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North America Equity Research

December 2019

J.P. Morgan US Biotechnology Research

2020 US Biotech Outlook: Roaring into the New Year with 2020 Vision

Conference Call Details

We are hosting a conference call on Wednesday, December 18 @ 11:00am ET / 16:00 UK

Please contact us or your JPM salesperson for details

Anupam Rama

212-622-0900

anupam.ram[email protected]

US Large Cap Biotechnology

Cory Kasimov

212-622-5266

cory.w.kasimov@jpmorgan.com

US SMID Biotechnology

Jessica Fye

212-622-4165

Jessica.m[email protected]

Eric Joseph

212-622-0659

[email protected]

Yuko Oku

212-622-5374

yuko.oku@jpmorgan.com

Tessa T Romero

212-622-4484

tessa.t.romero@jpmorgan.com

Gavin Scott

212-622-0579

[email protected]

Matthew Holt, Ph. D.

212-622-9602

[email protected]

Daniel Wolle

212-622-5805

daniel.wolle@jpmorgan.com

Matthew Bannon

212-622-0001

[email protected]

Turner Kufe, M.D.

212-622-4104

turner.kuf[email protected]

See the end pages of this presentation for analyst certification and important disclosures, including non-US analyst disclosures.

J.P. Morgan does and seeks to do business with companies covered in its research reports. As a result, investors should be aware that the firm may

have a conflict of interest that could affect the objectivity of this report. Investors should consider this report as only a single factor in making their

investment decision.

J.P. Morgan Securities LLC

2020 US Biotech Outlook: Key Takeaways

Source: J.P. Morgan Research

Solid Fundamentals, Stabilizing Sentiment Favor Rotation to Biotech...the Question is When and How Much

Valuation gap for large caps vs other HC subsectors / broader market may finally be too big to ignore. . .

While there were early signs of rotation late in 2019, the group remains significantly under-owned

 Large cap valuations continue to trade at a steep discount to pharma (~11x 2020 EPS for the remaining big 3 biotech vs. ~14x for major pharma),

healthcare overall (~21x), and the broader market (~18x for the S&P500)

 Commercial / near commercial / platform / larger cap SMIDs could also benefit from sector rotation

. . . and fundamentals remain strong. . .

The innovation engine is still running at a very high level

 Clinical data / innovation continues to be the #1 ranked sector tailwind for 2020 in our buyside survey (tied with increasing M&A)

 The FDA remains constructive, with 40+ new drugs or biologics approved YTD (vs. 59/46/22 in 2018/2017/2016)

 A number of high-profile/innovative biotech IPOs captured investors’ attention in 2019 (e.g., Stoke, Springworks, BioNTech, etc.)

 Importantly, many of the new offerings of last 12-24 months have created substantive value, underscoring the impact of innovation

. . . which, coupled with easing headwinds, has encouraged movement from the sidelines

Healthcare policy discussion remains at the forefront, but political/pricing headline (and fundamental) fears appear to be receding

 As the challenge of implementing anything close to “Medicare for All” becomes better appreciated, along with the overall lack of alignment on specific

reform policies, we believe the valuation disconnect vs the broader market could lessen

 With the expectation of divided government in 2021 (e.g., if a Dem wins the White House, we would still expect a Republican majority Senate), we see

“gridlock” as ongoing impediment to large scale legislation, despite general bipartisan rhetoric in support of healthcare reform

 Although 52% of buyside survey respondents expected drug pricing to be a similar or bigger overhang in 2020 vs. 2019, this is significantly down form the

82% of respondents in our 2019 survey (for 2019 vs. 2018)

What, if anything, can carry 4Q19’s sentiment momentum into 2020?

One perpetual swing factor remains M&A. . .

 Uptick in M&A activity helped to lift biotech sentiment exiting 2019, after what had largely been an underperforming year vs. the broader market

 While our buyside survey suggests heightened M&A is still anticipated, a year of increased deal activity could act as a rising tide across the group

. . . and demonstrating meaningful medical advances with clinical data “wins” will always factor into enthusiasm for biotech

 This is particularly important for broader investor engagement in SMIDs

2020 US Biotech Outlook: Top Picks Summary

Cory Kasimov

 Vertex (VRTX) – As we’ve been signaling going back to last summer, we think VRTX is very well positioned for 2020 (even with the run

into year end). This is the cleanest story in biotech, and the next 12 months potentially offers something for everyone. On the one hand,

the earlier than expected approval of the triple regimen could trigger a substantial growth spurt that attracts a broader investor

audience. Further, important non-CF pipeline updates (AAT in particular) have the potential to keep specialists engaged and, if positive,

lead to another re-rating of shares.

