Analyst Conference Summary

biotechnology

conference date: November 5, 2020 @ 1:30 PM Pacific Time
for quarter ending: September 30, 2020 (third quarter, Q3)

Forward-looking statements

Overview: Nedosiran pivotal trial enrollment to complete before year end.

Basic data (GAAP):

Revenue was $49 million, up sequentially from $40 million, and up from $8 million year-earlier.

Net income was negative $22 million, up sequentially from negative $32 million, and up from negative $31 million year-earlier.

Diluted EPS was negative $0.29, up sequentially from negative $0.43, and up from negative $0.45 year-earlier.

Guidance:

Cash and achievable milestones should allow operations through 2023.

Conference Highlights:

Douglas Fambrough, CEO of Dicerna said "We remain on track to complete enrollment of our PHYOX2 pivotal trial supporting nedosiran, our wholly owned product candidate for patients with primary hyperoxaluria, or PH, in the fourth quarter of 2020. Recent interim data from our ongoing PHYOX3 open-label trial of nedosiran instill great confidence in our organization that nedosiran may have a best-in-class profile as the first potential treatment for all known subtypes of PH and position us well as we transition into a fully integrated biopharmaceutical company. Further supporting this transition is over $500 million in upfront payments secured through collaboration agreements over the last two years, which also carry tremendous future value through a stream of potential near- and long-term milestone payments, the first of which will be from our collaboration with Lilly in the fourth quarter of this year. Over the balance of 2020 and 2021, we anticipate receiving over $100 million in cash from our collaborations and look forward to updating investors as these partnerships mature and our internal pipeline expands."

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 $610 million, down sequentially from $669 million.

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. In October, released positive updated interim data as of Sept. 30, 2020 from its ongoing open-label extension study of nedosiran for the treatment of PH. Could file an NDA in Q3 2021.

In August, Dicerna announced positive preclinical data demonstrating expansion of its technology beyond its hepatocyte-focused GalXC RNAi technology to central nervous system (CNS), skeletal muscle and adipose tissues. The data showed consistent and durable CNS-wide target messenger RNA (mRNA) knockdown using novel constructs regardless of route of administration and reduction in target mRNA in skeletal muscle and adipose tissue.

In August, Dicerna announced positive interim data as of June 25, 2020 for RG6346, an investigational candidate for the treatment of chronic hepatitis B virus (HBV) infection being developed in collaboration with Roche. The data demonstrated a strong and durable reduction in hepatitis B surface antigen (HBsAg). Dicerna received $200 million up front in January 2020, and could receive up to $1.47 billion in potential milestone payments.

Alnylam partnered DCR-A1AT/ALN-AAT02 program selection for advancement into Phase 2 expected in the first quarter 2021. Alnylam has an option to pick up ex-US commercial rights.

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.

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. Alynlam will have the right to opt-in to overseas rights. Royalties would depend on the candidate chosen and geography.

Operating expense of $72 million consisted of $54 million for R&D and $71 million for general and administrative expense. Loss from operations was $23 million. Interest income $1 million.

Q&A summary:

HBV data, patients, antigen reduction? We anticipate we will have data for all patients through day 112 for the next update.

HBV dosing frequency? The long duration should have benefit for functional cure rates. It works in the animal models, and the early data indicates it carries over to humans.

A1AT choice? There is no bad choice, but it only makes sense to take one forward. We will look at all the preclinical and clinical details. Differentiation would be in the details.

A1AT choice, what data would be released then? We will share the human healthy volunteer data from the molecule we choose. Probably not preclinical data at that point, or how we made the choice.

Will PH3 be part of the first filing, or be its own filing? PH1, PH2, we know the markers. PH3 is less well understood, so we had to launch a natural history study. We believe we can show nedosiran works for PH3, with accelerated approval.

We are not releasing our non-liver targets. But in our first neuro program, that will be in the orphan space.

Lung function markers for A1AT? We know the target we are silencing, when not present, causes lung problems. The KOL general view is a late in life treatment for liver is unlikely to cause a lung problem. We have not finalized on a late-stage protocol for lung function.

Arrowhead data in A1AT? What we know so far, it looks good, we would expect it to be an effective mechanism. The Vertex issues are concerning, but not much is know about their liver toxicity signal. Neither changes our strategy.

We are in active discussions about an ex-US partner for PH.

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