Analyst Conference Summary

biotechnology

conference date: February 25, 2021 @ 1:30 PM Pacific Time
for quarter ending: December 31, 2020 (fourth quarter, Q4)

Forward-looking statements

Overview: First NDA submission planned for later in 2021.

Basic data (GAAP):

Dicerna released full year 2020 without specific Q4 2020 data, which I find unacceptable.

Revenue was $ million, up sequentially from $49 million, and up from $ million year-earlier.

Net income was negative $ million, up sequentially from negative $22 million, and up from negative $ million year-earlier.

Diluted EPS was negative $, up sequentially from negative $0.29, and up from negative $ year-earlier.

Guidance:

Cash should last to 2023. Expects about $100 million from collaborators in 2021.

Conference Highlights:

Douglas Fambrough, CEO of Dicerna said " Led by pivotal-stage nedosiran for primary hyperoxaluria, belcesiran for alpha-1 antitrypsin deficiency-associated liver disease, RG6346 for chronic HBV and ANGPTL3-targeted LY3561774 for dyslipidemia, we expect the clinical-stage pipeline of GalXC and GalXC-Plus drug development candidates to grow by two more programs by mid-year 2021 and at least four more by the end of 2022. We also expect a similar number of preclinical development programs targeting diverse tissues moving forward. Meanwhile, our balance sheet is strong, and our collaborative partnerships are expected to continue contributing non-dilutive funding to support our burgeoning clinical development efforts over the coming years. Of particular note, we look forward to Roche’s near-term planned initiation of RG6346 in a Phase 2 combination trial for HBV, the initiation of our Phase 2 trial of belcesiran and top-line data from our PHYOX2 pivotal trial of nedosiran approximately mid-year."

On Feb. 25, 2021 announced DCR-AUD, an investigational GalXC RNAi therapy for the treatment of alcohol use disorder (AUD), is planned for an IND in Q3 2021. Dicerna believes the properties of its RNAi technology have the potential to transform AUD treatment by developing, for the first time, a potentially long-acting, well-tolerated, conveniently subcutaneously delivered, highly targeted therapeutic candidate to inhibit ALDH2, a key enzyme in alcohol metabolism.

5 developmental candidates are nearing initial clinical trials. Over 5 years expects to have 2 commercial products in the U.S. plus royalties from collaborations.

Cash and equivalents balance ended at $569 million, down sequentially from $610 million.

The PHYOX2, a long-term, double blind, study of nedosiran (DCR-PHXC) for the treatment of PH (primary hyperoxaluria) types 1 and 2 completed enrollment in Q1 2021. Has Breakthrough Therapy Designation for DCR-PHXC for the treatment of primary hyperoxaluria type 1. Expects top line data midyear and could file an NDA in Q3 2021. In October 2020 presented positive interim daa from the open label extension study. In Feb 2021 dosed first patient in PHYOX4 for PH3. Dicerna believes its data will be sufficient for approval and are best-in-class.

RG6346 for HBV reported positive interim Phase 1 data in Q4 2020. Roche plans to initiate RG6346 in a Phase 2 combination trial in Q1 2021.

In November 2020 the FDA accepted the IND filed by Eli Lilly for LY3561774, the first clinical-stage candidate targeting the ANGPTL3 gene for the treatment of dyslipidemia. The IND milestone achievement triggered a $10.0 million payment to Dicerna.

Alnylam partnered DCR-A1AT/ALN-AAT02 program selection for advancement into Phase 2. Alnylam has an option to pick up ex-US commercial rights. Selected the Dicerna DCR-A1AT molecule for Phase 2. Phase 1 data at a midyear medical meeting.

Dicerna announced, in Q3 2020, positive preclinical data demonstrating expansion of its technology and discovery efforts beyond its hepatocyte-focused GalXC RNAi technology to central nervous system (CNS), skeletal muscle and adipose tissues. The data demonstrated consistent and durable CNS-wide target mRNA knockdown using novel constructs regardless of route of administration (intrathecal [IT] or intracisterna magna [ICM]) and reduction in target mRNA (messenger RNA) in skeletal muscle and adipose tissue using optimized chemistries, resulting in equivalent and potentially highly durable target knockdown regardless of dosing regimens.

Collaborations with Novo Nordisk, Alnylam and Lilly are proceeding well.

No Q4 2020 numbers issued. Operating expense of $ million consisted of $ million for R&D and $ million for general and administrative expense. Loss from operations was $ million. Interest income $ million.

Q&A summary:

AUD subpopulations, endpoints, regulatory path? It is a diverse indication, so we are thinking about subgroups. Harm reduction is the crucial metric.

Differences in PH 1, 2, 3, commercialization? PH2/3 is a singular commercialization effort, the PH community has not been divided into types for long. Diagnosis, support, genetic testing are important.

HBV combinations? Roche's combination trials are exploring for the highest cure rate. Will be with nuc and (missed) other specifics.

ANG3 differentiation from multiple competitors? We decided to pursue cardiovascular partners several years ago, partly because of competition, plus large target populations. No rare disease economics. So we are working with Lilly.

C3 and CFB are very interesting targets to move forward, we just have to work with Alexion on the path forward.

The $100 million guidance is cash in, it is independent of revenue accounting.

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