 BioMarin (BMRN) – After being range bound for several years, BMRN could finally be poised to break out in 2020. On the heels of recent

positive Phase 3 data for vosoritide in achondroplasia, shares could benefit in 2020 from budding anticipation for the approval of two

new potential blockbuster products (valrox and vosoritide), improving financial performance (growing sales with stabilizing expenses),

more valrox data (though admittedly some might debate whether this is a good or bad thing), and increased strategic value.

Anupam Rama

 MyoKardia (MYOK) – The key catalyst to monitor in 2020 for MyoKardia will be the phase 3 EXPLORER-HCM readout of mavacamten in

obstructive hypertrophic cardiomyopathy (oHCM). These data are expected in 2Q20 and have the potential to be transformational for

the company. We continue believe EXPLORER has a high probability of success based on prior phase 2 / long-term extension PIONEER

data and trial design. We believe mavacamten has blockbuster potential in oHCM alone.

 Apellis (APLS) – As an early 2020 catalyst, results from the phase 3 PEGASUS study of APL-2 in paroxysmal nocturnal hemoglobinuria

(PNH) could topline the week before the 2020 J.P. Morgan Healthcare Conference. In our view, PEGASUS has a high probability of

showing a clinically meaningful increases in hemoglobin relative to Soliris in baseline anemic patients (a potential ~$400-500 peak

opportunity). Importantly, PEGASUS success could expand interest in the broader set addressable indications, including cold agglutinin

disease (CAD), complement 3 glomerulopathy (C3G), and geographic atrophy (GA).

Source: J.P. Morgan Research

2020 US Biotech Outlook: Top Picks Summary, Continued

Jessica Fye

 Ascendis (ASND) – Looking to 2020, we see a string of potential catalysts, each of which we expect to be de-risking for the pipeline and

have positive impact on the stock. Building on the recent positive phase III vosoritide data supporting proof of concept for TransCon CNP

we will watching for 1) PTH phase II data in 1Q, 2) phase III details for somatrogon which should reinforce the differentiation of TransCon

GH, 3) the 6-month PTH phase II data (potential topline in 3Q), and 4) initial data for TransCon CNP perhaps late in 2020/ 2021. Taken

together, we see a compelling case for continued steady value creation from a now de-risked platform and believe the Ascendis story is

still in the early innings with multiple untapped levers in the model. Bigger picture, we believe the heiGHt results support Ascendis’s

ability to meaningfully improve upon the profile of parent molecules and see positive read-across not only to the other orphan

endocrine products (PTH and CNP) but also to the emerging oncology pipeline for which we expect the first IND next year.

Eric Joseph

 Karyopharm (KPTI) – Core focus remains on top-line data from the phase 3 BOSTON study (selinexor plus Velcade/dex) anticipated early

2020 (a $1.2B+ blockbuster sales opportunity for selinexor). We maintain high level confidence in BOSTON’s probability of success based

on i) SVd PFS performance in phase 1b STOMP study (17.8mos), ii) consistent historical performance of the control Vd regimen (~7 to

~9.5 mos), and iii) FDA commentary in conjunction with the accelerated approval of Xpovio (selinexor) in July. In addition, on the heels of

impressive early launch metrics (putting to rest real-world tolerability concerns, in our view), we see upside potential to an already

healthy mid-term growth outlook for Xpovio (FY20/21 JPMe: $71M/$143M), given the label expansion opportunity in r/r DLBCL (sNDA

submission in 4Q19). While we view myeloma and B-cell malignancy as the primary value drivers for Xpovio (peak combined US

forecasts of ~$1.5B), we note that additional phase 3 readouts in solid tumor settings beginning in 2020 have the potential to expand

commercial breadth longer-term. Finally, with commercial-stage small molecule oncology maintaining a dominant core within biotech

M&A, we anticipate a rising strategic premium to KPTI shares provided BOSTON success.

Source: J.P. Morgan Research

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