Analyst Conference Summary

biotechnology

conference date: August 6, 2020 @ 7:00 AM Pacific Time
for quarter ending: June 30, 2019 (second quarter, Q2)

Forward-looking statements

Overview: Heavy R&D spend, more good early-stage data.

Note that though simultaneous to earnings release, this 2 hour conference was billed as an R&D; Day, so presentation and questions were mainly about RG6346 and Nedosiran.

Basic data (GAAP):

Revenue was $40 million, up sequentially from $34 million, and up from $6 million year-earlier.

Net income was negative $32 million, down sequentially from negative $25 million, and down from negative $24 million year-earlier.

Diluted EPS was negative $0.43, down sequentially from negative $0.31, and down from negative $0.35 year-earlier.

Guidance:

Cash, cash equivalents and held-to-maturity investments will be sufficient to fund the clinical and operating plan into 2023.

Conference Highlights:

This was billed as an R&D; conference. Q2 results are from the press release.

Douglas Fambrough, CEO of Dicerna said "In addition to our financial results, today we also updated our guidance for completion of enrollment of our pivotal PHYOX2 trial of nedosiran in patients with primary hyperoxaluria types 1 or 2, to the fourth quarter of 2020. We look forward to discussing our clinical progress in detail at our R&D Day event today, which will include interim positive data from our ongoing PHYOX3 multidose trial of nedosiran, Phase 1 proof-of-concept data from our ongoing trial of RG6346 for the treatment of chronic hepatitis B virus infection, and our first public presentation of preclinical data demonstrating delivery of our proprietary technology to multiple tissues outside the liver, all of which reinforce Dicerna's position as a best-in-class company in RNAi therapeutics."

Cash and equivalents balance ended at $ million, up sequentially from $707 million.

On May 10, 2020 Dicerna announced Roche has formally nominated the first selected target and thus has initiated the research and development portion of its agreement.

In April 2020, Dicerna and Alnylam completed a cross-license agreement for Alnylam's lumasiran and Dicerna's nedosiran for the treatment of PH (type 1 or 2). This provides for Alnylam to pay mid- to high-single-digit royalties to Dicerna based on global net sales of lumasiran and for Dicerna to pay low-single-digit royalties to Alnylam on global net sales of nedosiran. The two companies will also cooperate in developing the A1AT program, with Dicerna able to choose between its own product and ALN-AAT02. Alynlam will have the right to opt-in to overseas rights. Royalties would depend on the candidate chosen and geography.

The Anylam agreement eliminates the portential for costly IP litigation.

The PHYOX2, a long-term, double blind, study of nedosiran (DCR-PHXC) for the treatment of PH (primary hyperoxaluria) types 1 and 2 should complete enrollment in Q4 2020. Has Breakthrough Therapy Designation for DCR-PHXC for the treatment of primary hyperoxaluria type 1. PHYOX3 interim positive data was updated on August 7, 2020. Considering partnering outside the US for commercialization. In June the FDA granted rare pediatric disease designation.

The multi-center Phase 1/2 trial of DCR-A1AT began Q3 2019, with first dosing expected in Q4 2019. The study will evaluate DCR-A1AT in adult healthy volunteers and patients with A1AT deficiency-associated liver disease. Dicerna in Q2 2019 submitted a clinical trial application to the Swedish Medical Products Agency for DCR-A1AT for the treatment of patients with alpha-1 antitrypsin deficiency-associated (A1AT) liver disease. In December 2019 receiped oprhan drug designation in the EU. In Q1 2020 received orphan drug designation from the FDA.

During the first quarter of 2020, Eli Lilly selected LY3819469, a GalXC molecule for the second collaboration target in cardiometabolic disease, for advancement into preclinical development.

In collaboration with Roche, RG6346 for chronic hepatitis B virus (HBV) infection using GalXC RNAi platform technology, Phase 1 enrollmenbt was completed in June 2020. Data from group C was positive, announced 8/7/2020. Roche will be responsible for initiating a Phase 2 study. Dicerna received $200 million up front in January 2020, and could receive up to $1.47 billion in potential milestone payments.

In late April 2020, the Scientific Review Committee for the DCR-A1AT Phase 1/2 trial confirmed that the study could continue, and the Company began enrolling the following dosing cohort in May, which has since been completed. The Company is currently targeting program selection and potential initiation of patient dosing in the first quarter of 2021.

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.

Cash and equivalents were $669 million.

Operating expense of $74 million consisted of $53 million for R&D and $21 million for general and administrative expense. Loss from operations was $33 million. Interest income $2 million.

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