Targeting Genetic Diseases at Their Source

Targeting Genetic Diseases at Their Source

We believe all people are created equal, and we reject racism and bigotry.

Who We Are

We are a collection of individuals focused on discovering and developing drugs for patients with grievous genetic diseases. We do this by:

1

Finding the right starting points to target diseases at their source.
Using a combination of a systematic assessment of the genetic disease landscape and informed diligence on the latest research, we find indications with clear mechanisms of pathogenesis that are ripe for translation into disease-modifying drugs.

2

Building products with world-class R&D personnel.
We employ over 50 scientists and work closely with leading academics to prosecute a wide variety of drug programs at the highest level of scientific rigor. We combine biopharmaceutical veterans with up-and-coming leaders to drive entrepreneurial thinking grounded by experience.

3

Employing a lean, capital efficient model.
We house each effort in an individual subsidiary company supported by shared central resources. This efficient, de-centralized corporate structure enables focus at level of each disease, but provides diversification and the ability to scale across many opportunities.

Our core values are:

Put Patients First

Think Independently

Do Everything as Fast as Possible

Be Radically Transparent

Starting with Today‘s Insights to Make Tomorrow’s Medicines

A changing scientific and clinical landscape allows our unique corporate model to efficiently create products that address significant disease burdens.

Exome and whole genome sequencing (cost and quality) Molecular biology and pathway mapping Extreme Phenotyping Big data and patient registries Deep sequencing Variant calling and bioinformatics

Find well described high unmet diseases that are ripe for intervention Develop medicines that target genetic drivers directly (at the source) Operate with world-class R&D capabilities and personnel that have developed significant, marketed products Employ a scale-able and low cost corporate structure

Disease modifying products that help patients with significant disease

Pipeline

BridgeBio Business UnitDiseaseGenetic SourceTargeted ApproachModalityStageCompany
Cardiology / NeurologyATTR-CM


ATTR-PN

TTR (transthyretin)Transthyretin stabilizerSmall moleculePhase 1Eidos Therapeutics
NeurologyPKANPANK2 (pantothenate kinase) Pantothenate kinase activatorSmall moleculeLead optimizationUndisclosed
OncologyMultiple tumor typesPTPN11 (SHP2) and MAPK pathwaySHP2 inhibitorSmall moleculeIND enabling
OncologyMultiple tumor typesRAF1 (C-RAF) and MAPK pathwayC-RAF degradationSmall moleculeLead findingUndisclosed
OncologyMultiple tumor typesKRAS and MAPK pathwayKRAS inhibitorSmall moleculeLead findingUndisclosed
Dermatology / OncologyGorlin syndrome


Frequent basal cell carcinoma

PTCH1 (patched-1, a smoothened inhibitor)Smoothened inhibitorSmall moleculePhase 2/3PellePharm
DermatologyDystrophic epidermolysis bullosa
COL7A1 (collagen 7) Collagen 7 replacement Protein replacement therapyIND enablingPhoenix Tissue Repair
DermatologyNethertonSPINK5 (LEKT1, a kallikrein 5/7/14 inhibitor) Kallikrein 5/7/14 inhibitorSmall moleculeLead optimizationUndisclosed
DermatologyDarier


Hailey-Hailey

ATP2A2, ATP2C1 S1P-lyase inhibitorSmall moleculePhase 2Undisclosed
EndocrineUndisclosedUndisclosedUndisclosedGene therapyLead findingUndisclosed

People

Laura Brege
Marta Cruz
Jonathan Fox, MD, PhD
Bruno Gagnon
Michael Henderson, MD
Charles Homcy, MD
Hoyoung Huh, MD, PhD
Jesper Jernelius, PhD
Neil Kumar, PhD
Andrew Lo, PhD
Frank McCormick, PhD
Michael Pettigrew
Len Post, PhD

Asha Rajagopal, CPA
Satish Rao, PhD

Phil Reilly, MD, JD
Hector Rodriguez, PhD
Richard Scheller, PhD*
Uma Sinha, PhD
Sabina Sood
Kahlil D’Souza
Justin To

