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BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation...

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BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL OFFICER October 2019
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Page 1: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

B A R R E T T K A T Z , M D , M B AP R E S I D E N T A N D C H I E F M E D I C A L O F F I C E ROctober 2019

Page 2: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

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Forward-Looking StatementsStatements in this Presentation that are not statements of historical fact are forward-looking statements. Such forward-looking statements include, without limitation, statements regarding our research and clinical development plans, expected manufacturing capabilities, strategy, regulatory matters, market size and opportunity, future financial position, future revenue, projected costs, prospects, plans, objectives of management, and the Company’s ability to complete certain milestones. Words such as “believe,” “anticipate,” “plan,” “expect,” “intend,” “will,” “may,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements necessarily contain these identifying words. These forward-looking statements are based on the beliefs of the Company's management as well as assumptions made by and information currently available to the Company. Such statements reflect the current views of the Company with respect to future events and are subject to known and unknown risks, including business, regulatory, economic and competitive risks, uncertainties, contingencies and assumptions about the Company, including, without limitation, risks inherent in developing therapeutic products, future results from the Company's ongoing and planned preclinical studies and clinical trials, the Company's ability to obtain adequate financing to fund its preclinical studies and planned clinical trials and other expenses, trends in the industry, the legal and regulatory framework for the industry and future expenditures. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. The actual results may vary from the anticipated results and the variations may be material. These forward-looking statements should not be taken as forecasts or promises nor should they be taken as implying any indication, assurance or guarantee that the assumptions on which such forward-looking statements have been made are correct or exhaustive or, in the case of the assumptions, fully stated in the Presentation. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date this Presentation is given. This Presentation discusses product candidates that are under preclinical study or clinical trial and which have not yet been approved for marketing by the U.S. Food and Drug Administration (the “FDA”). No representation is made as to the safety or effectiveness of these product candidates for the therapeutic use for which such product candidates are being studied.

Certain information contained in this Presentation relates to or is based on studies, publications, surveys and other data obtained from third-party sources and the Company’s own internal estimates and research. While the Company believes these third-party sources to be reliable as of the date of this Presentation, it has not independently verified, and makes no representation as to the adequacy, fairness, accuracy or completeness of, any information obtained from third-party sources. In addition, all of the market data included in this Presentation involves a number of assumptions and limitations, and there can be no guarantee as to the accuracy or reliability of such assumptions. Finally, while the Company believes its own internal research is reliable, such research has not been verified by any independent source.

Page 3: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

BridgeBio Pharma

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Our mission

• To find, develop, and deliver breakthrough medicines for genetic diseases to patients as quickly and safely as possible.

Our approach

• Create a bridge between remarkable advancements in genetic science and patients with unmet needs, driven by an entrepreneurial engine, to develop lifechanging medicines as rapidly as possible.

Page 4: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

BridgeBio Pharma core values

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Put patients first

Think independently

Do everything as fast as possible

Be radically transparent

Let science speak

Page 5: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

BridgeBio: A new model for the genetic disease space

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Deep understanding of a privileged target spaceIdentify high value opportunitiesand novel approaches

Unencumbered target selectionNew opportunities not limited by potential peak-year sales orexisting infrastructure.

Capabilities to enter the “chasm of death” Unparalleled team of experienced translational scientists and company builders that enables us to pursue ultra-early-stage academic assets.

Fit-for-purpose program building

Minimum viable program startup with extreme willingness to fail.

Aligned agendas Incentives at the level of each

program for program management teams and scientists.

Play-calling on the fieldDecentralized decision-making left

to the most appropriatedecision makers.

We build a focused team of experts around each program, dedicated to advancing it towards patients

Our model The genetic disease space

Page 6: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

Mendelian

BBP-265 (Eidos) TTR stabilizer for ATTR >400K

BBP-831 FGFR1-3i for achondroplasia 55K

BBP-870 cPMP replacement for MoCD Type A 100

BBP-0094 Topical SMOi for Gorlin / frequent BCC 120K

BBP-589 COL7A protein replacement for RDEB 1.5K

BBP-681 Topical PI3Kai for venous malformations 117K

BBP-671 PanK 1/3 activator for PKAN, OAs 7K

BBP-418 Glycosylation substrate for LGMD2i 7K

BBP-711 GO1i for PH1 / FSF 5K / 1.5M

BBP-561 Topical KLKN 5/7i for Netherton 11K

BBP-761 Succinate pro-drug for LHON 20K

Oncology

BBP-831 FGFR1-3i for FGFR+ cancers 37K*

BBP-454 Pan-mutant KRASi for KRAS+ cancers >500K*

BBP-398 SHP2i for multiple cancer types >500K*

BBP-954 GPX4i for multiple cancer types >500K*

Gene Therapy

BBP-631 AAV5 gene therapy for CAH >75K

BBP-812 AAV9 gene therapy for Canavan 1K

We have built a sizeable pipeline over the past four years

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ClinicalPre-Clinical

Lead Finding / Optimization

IND Enabling Phase 1 Phase 2 Phase 3

Patient pop.

