2026 J.P. Morgan Healthcare Conference

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Aardvark Therapeutics

Targeting the biological drive for hunger to treat obesity & metabolic diseases

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Aardvark Therapeutics

Targeting the biological drive for hunger to treat obesity & metabolic diseases

Key Highlights

Differentiating hunger vs appetite: Hunger, the feeling that drives the physiological need to eat, is different from appetite, the desire for food. By tapping the natural defense mechanism behind bitter taste receptors, Aardvark’s lead therapeutic candidate targets the signaling pathways for hunger to restore satiety and metabolic balance for hyperphagia-driven diseases (a severe and insatiable feeling of hunger).
Solving the GLP-1 shortcoming in obesity: Aardvark aims to solve the problems of lean mass loss and GLP-1 weight rebound following withdrawal, while reducing GI side effects and potentially eliminating the need for injections.
Key Events in 2026:
- Topline data from the Phase 3 HERO trial evaluating ARD-101 for the treatment of hyperphagia associated with PWS in 3Q 2026
- Initiation of the Phase 2 POWER trial evaluating ARD-201’s potential to prevent weight regain in subjects who discontinue GLP-1RA therapy after achieving substantial prior weight loss (~15%) in 2H 2025
- Initiation of the Phase 2 STRENGTH trial evaluating placebo-adjusted weight loss and the additive effects of ARD-201 combined with GLP-1RA therapy in 1H 2026

Interview

Tien Lee, M.D., Chief Executive Officer

NASDAQ

AARD

Website

aardvarktherapeutics.com

Disease Area

Metabolic Disorders
Obesity
Rare Disease

Technology

Small Molecule

BBOT

Taking Precision Oncology to New Heights

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BBOT

Taking Precision Oncology to New Heights

Key Highlights

Bridging to a Better Future in Oncology: BBOT is advancing a pipeline of potentially first- and best-in-class therapies targeting the RAS signaling pathway, including ON/OFF KRAS inhibitors and a novel PI3Kα inhibitor for two of the most frequently mutated pathways in cancer, RAS and PI3Kα. The company is redefining RAS inhibition by targeting both the ON and OFF states of RAS to enable deeper inhibition of cancer driving signals, reduce resistance, and improve safety. This next-gen approach has the potential to deliver deeper, safer and more durable responses for patients with RAS driven cancers.
Synergy by Design: BBOT’s pipeline is designed for rational combinations – each asset targeting complimentary nodes in the RAS signaling cascade. Preclinical data (here and here) show synergy between these agents, supporting a plug-and-play strategy that could unlock deeper, more durable responses and delay resistance.
2026 Milestones: BBOT is well-positioned to execute on near-term data milestones for all three of its programs, which have the potential to address the needs of approximately 250,000 U.S. patients annually across breast, lung, colorectal, and pancreatic cancers, including:
- BBO-8520 (KRAS^G12C ON / OFF) Phase 1 monotherapy and combination with pembrolizumab data readout in Q1 2026
- BBO-10203 (RAS:PI3Kα Breaker) Phase 1 data readout in 1H 2026
- BBO-11818 (panKRAS ON / OFF) Phase 1 data readout and broader expansion datasets across BBO-8520 and BBO-10203 in 2H 2026 onward

Interview

Eli Wallace, Ph.D., Chief Executive Officer

NASDAQ

BBOT

Website

bbotx.com

Disease Area

Oncology

Technology

Small Molecule

Kelonia Therapeutics

in vivo CAR-T Pioneer Making Scalable & Accessible Cell Therapies

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Kelonia Therapeutics

in vivo CAR-T Pioneer Making Scalable & Accessible Cell Therapies

Key Highlights

Democratizing CAR-T Therapy: Kelonia’s best-in-class in vivo gene placement system (iGPS) enables the efficient delivery of genetic medicine with high tissue specificity and avoids costly and lengthy ex vivo manufacturing, burdensome lymphodepleting chemotherapy and reliance on large research hospitals
First-in-human data provides early validation in multiple myeloma: A late-breaking presentation at ASH 2025 shows clinical candidate, KLN-1010, achieved rapid responses in all four patients with a single infusion. Armed with preliminary clinical data and partnerships with multiple pharma companies, Kelonia is positioned well to capitalize on the full potential of its iGPS platform.
Key Events in 2026: Additional clinical data from Phase 1 trial of KLN-1010, its in vivo CAR-T for multiple myeloma

