Analyst Conference Summary

biotechnology

conference date: May 6, 2021 @ 1:30 PM Pacific Time
for quarter ending: March 31, 2020 (first quarter, Q1)

Forward-looking statements

Overview: First NDA submission planned for later in 2021.

Basic data (GAAP):

Revenue was $47.6 million, up sequentially from $na million, and up from $34.0 million year-earlier.

Net income was negative $30.0 million, down sequentially from negative $na million, and down from negative $22.5 million year-earlier.

Diluted EPS was negative $0.39, up sequentially from negative $na, and down from negative $0.31 year-earlier.

Guidance:

Cash should last to 2024.

Conference Highlights:

Douglas Fambrough, CEO of Dicerna said "We continue to execute across our expanding clinical pipeline, particularly across our Phyox development program for nedosiran, where we anticipate the data from our Phyox2 pivotal trial in patients with PH1 or PH2 sometime mid-year. We also recently dosed our first patient in Phyox7, a multidose trial in patients with PH1 or PH2 who have severe renal impairment. Although enrollment in our Phyox4 single-dose trial in patients with PH3 has been slower than anticipated in the current COVID-19 environment and will push our NDA submission from late in the third quarter to sometime in the fourth quarter, we expect that nedosiran's profile may offer compelling advantages in PH1 and be a significant, and the only, therapy for people with PH2 or PH3 who currently have no treatment options to impact disease progression."

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.

Belceiran, the GalXC RNAi candidate for the treatment of alpha-1 antitrypsin deficiency-associated liver disease (AAT-LD), should report Phase 1 data from healthy volunteers in mid-2021.

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.

In March 2021 Dicerna presented data from preclinical studies of GalXC-Plus RNAi technology demonstrating its potential to deliver deep and sustained mRNA knockdown against prespecified gene targets across the CNS and to oligodendrocytes, astrocytes and neurons, at the sixth annual Oligonucleotide and Precision Therapeutics Virtual Congress.

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. There are also 20 research-stage programs in development.

Cash and equivalents balance ended at $545 million, down sequentially from $569 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 and should read out data next year. An NDA could be submitted in Q4 2021. Has Breakthrough Therapy Designation for PH1. 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. 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.

RG6346 for HBV reported positive interim Phase 1 data in Q4 2020. Roche initiated RG6346 in a Phase 2 combination trial in March 2021, earning Dicern a $25 million milestone payment. That is part of a larger trial trying multiple combinations.

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.

Lilly expected to file the IND for GalXC RNAi candidate LY3819469 targeting the LPA gene in the second quarter of 2021

Operating expense of $77 million consisted of $56 million for R&D and $21 million for general and administrative expense. Loss from operations was $29 million. Other expense $1 million.

Q&A summary:

PH filing delay to include type 3? Primary value is the ability to treat all three types. The delay in enrollment is not bad, so we will do an integrated file. We are looking at the size of the PH3 market, there does appear to be a group exibiting symptoms, perhaps 20% of PH3 patients show symptoms.

A1AT? Topline data will show topline protein suppression in healthy volunteers, plus safety data. We have plans for Phase 2 based on this data.

PHYOX7 and 8 trial populations? Under 6 years of age, do live a while, patients are across an age spectrum. The easiest ones to find are the infants. There are many silent sufferers.

Partnered program timelines? We do not know the Lilly ANG3 timeline.

Key nedosiran metrics v. the approved RNAi drug? The key is the ability to dose all 3 types. Type 2 and 3 are not addressed by lumiseran (Alnylam's Oxlumo). We hope to report a higher level of oxilate normalization in Type 1 patients. Also the prefilled syringe for self administration should be attractive.

We are in a dialog with Lilly about a couple of orphan indications. But CNS is a crowded space. We expect to advance mutliple GalXC+ programs, not necessarily orphan ones. There are specific reasons in specific indications that could influence whether a small molecule or CRISPR could compete.

PH3 small number of subjects, could that be a problem? Potential ex-US partners understand lumiseran launch, and nedosiran potential. PHYOX4 data is in the context of the similar nature of oxalate. It is a double blind placebo controlled study. The PH3 part is less agreed to by the FDA at this point.

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