Our Pipeline

We place a premium on pursuing programs with strong biological validation, mechanistic diversification, and teams with proprietary capabilities and insights. This framework has resulted in a portfolio consisting of preclinical to phase 3 uncorrelated programs spanning diseases with high unmet need across oncology, hematology, immunology, inflammation, neuroscience, and rare diseases.

Collectively, our portfolio provides enhanced diversification with binary risk removed and asymmetric upside. The portfolio is positioned to withstand the inherent low probability of success associated with drug development of any single asset.

View our Expanded Access Policy for investigational medical products.

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Palladio Biosciences is developing lixivaptan, a selective, oral, small molecule vasopressin V2 receptor antagonist for autosomal dominant polycystic kidney disease (ADPKD) with potential for a differentiated profile over the currently available treatment, tolvaptan.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
Epidemiology~140,000 patients diagnosed in the United States	DiseaseAutosomal dominant polycystic kidney disease (ADPKD)

Leadership

Alex Martin, MBA – Chief Executive Officer
Lorenzo Pellegrini, PhD – Founder & Chief Operating Officer
Neil H. Shusterman, MD – Chief Medical Officer

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

ApcinteX is developing SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A (HA) and hemophilia B (HB), representing a potential “one-size-fits-all” treatment.

Reason to Believe in Target

Human Genetics Support

Epidemiology	Disease
Epidemiology~20,000 persons with hemophilia in the United States 500,000 estimated global prevalence	DiseaseHemophilia A, B

Leadership

Trevor Baglin, MedScD, PhD – Co-Founder & Chief Medical Officer
James Huntington, PhD – Co-Founder & Chief Scientific Advisor

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Pega-One is developing imgatuzumab, a next-generation EGFR targeting antibody, with enhanced antibody derived cell cytotoxicity (ADCC) and antibody derived cell phagocytosis (ADCP) properties, initially for the treatment of advanced cutaneous squamous cell carcinoma (CSCC), with further potential across multiple oncology indications.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
Epidemiology~10,000 new advanced stage CSCC patients diagnosed in the United States; ~5,000 in Europe	DiseaseCutaneous Squamous Cell Carcinoma (CSCC)

Leadership

Steffen Heeger, MD, PhD – Chief Medical Officer
Aurélien Marabelle, MD, PhD – Advisor
Jean-Pierre Armand, MD, PhD – Advisor

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Z Factor is developing small molecule folding correctors of the Z variant of alpha-1-antitrypsin, for the treatment of alpha-1-antitrypsin deficiency (AATD), to increase serum levels and reduce liver burden to treat or prevent associated lung and liver disease manifestations.

Reason to Believe in Target

Human Genetics Support

Epidemiology	Disease
EpidemiologyApproximately 1 in 25 individuals of European descent are A1AT Z mutation carriers, with 1 in 1,800 homozygous for the Z mutation	DiseaseAlpha-1 Antitrypsin Deficiency (AATD)

Leadership

James Huntington, PhD – Co-Founder & Chief Scientific Advisor
David Grainger, PhD – Co-Founder

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Reason to Believe in Target

Human Genetics Support

Epidemiology	Disease
EpidemiologyApproximately 1 in 25 individuals of European descent are A1AT Z mutation carriers, with 1 in 1,800 homozygous for the Z mutation	DiseaseAlpha-1 Antitrypsin Deficiency (AATD)

Leadership

James Huntington, PhD – Co-Founder & Chief Scientific Advisor
David Grainger, PhD – Co-Founder

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Morphogen-IX is developing MGX292, a disease-modifying, protein-engineered variant of human bone morphogenetic protein 9 (BMP9), targeting the central causal pathway of pulmonary arterial hypertension (PAH).

Reason to Believe in Target

Human Genetics Support

Epidemiology	Disease
EpidemiologyPAH prevalence is 25 to 50 per million individuals, affecting approximately 70,000 patients in North America, Europe and Japan	DiseasePulmonary Arterial Hypertension (PAH)

Leadership

Wei Li, PhD – Co-Founder & Advisor
Nick Morrell, MBBS, MD – Co-Founder & Chief Executive Officer
Paul Upton, PhD – Co-Founder & Advisor

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Capella Bioscience is developing CBS001, a neutralizing therapeutic monoclonal antibody to the inflammatory membrane form of LIGHT (known as TNFSF14), for the treatment of idiopathic pulmonary fibrosis.

Reason to Believe in Target

Precedented Non-Clinical Activity

Epidemiology	Disease
Epidemiology~135,000 estimated US prevalence	DiseaseIdiopathic Pulmonary Fibrosis (IPF)

Leadership

Donald Drakeman, JD, PhD – Co-Founder
Steve Holmes, PhD – Co-Founder

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Capella Bioscience is developing CBS004, a therapeutic monoclonal antibody to blood dendritic cell antigen 2 (BDCA2), for the treatment of lupus erythematosus (systemic and cutaneous) and systemic sclerosis.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
Epidemiology~200 cases per 1 million adults worldwide (SSc) ~70 cases per 100,000 persons (CLE/SLE)	DiseaseSystemic Sclerosis (SSc) Lupus Erythematosus (CLE/SLE)

