Analyst Conference Summary

biotechnology

conference date: Februry 27, 2020 @ 1:30 PM Pacific Time
for quarter ending: December 30, 2019 (fourth quarter, Q4)

Forward-looking statements

Overview: Clinical stage biotechnology company continues to develop its RNA based therapies. In January 2020 received $375 million in upfront payments from Roche and Novo Nordisk collaborations.

Basic data (GAAP):

Revenue was $7.1 million, down sequentially from $8.0 million, and up from $1.5 million year-earlier.

Net income was negative $39.7 million, down sequentially from negative $30.8 million, and down from negative $18.6 million year-earlier.

Diluted EPS was negative $0.58, down sequentially from negative $0.45, and down from negative $0.29 year-earlier.

Guidance:

Believes cash should suffice until 2023. Believe expenses will increase significantly for the foreseeable future.

Conference Highlights:

Douglas Fambrough, CEO of Dicerna said "In 2019, within our core clinical pipeline, we initiated pivotal development of nedosiran, our lead product candidate for primary hyperoxaluria types 1, 2 and 3 and initiated patient dosing of DCR-HBVS, now referred to as RG6346, observing multiple patients with greater than or equal to one log10 IU/ml HBsAG reduction in this ongoing, blinded study. We are encouraged to now have representation from both Groups B and C of our Phase 1 study of RG6346 in the extended follow-up phase. We also began clinical development of DCR-A1AT, all while progressing and expanding programs under our existing collaboration agreements. In 2019, we also established new, high-value strategic relationships with Roche and Novo Nordisk."

Cash and equivalents balance ended at $349 million, up sequentially from $313 million. Received $375 milion in January from Novo Nordisk and Roche.

In Q4 2019 Dicerna made a collaboration and licensing agreement with Roche to develop therapies for chronic hepatitis B virus (HBV) infection using GalXC RNAi platform technology, including RG6346 (was DCR-HBVS), which is in Phase 1 clinical development. The agreement also includes the discovery and development of therapies targeting human genes associated with HBV infection, or additional targets within the HBV genome, using the technology platforms of both companies. Dicerna received $200 million up front in January 2020, and could receive up to $1.47 billion in potential milestone payments.

In early Q4 Dicerna initiated dosing in 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 2020. Has Breakthrough Therapy Designation for DCR-PHXC for the treatment of primary hyperoxaluria type 1. First multi-dose data from PHYOX3 open-label clinical trial will be presented at OxalEurope International Congress, March 31, 2020. Considering partnering outside the US for commercialization.

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 December 2019, Alexion exercised its options for exclusive rights to two additional targets within the complement pathway, expanding the existing research collaboration and license agreement to now encompass four targets. In connection with the option exercise, Alexion paid Dicerna a total of $20 million, or $10 million in option exercise fees per additional new target.

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

Expects and IND in partnership with Lilly later this year.

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

Operating expense of $48 million consisted of $35 million for R&D and $14 million for general and administrative expense. Loss from operations was $41 million. Interest income $1.5 million.

Q&A summary:

PH3 dialog with FDA? Breakthrough designation for PH1 allows for continuing discussions on PH2 and PH3. Rare, PH3 has only been genetically confirmed 10 years, so less data available. Working with FDA towards a regulatory pathway.

Commercial buildout? First phase is bringing in leadership, which we have completed. Phase 2 is to begin communications with physicians and payers, which will to into 2021. Then the sales force, ready to go if we get an FDA approval, perhaps the second half of 2021.

BI collaboration? Going fine, shifted to second target, made an option payment for it, NASH target.

Combination nature of HPV therapy does introduce some complexity into our collaboration deal. We were in a competitive process. We are impressed by the Roche pipeline. The generic component can't be seen as bringing significant value to the combination, and the contract takes that into account.

PHYOX3 low end of range? Primary endpoint is decline in renal function. We do continue to measure oxalate in the urine. For hyperhydration, the protocol is to wean off the hydration therapy, when they reach near-normal concentration.

HBV patient statification? Groups will be distinct. One group similar to what other companies use. Group D will be single dose monotherapy. Most HBV patients have not been diagnosed, so would be like Group D.

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