Cameron Turtle, DPhil
Dru van Dam
Shafique Virani, MD
Robert Zamboni, PhD

* = Special Advisor

Board of Directors

Neil Kumar (BridgeBio Pharma)
Jim Momtazee (KKR)
Ali Satvat (KKR)
Rohan Nirody (Observer, Viking Global Investors)
Eric Aguilar (Observer, Aisling Capital)
Doug Dachille (Observer, AIG)

Careers

BridgeBio is committed to creating leaders as well as drugs. Our continued growth and success depends on recruiting and retaining top talent who share our mission and values. In particular, we are looking for individuals whose passion for creating life-changing medicines will inspire hands-on engagement and an ability to seek out novel solutions in the face of adversity. If you are interested in joining the BridgeBio team, please contact us at info@bridgebio.com

Partners

BridgeBio is on the lookout for partners with whom we can establish new programs or collaborate on our existing projects that aim to make a meaningful difference for patients with genetic diseases.

If you have an investment opportunity in which we might be interested, please contact Michael Henderson (mh@bridgebio.com). For interest in any current BridgeBio program, please contact Cameron Turtle (ct@bridgebio.com).

Investors

Contact

421 Kipling St
Palo Alto, CA 94301

Phone
650-391-9740

Email
info@bridgebio.com

© 2017 BridgeBio Inc.

KRAS cancers

KRAS is a protein involved in a regulating cell proliferation. Mutations in the gene that encode the protein lead to the development of numerous cancer types including lung, colorectal, and pancreatic.

BridgeBio’s subsidiary, Theras, is applying two novel approaches to inhibit KRAS. The treatment is currently in pre-clinical development.

PKAN

Pantothenate kinase-associated neurodegeneration (PKAN) is caused by mutations in PanK2, an enzyme involved in the synthesis of coenzyme A. PKAN causes a variety of neurological symptoms that begin in childhood and are progressive, often resulting in death by early adulthood.

BridgeBio’s subsidiary Coa is developing a targeted treatment for PKAN that is currently in preclinical development.

C-RAF cancers

C-RAF proteins regulate a variety of cellular processes including growth, differentiation, and survival. Mutations in these proteins can cause unintended and overactive signaling resulting in tumor development, particularly in pancreatic and colorectal cancers.

BridgeBio’s subsidiary Quartz is developing a molecule that selectively binds C-RAF and targets the protein for degradation, halting the overactive signals.

SHP-2 cancers

SHP-2 is a signaling protein involved in cell growth, differentiation, and the immune response. Mutations in the protein can cause rare cancers, and altered SHP-2 activity may contribute to numerous common tumor types. BridgeBio’s subsidiary Navire is developing a SHP-2 inhibitor to treat cancers driven by abnormal SHP-2 activity or in combination with agents targeting other anti-tumor pathways. The program is currently in pre-clinical development.

Dystrophic epidermolysis bullosa

Dystrophic epidermolysis bullosa (DEB) is a rare, genetic disease caused by mutations in the gene encoding collagen 7, a protein involved in the attachment of the epidermal layer of skin to the dermis. The disease results in children with incurably fragile, blistering skin, deformed limbs, and significant co-morbidities leading to early death.

BridgeBio’s subsidiary, Phoenix Tissue Repair, is developing a collagen replacement therapy to treat DEB. The treatment is in pre-clinical development.

Netherton syndrome

Netherton syndrome is caused by mutations in the SPINK5 gene, which encodes a protein called LEKT1 that helps control the breakdown of other proteins in the skin and thymus. These mutations cause a severe disorder of the skin, hair, and immune system.

BridgeBio’s subsidiary, MoST, is develop a kallikrein inhibitor that to address the dermatologic consequences of Netherton syndrome. The molecule is currently in pre-clinical development.