Leadership

Donald Drakeman, JD, PhD – Co-Founder
Steve Holmes, PhD – Co-Founder

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

LockBody is pioneering a platform technology to develop LockBody CD47 (LB1) for optimal targeting of solid tumors by the innate immune system. LB1 is designed to bypass the CD47 sink, minimize peripheral toxicity and drive maximal CD47 intra-tumoral blocking activity.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
EpidemiologyUp to ~19 million new cases and ~10 million deaths globally, including ~1.6 million and ~500,000 within the United States	DiseaseSolid Tumors

Leadership

Jonny Finlay, PhD – Co-Founder & Chief Executive Officer
Jamie Coleman, PhD – Co-Founder & Chief Operating Officer

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

LockBody is pioneering a platform technology to develop LockBody CD3 (LB2) for safe and effective targeting of solid tumors by the innate immune system. LB2 is designed to be gradually unlocked in the tumor microenvironment to allow for potent CD3 recruitment and T cell mediated killing.

Reason to Believe in Target

Human Genetics Support

Epidemiology	Disease
EpidemiologyUp to ~19 million new cases and ~10 million deaths globally, including ~1.6 million and ~500,000 within the United States	DiseaseSolid Tumors

Leadership

Jonny Finlay, PhD – Co-Founder & Chief Executive Officer
Jamie Coleman, PhD – Co-Founder & Chief Operating Officer

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Orexia is developing oral orexin receptor agonist small molecules that target Orexin Receptor-2 (OX2R) for the treatment of Narcolepsy Type 1 and other neurological disorders.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
EpidemiologyEstimated narcolepsy prevalence of ~150,000 in the United States, of which approx. ~50% have NT1 ~3 million prevalence of narcolepsy worldwide	DiseaseNarcolepsy Type 1

Leadership

Mario Alberto Accardi, PhD, MEng – Co-Founder & Chief Executive Officer
Deborah Hartman, PhD – Chief Scientific Officer
Emiliangelo Ratti, PhD – R&D Strategic Advisor
Sarah Wurts Black, PhD – Head of Biology

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Orexia Therapeutics is developing intranasally administered Orexin Receptor-2 (OX2R) preferring peptides and intranasal OX2R small molecule agonists for the treatment of Narcolepsy Type 1 and other neurological disorders.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
EpidemiologyEstimated narcolepsy prevalence of ~150,000 in the United States, of which approx. ~50% have NT1 ~3 million prevalence of narcolepsy worldwide	DiseaseNarcolepsy Type 1

Leadership

Mario Alberto Accardi, PhD – Co-Founder & Chief Executive Officer
Deborah Hartman, PhD – Chief Scientific Officer
Emiliangelo Ratti, PhD – R&D Strategic Advisor
Sarah Wurts Black, PhD – Head of Biology

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

Janpix is developing first-in-class small molecule protein degrader therapeutics designed to covalently and selectively bind to and degrade STAT3 and STAT5 proteins for the treatment of hematological malignancies.

Reason to Believe in Target

Human Genetics Support

Epidemiology	Disease
Epidemiology~150,000 leukemia and lymphoma patients in the US ~45,000 new cases per year of AML in the US and EU	DiseaseAcute Myeloid Leukemia

Leadership

Roman Fleck, PhD – Co-Founder & Chief Executive Officer
Patrick Gunning, PhD – Co-Founder & Chief Scientific Officer

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

PearlRiver Bio is developing potent and selective oral exon20 insertion mutation inhibitors intended to have minimal activity on wild-type EGFR and optimal pharmacokinetic properties, for the treatment of EGFR exon 20 insertion (with potential to target and treat Her2 exon 20 insertions) non small cell lung cancer.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
Epidemiology4,500 incident cases of exon 20 insertion mutations in the US	DiseaseNon-Small Cell Lung Cancer (NSCLC)

Leadership

Joseph Birkett, PhD – Chief Executive Officer
Johannes Heuckmann, PhD – Co-Founder & Chief Scientific Officer
Roman Thomas, MD – Co-Founder

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

PearlRiver Bio is developing oral EGFR C797S inhibitors for the treatment of patients with EGFR non small cell lung cancer who have acquired the C797S mutation following treatment with and resistance to EGFR TKIs.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
EpidemiologyEGFR is the most frequent mutation with prevalence of ~15% of NSCLC patients	DiseaseNon-Small Cell Lung Cancer (NSCLC)

Leadership

Joseph Birkett, PhD – Chief Executive Officer
Johannes Heuckmann, PhD – Co-Founder & Chief Scientific Officer
Roman Thomas, MD – Co-Founder

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Overview

PearlRiver Bio is utilizing the ERBBinator proprietary platform technology to support design of next generation EGFR tyrosine kinase inhibitors for the treatment of non small cell lung cancer.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
EpidemiologyEGFR is the most frequent mutation with prevalence of ~15% of NSCLC patients	DiseaseNon-Small Cell Lung Cancer (NSCLC)

Leadership

Joseph Birkett, PhD – Chief Executive Officer
Johannes Heuckmann, PhD – Co-Founder & Chief Scientific Officer
Roman Thomas, MD – Co-Founder

Discovery

Lead Op / IND-Enabling

Phase 1

Phase 2

Phase 3

Our Pipeline

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Overview

Reason to Believe in Target

Leadership

Your browser is out-of-date!