(US+EU)2MOA / DiseasesProgram1 Modality

1 Each of our programs is housed in a separate subsidiary; 2 Patient population: Prevalence except for asterisked figures which represent incidence; 3A clinical trial we believe could support filing an application for marketing authorization, although the FDA and other regulatory authorities have not indicated their agreement or that additional trials will not be required; 4 We are party to an option agreement pursuant to which LEO Pharma A/S has been granted an exclusive, irrevocable option to acquire PellePharm, including the BBP-009 program. If the option is exercised by LEO Pharma A/S, we will no longer have rights to develop and commercialize BBP-009. See “Business —Our Material Agreements—BBP-009 (Patidegib): Option Agreement with LEO Pharma A/S.” 5Planned New Drug Application (NDA) submission for the treatment of cholangiocarcinoma (CCA) as a second-line or later therapy (2L)

Key Value Drivers Phase 2/3 clinical development3 Small molecule Biologics Gene therapyTopical small molecule

5

Page 7: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

• KKR

• Viking Global Investors

• Perceptive Advisors• Aisling Capital

• Cormorant Capital• AIG

• Sequoia Capital

• Hercules Capital

BridgeBio closes $300 million financing, January 2019

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Page 8: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

BridgeBio upsizes IPO to $348M, June 2019

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Page 9: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

What does this mean for the future?

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Is positioned to be a major player in genetic disease space

Is focused on diseases with clear genetic drivers and unmet patient need

Has the capital, intellectual horsepower, and model to deliver

Is always searching for programs to add to its pipeline

Has a significant interest in Ophthalmology

BridgeBio Pharma…

Page 10: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

Mutations in LRAT or RPE: inability to recycle 11-cis-retinal

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All-trans-retinol

Retinyl esters

All-trans-retinal

11-cis-retinol

11-cis-retinal

11-cis-retinal

All-trans-retinalconjugates

Retinal pigmented epithelium

(RPE)

Rod outer segment

(ROS)

Rhodopsin

Opsin

Light

All-trans-retinol

X

Mutations in LRAT or RPE65 lead to deficiency in the ability to cycle 11-cis-retinol/retinal and an inability to complete the visual cycle, resulting in compromised visual function

Page 11: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

Oral 9-cis retinoid for childhood blindness due to Leber congenital

amaurosis caused by RPE65 or LRAT mutations: a phase 1b trial

Lancet. 2014 Oct 25; 384 (9953):1513-20. Epub 2014 Jul 13.

Koenekoop RK, Sui R, Sallum J, van den Born LI, Ajlan R, Khan A, den Hollander AI, Cremers FP, Mendola JD, Bittner AK, Dagnelie G, Schuchard RA, Saperstein DA

Oral retinoid replacing missing chromophore

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Page 12: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

Natural history – RPE65 mutations

Chung, 20xx

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Page 13: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

Natural history – RPE65 mutations

Chung, 20xx

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Page 14: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

Results of Ph Ib trial

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GVF Retinal area (mm2) IRD 01 IRD 02

Increase from baseline of ≥ 20% 18 (56%) 19 (70%)Increase from baseline of ≥ 30% 13 (41%) 15 (56%)

Visual Acuity IRD 01 IRD 02

Increase from bl of ≥ 5 ETDRS letters 18 (56%) 19 (70%)Increase from bl of ≥ 10 ETDRS letters 9 (28%) 2 ( 7%)

Page 15: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

Mean change in GVF retinal area

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Page 16: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

RP functional retinal area responders

15

8679

64

36

0

25

50

75

100

One eye Both eyes One eye Both eyes

RP (N=14)

≥ 20% response ≥ 40% response

% re

spon

ders

Change from baseline in GVF retinal area

Page 17: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

LCA functional retinal area responders

16

54

46

38 36

0

20

40

60

One eye Both eyes One eye Both eyes

LCA (N=13)

Change from baseline in GVF retinal area

≥ 20% response ≥ 40% response

% re

spon

ders

Page 18: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

We see ophthalmology as a family of diseases that will likely be shown increasingly to be genetically driven

BridgeBio knows ophthalmology

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We are constantly seeking programs wherein we can target at the source diseases with well-defined genetic drivers

We welcome your interests and collaboration

Page 19: BARRETT KATZ, MD, MBA PRESIDENT AND CHIEF MEDICAL … · BridgeBio_FINAL AAO OIS BB BK presentation 9.27 - VB Edits 9.29 WideScreen - Read-Only Created Date: 10/10/2019 12:45:35 AM

B A R R E T T . K A T Z @ B R I D G E B I O . C O MOctober 2019


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