Interview

Kevin Friedman, Ph.D., Chief Executive Officer

Website

keloniatx.com

Disease Area

Oncology

Technology

Cell Therapy
Gene Therapy

Kura Oncology

FDA Approval Strengthens Kura’s Leadership and Drives Momentum to Frontline AML

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Kura Oncology

FDA Approval Strengthens Kura’s Leadership and Drives Momentum to Frontline AML

Key Highlights

KOMZIFTI^TM (ziftomenib) receives FDA approval: The first targeted, once-daily oral therapy for adult patients with relapsed/refractory NPM1-mutant AML. Approval based on strong efficacy and safety profile, notably with no Boxed Warning for QTc prolongation or Torsades de Pointes – a class-differentiating benefit risk profile.
Expansion to frontline AML: Kura aims to advance KOMZIFTI to the broader AML population, potentially supporting up to 50% of AML patients across multiple genetic subtypes. Kura will share P1 data from the KOMET-007 trial at the 2025 ASH Annual Meeting in December and recently initiated its pivotal Phase 3 KOMET-017 trial, evaluating KOMZIFTI for frontline AML.
Advancing next-generation FTIs: At ESMO 2025, Kura presented encouraging early data for darlifarnib, its next-generation FTI designed to overcome resistance mechanisms to targeted therapies. Darlifarnib’s potential combinations with TKIs, PI3Kα inhibitors and KRAS inhibitors could benefit up to ~200,000 patients annually across multiple solid tumor types.
Key upcoming milestones:
- Frontline AML data on menin inhibitor combinations at ASH 2025
- Dose-escalation data of darlifarnib + cabozantinib in advanced RCC (2026)
- Preliminary data of darlifarnib + adagrasib in KRASG12C-mutated solid tumors (2026)

Interview

Troy Wilson, Ph.D., J.D., Chief Executive Officer and Co-Founder

NASDAQ

KURA

Website

kuraoncology.com

Disease Area

Acute leukemias
Head and neck squamous cell carcinoma
Gastrointestinal stromal tumors
Diabetes

Technology

Small molecules - menin inhibitors and farnesyl transferase inhibitors (FTIs)

MBX Biosciences

Clinical Validation of Precision Peptides for Endocrine and Metabolic Care

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MBX Biosciences

Clinical Validation of Precision Peptides for Endocrine and Metabolic Care

Key Highlights

Competitive Phase 2 data in hypoparathyroidism (HP): Once-weekly canvuparatide hit the primary endpoint in the Phase 2 Avail™ study (63% responders at 12 weeks vs. 31% placebo) with 79% responders at 6 months in the open-label extension, supporting best-in-class potential and Phase 3 planning for 2026. End-of-Phase-2 FDA and EMA meetings are slated for Q1 2026.
Platform for precision: Built on peptide pioneer Richard DiMarchi, Ph.D.’s science, MBX’s Precision Endocrine Peptide™ (PEP™) platform is engineered to extend peptide half-life and deliver sustained, consistent exposure—aiming to reduce dosing burden and variability while improving outcomes—now clinically validated by canvuparatide’s Phase 2 success.
Recent $200M upsized offering extends cash runway into 2029 to fund upcoming milestones across obesity and endocrine pipeline:
- HP: present once-weekly canvuparatide Phase 2 data and 1-year OLE in Q2 2026; initiate Phase 3 in Q3 2026.
- PBH: imapextide (MBX-1416) has begun a Phase 2a STEADI™ trial with results expected in Q2 2026.
- Obesity: MBX-4291 has dosed its first participant in Phase 1; 12-week MAD readout expected in Q4 2026.

