March 29, 2018

What a Difference Another Year Makes

(From the 2017 Annual Report)  

Dear Shareholders,

We are a little more than one year into our new business plan focused on BLU-5937 for chronic cough. I would like to tell you about the feedback we’ve received to date and how 2018 is shaping into a milestone year as we enter our first in-human studies.

Positive Feedback

When we first in-licensed BLU-5937 in February 2017, we vetted the project internally and with our Board of Directors; as well as with select key opinion leaders and consultants. We were confident that we had a robust project and appreciated the positive feedback we received once we announced our new core program. Public markets responded positively, and physicians and patients acknowledged that chronic cough is an important condition with real unmet need. Importantly, for a project that needed to be financed, prospective investors opened their doors and took meetings.

Data Drives Financing

Our pitch deck was 20 slides, but the investment proposition was straightforward and compelling:

Refractory Chronic Cough – Large Unmet Medical Need

Important patient population that does not respond to current therapies

P2X3 – A Validated Target

Merck’s MK-7264 is a P2X3 antagonist that has shown strong efficacy but with altered or loss of taste in 80% of patients

BLU-5937

P2X3 antagonist with potential to have similar efficacy as MK-7264 with limited or no taste loss side effect

Experienced Team

Track record of drug development plan execution

Key Data (September 2017)

BLU-5937 showed equivalent anti-cough effect and no taste loss compared to MK-7264 in animal studies

Efficient Plan

First in-human study in 2018 to assess drug safety, including taste loss, in healthy subjects. Study in chronic cough patients in 2019

In September 2017, we released additional animal data that supported BLU-5937 as a potentially best in class therapeutic. Having demonstrated enough progress to provide a clear path toward clinical testing by Q3 2018, investor traction culminated in a $20M financing that closed in mid-December.

Taste Data in 2018

Obtaining human data is a key driver of our chronic cough program, and we are planning to start our clinical Phase 1 study in healthy subjects in Q3 2018. This study will provide data on dosing, safety and tolerability of BLU-5937 including whether subjects experience taste alteration, or partial or complete taste loss. Based on our animal studies, we are hopeful that we will observe few or possibly no adverse effects on taste function in humans at the targeted therapeutic dose, which would solidify BLU-5937’s potential as a best in class P2X3 antagonist for chronic cough.

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We would like to thank all of our shareholders, new and longstanding, for their valuable support. We are proud of the progress we made in 2017 and plan to build on this momentum with continued positive execution of our plan in 2018 and beyond.

Sincerely,

Roberto Bellini

President and Chief Executive Officer

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