Analyst Conference Summary

biotechnology

conference date: August 8, 2019 @ 1:30 PM Pacific Time
for quarter ending: June 30, 2019 (second quarter, Q2)

Forward-looking statements

Overview: Clinical stage biotechnology company continues to develop its rna based therapies.

Basic data (GAAP):

Revenue was $5.7 million, up from $1.5 million year-earlier.

Net income was negative $23.8 million, up from negative $35.6 million year-earlier.

Diluted EPS was negative $0.35, up from negative $0.68 year-earlier.

Guidance:

Dicerna believes that its current cash, cash-equivalents, and held-to-maturity investments will be sufficient to fund the execution of its current clinical and operating plan beyond 2020.

Conference Highlights:

Douglas Fambrough, CEO of Dicerna said "Dicerna is dynamically growing as we move into pivotal development of our lead program in primary hyperoxaluria with an eye toward commercialization. Simultaneously, we are increasing clinical development and discovery efforts for our own proprietary programs and those in conjunction with our corporate collaborators while expanding the reach of our GalXC RNAi technology. I am particularly pleased with the strength and depth of the new leaders who have joined Dicerna to drive these efforts." Strategy is to go deep on good commercial opportunities.

Cash and equivalents balance ended at $345 million

In Q2 Dicerna initiated dosing in PHYOX3, a long-term, multi-dose, open label, roll-over extension initially for our Phase 1 study for DCR-PHXC for the treatment of PH (primary hyperoxaluria). Received Breakthrough Therapy Designation for DCR-PHXC for the treatment of primary hyperoxaluria type 1. Initiated PHYOX2, a multi-dose, double-blind, randomized, placebo-controlled pivotal trial of DCR-PHXC, which is being investigated for the treatment of all forms of PH.

The first patient was dosed in the Phase 1 clinical trial of DCR-HBVS for the treatment of patients with chronic hepatitis B virus (HBV) infection. Looking for a partner for development. Believes cures will require multiple lines of therapy.

Dicerna 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.

Initiation of a multi-center Phase 1/2 trial of DCR-A1AT is expected in Q3 2019. The proposed parallel-group, placebo-controlled study will evaluate DCR-A1AT in adult healthy volunteers and patients with A1AT deficiency-associated liver disease

Dicerna expects its overall R&D expense to continue to increase for the foreseeable future.

Expects to expand its internal pipeline in the coming quarters. Believe GalXC platform can be extended beyond the liver.

Q&A summary:

A1AT mutation variance by country? It is mainly found in caucasians. Ireland, Germany, Spain, Italy have high prevalence.

A1AT trials, why starting outside the US? In EU because their regulators are allowing us to run the cohorts in parallel, like safety hurdles. It compresses the timeline. We had an extended dialog with the FDA, the feedback had us make some changes, so we believe we are now in alignment. We believe we have filled all requivements to open a trial in the U.S.

Hep B data target? The n is small, we want to see a substantial effect on the antigen. We are looking for a greater than one log effect in the initial cohort.

PH types and oxalate levels? Based on preclinical and Phase 1 data, both PH1 and PH2 patients can be normalized. Also likely in PH3, but no data for it.

Expects to see less s antigen suppression moving into humans with the Hep B therapy, much like others who have tried this, but the data will tell. Dicerna is just looking for a nice, solid suppression signal.

We will complete cohort 1 by the end of the year, just 4 of 6 would be on drug. We do not have a data release timeline.

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