Hailey-Hailey disease

Hailey-Hailey disease is caused by mutations in the ATP2C1 gene, which encodes a protein called hSPCA1 that transports the signaling ions Ca2+ and Mn2+. These mutations impair protein processing, causing rashes and blisters affecting neck, armpits, skin folds and genitals. Symptoms can present at any stage of life, though most commonly by the third or fourth decade, but do not generally impair life expectatncy.

BridgeBio’s subsidiary, Dermecular, is developing a S1P-lyase inhibitor to treat Hailey-Hailey disease. The molecule is currently in Phase 1 clinical trials.

Darier disease

Darier disease is caused by mutations in the ATP2A2 gene, which encodes a protein called SERCA2 that transports the signaling ion Ca2+. These mutations may affect the production and distribution of essential proteins in the skin, leading to rashes and sometimes pain in affected regions. Neuropsychiatric conditions are also associated with the disease. Darier disease can present at any stage of life, though usually from age 6-20, but is not known to significantly impair life expectancy.

BridgeBio’s subsidiary, Dermecular, is developing a S1P-lyase inhibitor to treat Darier disease. The molecule is currently in Phase 1 clinical trials.

Transthyretin polyneuropathy

Transthyretin (TTR) is a relatively abundant protein in the blood, but can destabilize due to mutations or age, leading to aggregation and accumulation of TTR amyloid fibrils in the peripheral nerves. The accumulation causes amyloid TTR polyneuropathy (ATTR-PN), which sensory, motor, and autonomic dysfunction. ATTR-PN generally onsets later in life (60+), but can be fatal and progress rapidly.

BridgeBio’s subsidiary, Eidos, is developing AG10 to treat ATTR-PN. AG10 binds to TTR in the blood and stabilizes the protein, preventing its mis-folding and aggregation in the peripheral nerves. Ph1 clinical trials of AG10 are planned for summer 2017.

Transthyretin cardiomyopathy

Transthyretin (TTR) is a relatively abundant protein in the blood, but can destabilize due to mutations or age, leading to aggregation and accumulation of TTR amyloid fibrils in the heart. The accumulation causes amyloid TTR cardiomyopathy (ATTR-CM), which is characterized by diastolic heart failure and conduction abnormalities. ATTR-CM generally onsets later in life (50+), but can be fatal and progress rapidly.

BridgeBio’s subsidiary, Eidos, is developing AG10 to treat ATTR-CM. AG10 binds to TTR in the blood and stabilizes the protein, preventing its mis-folding and aggregation in the heart. Ph1 clinical trials of AG10 are planned for summer 2017.

Frequency basal cell carcinoma

Basal cell carcinomas (BCCs) are the most common form of skin cancer and are known to recur. Patients with >5 BCCs are termed “frequent flyers” and often have mutations in at least one PTCH1 gene, which encodes the patched-1 receptor that acts to suppress cell proliferation.

BridgeBio’s subsidiary, PellePharm, is developing Patidegib to treat frequent BCC. Patidegib acts downstream of patched-1 on the hedgehog pathway by inhibiting a protein called smoothened. Patidegib is in Phase 2 clinical trials in the US and UK.

Gorlin syndrome

Gorlin syndrome is caused by mutations in the PTCH1 gene, which encodes the patched-1 receptor that acts to suppress cell proliferation. These mutations prevent the production of patched-1, or result in a defective form of the protein, causing cell proliferatio. People with Gorlin syndrome have an elevated risk of developing benign and cancerous tumors, commonly leading to the development of multiple basal cell carcinomas requiring repeat surgical excision, and a range of other clinical symptoms.

BridgeBio’s subsidiary, PellePharm, is developing Patidegib to treat Gorlin syndrome. Patidegib acts downstream of patched-1 on the hedgehog pathway by inhibiting a protein called smoothened. Oral, systemic smoothened inhibitors have demonstrated clinical efficacy in treating frequent basal cell carcinomas, but result in significant side effects. PellePharm aims to replicate the efficacy of these therapies while limiting side effects using the topical Patidegib.