEMMA Biotherapeutics, led by Dr. Jim Wilson. Strong cash position with runway to end of 2Q26.

Clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Leveraging its integrated and interrelated Gene Therapy, Cellular Regeneration and Precision Medicine platforms and proprietary core capabilities, the company is advancing a pipeline of novel therapies with diverse treatment modalities for rare genetic cardiovascular disorders and more prevalent heart conditions. Tenaya’s most advanced candidates include TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor being initially developed for heart failure with preserved ejection fraction (HFpEF). Tenaya also has multiple early-stage programs progressing through preclinical development.

Late-stage biotech focused on developing best-in-class therapies for neurological and psychiatric disorders. The Company’s late-stage program, valiloxybate, is positioned to become the only no-sodium, once-nightly oxybate therapy for the treatment of narcolepsy and idiopathic hypersomnia, addressing key unmet needs in a $2B market despite limitations of current therapies. XWPharma has aligned with the FDA in an End-of-Phase 2 on the protocol for a single pivotal Phase 3 trial for narcolepsy and is preparing to initiate the study. The Company’s second clinical program, XW10508, is an oral NMDA antagonist for treatment-resistant depression with a differentiated adverse event profile compared to the approved esketamine intranasal therapy, which is anticipated to be a global blockbuster despite its restrictive REMS requiring clinic-based administration. XW10508 has demonstrated safety and tolerability in Phase 1, and an exploratory Phase 2 trial is underway to assess efficacy, safety, and dosage ahead of a subsequent double-blind Phase 2 in the U.S.

Late-stage clinical biotechnology company focused on the discovery and development of drugs for the treatment of adult and pediatric cancer. The company is utilizing its novel proprietary Phospholipid Drug Conjugate™ (PDC) delivery platform to develop the next-generation of cancer cell-targeting treatments, delivering enhanced efficacy with fewer off-target effects resulting in improved safety. The company’s lead asset, iopofosine I 131, is a small-molecule PDC radiotherapeutic designed to provide targeted delivery of the radioisotope iodine-131. Iopofosine I 131 has demonstrated clinical activity in hematologic malignancies as well as tumors located across the blood brain barrier. Recently, the pivotal study in Waldenstrom’s macroglobulinemia (WM) was successfully completed and the NDA is being compiled. The company anticipates receiving an accelerated review which, assuming marketing approval, supports a projected commercial launch in the second half of 2025. WM represents a meaningful market and the company plans to expand iopofosine I 131 into additional liquid tumors while selectively advancing its robust pipeline of radiopharmaceuticals in liquid and solid tumors.

Late-stage biotechnology company focused on developing AR882, a potentially best-in-class, highly potent and selective next generation URAT1 inhibitor to reduce serum urate levels, flares and tophi in patients with gout. Gout remains a large and growing market with ~13M patients in the U.S. alone, ~2M of which have tophaceous gout. Phase 3 REDUCE 2 study is ongoing with a second pivotal study expected to initiate in 1Q25. In Phase 2 studies AR882 has demonstrated encouraging efficacy and safety compared to SOC and a Phase 2b study in tophaceous gout showed impressive results, with 29% of patients receiving AR882 monotherapy having a complete resolution of target tophi. Recently granted Fast Track Designation and expect to complete the Phase 3 program in late 2026.

Immuno-oncology biotech developing supercharged multi-functional antibodies that boost the cancer patient’s own immune cells solely in the tumor. Treatment with Avidicure’s supercharged antibodies results in unprecedented tumor burden control in animal models and potent activation of both the innate and adaptive arms of the immune system in freshly isolated human tumor tissues. Avidicure has achieved preclinical proof of concept with a portfolio of products and is now moving its lead product into development for treatment of non-small-cell lung cancer, head & neck cancer, endometrial cancer, bladder cancer and triple-negative breast cancer. Clinical trials will start in 2026. The company is founded and managed by four former members of the executive leadership teams of Kiadis, Ablynx and Crucell, is directed by a seasoned supervisory board and advisory board, and is backed with a €37 million seed round from a top-tier investor syndicate led by EQT Life Sciences.

