Our Pipeline

Our programs span discovery-stage to late-stage development and cover a range of high-value indications. We aim to pursue programs we believe could be first-in-class / best-in-class and where there is prior learning in human genetics or precedented human activity for a pathway of interest. We place a premium on learnings from the clinic, whereby a drug has established the relevance of a biological pathway contributing to disease outcome. Our approach is to pursue the best assets in a capital efficient manner and rapidly progress our programs through development, evaluating the unique biological advantage of our product candidates.

We categorize our current programs as registrational, emerging, or exploratory. Our R&D spend is commensurate with these three stages, with the highest spend on the programs that have already established clinical proof of concept. For programs in the earlier stages, we aim to implement capital-efficient plans to reach the next set of catalysts, gating more significant spending until after we obtain clinical proof of concept.

View our Expanded Access Policy for investigational medical products.

Preclinical

Phase 1

Phase 2

Phase 3

Lixivaptan

Overview

We are developing lixivaptan, an oral, non-peptide, new chemical agent that is designed to selectively suppress the activity of the hormone vasopressin at the V2 receptor with potential to avoid safety issues associated with the only drug approved for the treatment of ADPKD. Lixivaptan is currently in a Phase 3 registrational trial for the treatment of ADPKD.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
Epidemiology~140,000 patients diagnosed in the United States	Disease Autosomal 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

Preclinical

Phase 1

Phase 2

Phase 3

SerpinPC

Overview

We are developing SerpinPC, a specific inhibitor of activated protein C (APC), for the treatment of hemophilia A and hemophilia B. SerpinPC has been observed to be well-tolerated in the clinical setting, associated with promising reductions in bleeding rates, and has PK suitable for infrequent subcutaneous dosing without the need for factor replacement. SerpinPC has human genetic target validation and established proof of concept Phase 2a clinical data.

Reason to Believe in Target

Human Genetics Support

Epidemiology	Disease
Epidemiology~20,000 persons with hemophilia in the United States 450,000 estimated global prevalence	Disease Hemophilia A, B

Leadership

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

Preclinical

Phase 1

Phase 2

Phase 3

LB101 and LB201

Overview

We aim to develop novel therapeutics based on our unique platform technology, which is designed to selectively drive potent effector function activity, such as CD47 or CD3, while avoiding systemic toxicity. The lead compound is LB101, a PD-L1xCD47 LockBody®, which has two anti-CD-47 domains blocked by two anti-PD-L1 domains, with proprietary human IgG-derived hinges linking the anti-CD47 and anti-PD-L1 domains. CD47 cell-killing is blocked by the PD-L1 tumor targeting domain until the IgG-derived hinges are naturally degraded in the tumor microenvironment (TME), thus unlocking and activating the CD47 effector function activity in the tumor. We are also developing LB201, a PD-L1xCD3 LockBody®, which is designed to selectively drive potent CD3 effector function activity while avoiding systemic toxicity.

Reason to Believe in Target

Precedented Human Activity

Epidemiology	Disease
EpidemiologyUp to ~19 million new cases and ~10 million deaths globally, including ~1.9 million and ~600,000 within the United States	Disease Solid Tumors

Leadership

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

Preclinical

Phase 1

Phase 2

Phase 3

ZF874

Overview

We are developing ZF874, a small molecule pharmacological chaperone folding corrector of the Z variant of alpha-1-antitrypsin (Z-A1AT) intended to address the underlying pathology of both lung and liver manifestations of AATD.

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 &nbsp 1,800 homozygous for the Z mutation	Disease Alpha-1 Antitrypsin Deficiency (AATD)

Leadership

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

Preclinical

Phase 1

Phase 2

Phase 3

MGX292

Overview

We are developing MGX292, a recombinant modified BMP9 replacement protein designed to overcome the deficiency in BMP9 signaling in PAH, directly targeting a central underlying disease mechanism (BMP9/BMPR2/ALK1 signaling pathway genetically altered in PAH).

Reason to Believe in Target

Human Genetics Support

Epidemiology	Disease
EpidemiologyPAH prevalence is 11 to 26 per million individuals, affecting approximately 70,000 patients in North America, Europe and Japan	Disease Pulmonary 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

Preclinical

Phase 1

Phase 2

Phase 3

Orexin Agonists

Overview

We are developing oral and intranasal selective orexin receptor 2 (OX2R) agonists designed to leverage unique structural insights and to directly target the underlying pathophysiology of orexin neuron loss in NT1. Potential expansion into Narcolepsy Type 2 (NT2), rare hypersomnias, and additional disorders.

Reason to Believe in Target

Precedented Clinical Activity

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

Preclinical

Phase 1

Phase 2

Phase 3

CBS004

Overview

We are developing CBS004, a humanized mAb specific to BDCA2, which is expressed exclusively on plasmacytoid dendritic cells (pDCs).

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)	Disease Autoimmune Diseases

Leadership

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

Preclinical

Phase 1

Phase 2

Phase 3

Our Pipeline

Lixivaptan

Overview

Reason to Believe in Target

Leadership

SerpinPC

Overview

Reason to Believe in Target

Leadership

LB101 and LB201

Overview

Reason to Believe in Target

Leadership

ZF874

Overview

Reason to Believe in Target

Leadership

MGX292

Overview

Reason to Believe in Target

Leadership

Orexin Agonists

Overview

Reason to Believe in Target

Leadership

CBS001

Overview

Reason to Believe in Target

Leadership

CBS004

Overview

Reason to Believe in Target

Leadership

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