Analyst Conference Summary

Biotechnology

conference date: February 25, 2021 @ 2:00 PM Pacific Time
for quarter ending: December 30, 2020 (Q4, fourth quarter)

Forward-looking statements

Overview: Rapidly growing Reblozyl royalty revenue.

Basic data (GAAP):

Revenue was $25.9 million, up sequentially from $22.6 million, and down from $39.3 million year-earlier.

Net income was negative $57.4 million, down sequentially from negative $39.2 million, and down from negative $23.6 million year-earlier.

Earnings per Share (EPS, diluted) were negative $0.95, down sequentially from negative $0.66, and dpwm from negative $0.44 year-earlier.

Guidance:

Believes has cash sufficient for the foreseeable future.

Conference Highlights:

Habib Dable, CEO of Acceleron said: "2020 was a very productive year for Acceleron as we generated important results in our lead pulmonary program to advance sotatercept into a registrational Phase 3 trial and executed on a successful first full year of the commercial launch of Reblozyl in the United States. Throughout the year, we presented updates from our two Phase 2 trials with our lead pulmonary program, supporting our long-term vision of establishing sotatercept as a backbone therapy for patients with pulmonary arterial hypertension across all stages of the disease. As we continue to grow our capabilities in rare pulmonary disease, we are also advancing ACE-1334 into a Phase 1b/Phase 2 trial in patients with systemic sclerosis-associated interstitial lung disease later this year."

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

Reblozyl royalty revenue in Q4 was $23.0 million, up sequentially from $19.3 million. Based on BMY sales of $115 million. $2.9 million was cost share revenue.

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 could have results in Q1 2021.

But expects a flattening of Reblozyl royalty growth in Q1 2021; launches in new countries will help later in the year. Longer term there should be label expansion.

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.

In April 2020 the FDA gave sotatercept in patients with PAHi n Breakthrough Therapy designation and the EMA gave it Priority Medicines designation. Acceleron has full rights. 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 $671 million, down sequentially from $888 million. No debt. Believes has sufficient cash to operate into .

$57 million was spent on R&D and $26 million on general and administration. Total op ex was $83 million. Loss from operations was $58 million. Other income $0 million.

The share count increased from 46.2 million in Q4 2019 to 60.3 million in Q4 2020.

Q&A Summary:

Reblozyl flattening in Q1? Shift in demand to patients earlier in their MDS journey. But focus is on further penetration of the population. BMS is investing in Reblozyl education, so we have high confidence in rising sales long term.

BEYOND data still expected in the first half of 2021.

Duration of dosing? Pent up demand is high. Benefit approaches 2 years. Early patients had a higher transfusion burden. Newer patients are earlier in the disease, so persistence should increase over time.

In nations where there are new launches there should be a bolus due to pent up demand.

1334 target populations, subsets? We want patients with evidence of skin disease as well as with pulmonary involvement. We will learn more about the patients during the study.

1334 Phase 1b/2 study start time, size? In process of starting it now. Working on regulatory hurdles, site set up. 1b is cohort driven. Size will be a function of treatment effect. We are just getting started, but it could work in other fibrotic diseases.

$4 billion annual peak sales potential? Assumes all label expansion is successful, including unnamed potential indications.

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