CVAC is a multinational biopharmaceutical company pioneering transformative mRNA-based medicines to address areas of high unmet medical need, with a primary focus on oncology and infectious diseases. Oncology pipeline features CVGBM, an off-the-shelf shared-antigen cancer vaccine candidate targeting glioblastoma, with promising Phase 1 data presented at ESMO/SITC 2024 and ongoing dose-expansion enrollment. By late 2025, CureVac expects to bring a second shared-antigen candidate in squamous NSCLC into the clinic. In infectious diseases, CureVac recently initiated a program for urinary tract infections, one of the most prevalent bacterial infections globally, based on promising preclinical results. Its licensed prophylactic vaccine portfolio with GSK includes a seasonal flu mRNA vaccine demonstrating positive Phase 2 data in Q3 2024, set to enter Phase 3 in 2025, a Phase 2 avian influenza (H5N1) study, granted FDA Fast Track Designation with data expected in H1 2025, and a newly initiated flu/COVID-19 combo Phase 1/2 study. CureVac’s innovative mRNA platform, broad R&D capabilities, strategic partnerships, and financial stability, supported by a recent €400 million payment from GSK, underline its potential to deliver next-generation therapies and vaccines​.

Clinical-stage radiopharmaceutical biotechnology company developing an innovative technology that leverages alpha-emitting microparticle suspension designed for local treatment of cancer in body cavities. The lead product candidate, Radspherin® is a first-in-class, directly targeting alpha-emitter with mode of action that complements cytoreductive surgery for peritoneal carcinomatosis by eradicating micro-metastases post-operatively, without harming healthy tissue. Radspherin® is currently being evaluated in an ongoing Phase 2 trial in patients with peritoneal metastases from ovarian cancer with multiple readouts expected throughout 2026-27. The trial builds on promising interim results from Phase 1/2a studies demonstrating very low post-treatment recurrence rates in colorectal and ovarian cancer patients as well as a unusually benign safety profile. The Company is led by an experienced management team with previous experience in bringing radiopharmaceuticals to the market, as well as the inventors behind the only FDA-approved alpha radiation therapy.

Clinical-stage biotechnology company changing the standard-of-care for cancer patients by reimagining surgical oncology from a physical intervention only to a therapeutic one as well. By concentrating derisked assets at the site and time of surgical tumor resection, their proprietary SURGERx™ platform seeks to improve the efficacy and safety of immunotherapy, thereby preventing post-surgical cancer recurrence and metastasis. Lead program STM-416 is currently being evaluated in a Phase 1/2a trial in patients with recurrent bladder cancer, with initiation of Phase 2a expected in mid-2025. Learn more about their other IND-enabled programs and this enormous market opportunity.

Radiopharmaceutical company providing rare medical radioisotopes, patient dose manufacturing and development services to the Radiopharmaceutical market. Radiopharmaceuticals deliver precision diagnosis and therapy to treat serious disease such as cancer.  NorthStar is emerging as the largest commercial scale manufacturer of Copper-67 and non-carrier added Actinium-225.  We will provide patient dose manufacturing services from our 53,000 square foot CDMO facility.  In addition, NorthStar is providing value adding research and development services for our customers.  NorthStar performs this work in state of the art, built for purpose facilities.  All of our products and services are offered in a unique, one-campus concept.  NorthStar has established a leading book of business through long-term contracts with our biotech and big pharma customers.  NorthStar has built an outstanding team of 250 + individuals who daily contribute to our mission of providing patients global access to game changing radiopharmaceuticals.

Biotechnology company with operations in Europe and the U.S. that is dedicated to transforming patient outcomes through life-changing science and innovation. Focusing on high unmet medical needs, Galapagos synergizes compelling science, technology, and collaborative approaches to create a deep pipeline of best-in-class small molecules and cell therapies in oncology and immunology. With capabilities from lab to patient, including a decentralized cell therapy manufacturing platform, and the financial strength (>€3B) to invest strategically for the near- and long-term, Galapagos is committed to challenging the status quo and delivering results for patients, employees, and shareholders.

TCR-based drug development company with two near term clinical stage assets in acute myeloid leukemia (AML).  BlueSphere is leveraging its proprietary TCR discovery platform, TCXpress™, to build both its own pipeline and strategic partnership opportunities. The initial clinical candidate, BSB-1001 (from the TCX-101 program), targets the minor histocompatibility antigen-1 (HA-1), and will be dosed with allogeneic hematopoietic stem cell transplant.  The company anticipates enrolling the first patient in 1Q25.  A second clinical candidate, BSB-2001 (from the TCX-102 program) targets AML patients with mutant NPM1, will be autologous and not given in combination with stem cell transplant.  An IND is expected in the second quarter of 2025.