Analyst Conference Summary

biotechnology

conference date: November 9, 2021 @ 5:30 AM Pacific Time
for quarter ending: September 30, 2021 (third quarter, Q3)

Forward-looking statements

Overview:

Basic data (GAAP):

Revenue was $63.0 million, up sequentially from $41.3 million, and up from $48.9 million year-earlier.

Net income was negative $17.1 million, up sequentially from negative $40.8 million, and up from negative $21.8 million year-earlier.

Diluted EPS was negative $0.22, up sequentially from negative $0.53, and up from negative $0.29 year-earlier.

Guidance:

Cash should last to 2025.

Conference Highlights:

Douglas Fambrough, CEO of Dicerna said "We had an exciting and productive third quarter as we continued to execute and advance key initiatives that we believe help position Dicerna for multiple value-creating milestones over the next 12 to 24 months. We look to build on this progress as we continue advancing core programs that include nedosiran for primary hyperoxaluria (PH), RG6346 for chronic hepatitis B virus (HBV) infection with Roche, belcesiran for alpha-1 antitrypsin deficiency-associated liver disease (AATLD) and DCR-AUD for alcohol use disorder. We also look forward to unveiling the next wave of innovations from Dicerna in early 2022 with the first of several wholly owned extrahepatic programs harnessing our GalXC-Plus technology"

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 and reported positive data in August 2021 for type 1, but not for type 2. An NDA could be submitted in Q1 2022. Has Breakthrough Therapy Designation for PH1. In November 2021 presented PHYOX2 data at the American Society of Nephrology. In June 2021 completed dosing of PHYOX4 for PH3 and presented data in October 2021 did not meet the efficacy endpoint, but met the primary safety endpoint. Dicerna believes its PH1 data will be sufficient for approval and are best-in-class. PHYOX7 for patients with severe renal impairment dosed first patient in May 2021, but will not have data in time for the FDA. Looking for an ex-US partner.

In September 2021 announced DCR-AUD, an investigational GalXC RNAi therapy for the treatment of alcohol use disorder (AUD), dosed its first Phase 1 patient. 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. The Phase 1 dosing level trial will begin in Q2 2021.

Belcesiran, the GalXC RNAi candidate for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (AAT-LD), reported Phase 1 interim data from healthy volunteers in July 2021. Dose-dependent reductions of AAT were shown with a single dose. The final, highest dose cohort is ongoing. In June 2021 initiated a Phase 2 trial.

In April 2021, Royalty Pharma plc agreed to acquire Dicerna's royalty interest in Oxlumo (lumasiran) for an upfront cash payment of $180 million and up to $60 million in contingent sales-based milestone payments. Oxlumo, which has been approved by the FDA and EMA for the treatment of primary hyperoxaluria (PH) type 1, is marketed by Alnylam.

Currently, with parnters, has 16 programs in clinical or preclical development. There are also 20 research-stage programs in development.

Cash and equivalents balance ended at $646 million, down sequentially from $710 million.

In Q3 2021 expenses were up y/y mainly due to the cost of the new headquarters and software.

Operating expense of $83 million consisted of $61 million for R&D and $21 million for general and administrative expense. Loss from operations was $10 million. Other income $4 million. Tax expense $1 million.

Q&A summary:

Should we expect continued dose response at 12 mtk for the AT program? We will talk about the data when it comes out on Friday.

Extra hepatic program? We will do a rollout in 2022. Will then give the therapeutic areas and cell types of interest, plus supporting preclinical data.

Debate re RNAi platforms broadly? Outlicensing? We think a lot about it, earlier we used collaborations to build our balance sheet. Now that is not such a priority. Also moving from liver-targetted to Galazy-plus. We get requests for partnering, RNAi has shown a strong profile. We plan to remain focused on our own pipeline.

A1AT dosing? Belcesiran 12 mtk cohort was about healthy volunteers, not go forward. Chronic doses need to be different than single dose. We are achieving the level of knockdown we target and expect the Phase 2 trial to be successful.

Hepatitis B, 2023 data timeline? Trials began in 2020, 48 week dosing, 24 week off drug, some cohorts could complete enrollment before the end of the year. It is up to Roche.

We have some hypotheses about PH2 and PH3, there may still be a role for nedosiran. We are committed to helping the patients. We are in the process of outlicensing nedosiran globally, not just for PH2 or PH3. The interest has been strong from multiple parties.

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