Analyst Conference Summary

Biotechnology

conference date: May 6, 2021 @ 2:00 PM Pacific Time
for quarter ending: March 31, 2021 (Q1, first quarter)

Forward-looking statements

Overview: Rapidly growing Reblozyl royalty revenue, but deep in the red for further pipeline development.

Basic data (GAAP):

Revenue was $24.8 million, up sequentially from $25.9 million, and up from $4.3 million year-earlier.

Net income was negative $63.6 million, down sequentially from negative $57.4 million, and down from negative $51.6 million year-earlier.

Earnings per Share (EPS, diluted) were negative $1.05, down sequentially from negative $0.95, and down from negative $0.95 year-earlier.

Guidance:

Believes has cash sufficient for the foreseeable future.

Conference Highlights:

Habib Dable, CEO of Acceleron said: "With the Stellar Phase 3 trial already enrolling patients, we expect to initiate multiple mid- and late-stage clinical trials across our pipeline in pulmonary disease throughout 2021. We were thrilled to have data from the Pulsar Phase 2 trial of sotatercept in PAH recently featured in the New England Journal of Medicine. These data along with the results from our ongoing and planned Phase 3 trials will support our vision of establishing sotatercept as a backbone therapy for patients with all stages of PAH. In our hematology program, Acceleron and our partner Bristol Myers Squibb are leveraging the key learnings from the early success of the commercial launch of Reblozyl in the United States. We continue to transition from the initial bolus patient group earlier in the launch to underlying new patient demand. Bristol Myers Squibb expects launches in multiple additional countries across the globe this year as Reblozyl receives reimbursement. Most importantly, assuming success in current and future potential indications associated with anemia, we continue to estimate that peak annual sales of Reblozyl will surpass $4 billion."

All revenue was from collaboration partner Bristol Myers Squibb (BMY).

Reblozyl royalty revenue in Q1 was $22.4 million, down sequentially from $23.0 million. Based on BMY sales of $112 million. $2.4 million was cost share revenue. The sequential decline in Q1 2021 was due to the tapering off of the orginal bolus of patients. Believes will return to growth in 2H 2021, and later from label expansions.

Non-GAAP net income was negative $47 million, down from negative $43 million year-earlier. Non-GAAP EPS was negative $0.77, up from negative $0.80 year-earlier, due to increase in sharecount to 60.6 million from 53.4 million.

Reblozyl (Luspatercept) received FDA approval for MDS in April 2020 after having received for beta-thalassemia Q4 2019. Also approved in the EU and Canada. A phase 3 trial for myelofibrosis patients who are being treated with JAK inhibitor is ongoing. Bristol Myers Squibb expects to initiate the Phase 3 INDEPENDENCE trial in patients with MF on concomitant JAK 2 inhibitor therapy who require RBC transfusions in Q1 2021. Enrollment is ongoing in the COMMANDS Phase 3 trial in patients with treatment-naive, lower-risk MDS, with topline results expected in 2022. The BEYOND Phase 2 trial in NTD (non-transfusion dependent) beta thalassemia will report results in June 2021.

Reblozyl (luspatercept) royalties from BMY will be in the low to mid 20% range. Believes sales in the first two indications could reach $2 billion annually, resulting in about $400 million per year in royalties.

In Q4 2020 Acceleron signed a collaboration and license agreement with Fulcrum Therapeutics to identify small molecules to modulate specific pathways associated with a targeted pulmonary disease indication.

Acceleron plans to present updates from the PULSAR and SPECTRA Phase 2 trials of sotatercept in patients with PAH at the American Thoracic Society 2021 conference, held May 14-19. Acceleron launched the Phase 3 trial, STELLAR, in December 2020. Acceleron plans to initiate the HYPERION (early intervention) Phase 3 trial and the ZENITH (later intervention) Phase 3 trial in expanded PAH populations in the middle of 2021. In November 2020 positive Phase 2 trial results were presented.

Acceleron expects to start a Phase 1b/Phase 2 study to evaluate the activity of ACE-1334 in patients with SSc-ILD (Systemic Sclerosis-associated Interstitial Lung Disease) in 2021.

See also Acceleron pipeline.

Cash and equivalents ended at $795 million, up sequentially from $671 million. No debt. Believes has sufficient cash to operate into .

$57 million was spent on R&D and $31 million on general and administration. Total op ex was $88 million. Loss from operations was $64 million. Other income $0 million.

Q&A Summary:

European Reblozyl launches? So far Austria and Germany have driven launch. In 2H should see reimbursements kick in, could be Belgium, France, Italy, nordic countries.

Reason for expecting Reblozyl rebound in US in 2H? Continued increase in covid vaccinations, so patients seeing physicians more. MDS saw about a 10% to 20% decrease in new patient visits in 2020. Patient mix should improve in 2H, from bolus of heavily transfused patients to the target population that are expected to do better and stay on drug longer.

Sotatercept walk data? No change in medicines in trial protocol. Walk distance change was 50 meters for most patients. Even in very upfront setting our walk distance results compare well with prior double and triple therapies.

PAH data to be presented in May? Update is for week 48, trial in ongoing, safety update and walk update.

Commands data should be in by the end of 2022.

In a progressive disease like PAH we expect to see continued improvement over time. We believe the 6 month endpoint six minute walk is solid, including buy in from FDA. We also have a trial with morbidity and mortality as an endpoint. In Zenith we will look at both functional improvement and mortality. We believe a solid Phase 2 program greatly increases the probability of success in Phase 3.

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