Interview

Kent Hawryluk, M.S., MBA, President and Chief Executive Officer

NASDAQ

MBX

Website

mbxbio.com

Disease Area

Metabolic and Endocrine Disorders
Hypoparathyroidism (HP)
Post-bariatric hypoglycemia (PBH)
Obesity

Technology

Peptide

MindImmune

Neuroinflammation: The Next Frontier in Alzheimer’s and Other Neurodegenerative Diseases

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MindImmune

Neuroinflammation: The Next Frontier in Alzheimer’s and Other Neurodegenerative Diseases

Key Highlights

Rewriting Alzheimer’s: Targeting Immune Drivers of Neurodegeneration: MindImmune is shifting the paradigm in Alzheimer’s research by focusing on immune system dysfunction rather than traditional amyloid targets. The team identified a peripheral immune cell that infiltrates the brain and drives synaptic damage. To counter this, they’re advancing MITI-101, a monoclonal antibody designed to block the recruitment of these cells and potentially halt the neuroinflammatory cascade behind Alzheimer’s progression.
From Pharma to Frontier: After a distinguished career in big pharma, including leadership roles at Pfizer and Lundbeck, MindImmune Chief Science Officer, Dr. Stevin Zorn, made a pivotal shift. Frustrated by the limited impact of traditional approaches targeting brain circuitry, he co-founded MindImmune to pursue a bold new path targeting immune-driven mechanisms of neurodegeneration. Dr. Zorn and Chief Executive Officer, Isaac Stoner, are now leading a mission that could redefine how we treat Alzheimer’s disease.
2026 Milestones: Following the close of $10.2 million Series A extension in November 2025, bringing the total Series A capital raised to $30 million, MindImmune anticipates utilizing proceeds from the financing to initiate the Phase 1 clinical study of MITI-101 in 2026.

Interview

Isaac Stoner, Chief Executive Officer

Website

www.mindimmune.com

Disease Area

Neurodegenerative Diseases

Technology

Novel Antibody Therapeutic

Spruce Biosciences

From the brink to breakthrough: Delivering the first therapy for a critically underserved patient community

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Spruce Biosciences

From the brink to breakthrough: Delivering the first therapy for a critically underserved patient community

Key Highlights

Back from the Brink with a Near-Commercial Asset: After disappointing data from its lead asset tildacerfont in congenital adrenal hyperplasia in 2024, Spruce was on the precipice. Delisted by NASDAQ and a cash runway through the end of 2025, the company had an ace up its sleeve with the acquisition of TA-ERT, a near-commercial therapeutic for the treatment of Sanfilippo syndrome type B, a rare and ultimately fatal neurodegenerative disease. Following its receipt of Breakthrough Therapy Designation for TA-ERT, the company raised $50 million, is now back on NASDAQ and looking forward to a potential FDA approval in 2026.
Positioned to Bring the First Therapy to San Filippo Syndrome Type B Patients: With no FDA-approved therapies and limited options for even palliative care, the San Filippo Syndrome Type B community had been left by the wayside. A promising enzyme replacement therapy was developed by BioMarin but spun out to a company that later went bankrupt. Now Spruce Biosciences has picked up the baton with a plan to bring this potentially life-changing therapy to the patient community as early as 2026.
2026 Milestones: BLA Submission of TA-ERT for Sanfilippo Syndrome Type B anticipated 1Q 2026 and a PDUFA by September 30, 2026.

Interview

Samir Gharib, MBA, President & Chief Financial OfficerJavier Szwarcberg, M.D., MPH, Chief Executive Officer

NASDAQ

SPRB

Website

sprucebiosciences.com

Disease Area

Neurological disorders: Sanfilippo syndrome type B (mucopolysaccharidosis Type IIB)
Major depressive disorder (MDD)
Rare Disease

Technology

Enzyme replacement therapy
CRF-1 Receptor Antagonist

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Aardvark Therapeutics

BBOT

Kelonia Therapeutics

Kura Oncology

MBX Biosciences

MindImmune

Spruce Biosciences

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