Analyst Conference Summary

biotechnology

conference date: May 6, 2020 @ 5:30 AM Pacific Time
for quarter ending: March 31, 2020 (first quarter, Q1)

Forward-looking statements

Overview: Ramped revenue but lowered guidance.

Basic data (GAAP):

Revenue was $99.5 million, up 39% sequentially from $71.7 million, and up 199% from $33.3 million year-earlier.

Net income was negative $178 million, up sequentially from negative $278 million, and up from negative $182 million year-earlier.

Diluted EPS was negative $1.62, up sequentially from negative $2.47, and up from negative $1.73 year-earlier.

Guidance:

Lowered 2020 guidance to revenue between $270 and $300 million. That does not include potential Givlaari revenue.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "To that end, we are very pleased with our first full quarter as a multi-product, global commercial company where we delivered continued and steady worldwide growth of Onpattro and impressive early demand for Givlaari in the U.S." and of course talked about the pandemic. The guidance reduction was an arbitray 5%

In 2020 Alnylam entered into a broad strategic financing collaboration with Blackstone under which Alnylam will receive up to $2 billion that is expected to enable Alnylam to achieve a self-sustainable financial profile without the need for future equity financing.

The FDA approved givosiran for AHP (actue hepatic porphyria) at the end of 2019. Revenue in Q1 2020 was $5.3 million. In Q1 2020 received approval of Givlaari (givosiran) in the EU for acute hepatic porphyria (AHP) in adults and adolescents, with patients now on commercial product.

Revenue from collaborators: $27.5 million from Genzyme/Sanofi.

Onpattro (patisiran) revenue was $66.7 million, up 20% sequentially from $55.8 million and up from $26.3 million year-earlier." Making progress ex-US with new launches or getting reimbursement.

Cash and equivalents balance at the end of the quarter was $1.37 billion, down sequentially from $1.55 billion. Cash at the end of Q1 excludes $600.0 million from the Blackstone strategic financing collaboration, which closed in Q2. No debt.

Non-GAAP net income negative $172 million, up sequentially from negative $221 million, and down from negative $150 million year-earlier. EPS negative $1.52, up sequentially from negative $1.98, and down from negative $1.42 year-earlier.

Alnylam continued a Phase 1 trial for vutrisiran (ALN-TTRsc02), an ESC-GalNAc-siRNA conjugate targeting TTR for the treatment of ATTR amyloidosis, which is expected to enable a once-quarterly subcutaneous dosing regimen. A Phase 3 trial began in late 2018. Announced plans to initiate an additional Phase 3 study, HELIOS-B, in hereditary and wild-type ATTR amyloidosis with cardiomyopathy in late 2019.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study continued. Additional Phase 2 data was presented in Q2 2019. Sanofi Genzyme is a partner in the program.

Vutrisiran (ALN-TTRsc02) subcutaneously administered ATTR treatement completed enrollment of the HELIOS-A Phase 3 study. Continued the HELIOS-B Phase 3 study in patients with hereditary and wild-type ATTR amyloidosis with cardiomyopathy.

Lumasiran for PH1 (primary hyperoxaluria type 1) Phase 3 trial reported positive topline results in late 2019. Full results expected March 2020. An NDA planned for in 2020. Will present full results from the ILLUMINATE-A Phase 3 study of lumasiran at ERA-EDTA,, June 7, 2020 as a virtual event. Expects report topline results from the ILLUMINATE-B Phase 3 study in mid-2020.

Inclisiran for hypercholesterolemia moved over to Novartis, which acquired The Medicines Company in January 2020. 2 Phase 3 studies showed positive results, so an NDA was submitted to the FDA and the EU.

ALN-GO1 for primary hyperoxaluria type 1 (PH1) Phase 1/2 study continued.

ALF-F12 targeting factor XII is now in development for the treatment of hereditary angioedema and for thromboprophylaxis.

Plans to file a Clinical Trial Application for ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 for the treatment of non-alcoholic steatohepatitis (NASH), in collaboration with Regeneron, by mid-2020.

Vir Biotechnology, presented positive interim data from the ongoing Phase 2 trial in patients and results from the Phase 1 trial in healthy volunteers of ALN-HBV02 (VIR-2218), an investigational RNAi therapeutic for the treatment of chronic hepatitis B virus (HBV) infection.

In Q1 2020 expanded collaboration with Vir to include the development and commercialization of RNAi therapeutics targeting SARS-CoV-2 and up to three additional targets focused on host factors for SARS-CoV-2.

In Q1 2020 Alnylam agreed with Dicerna to develop and commercialize investigational RNAi therapeutics for the treatment of alpha-1 antitrypsin (A1AT) deficiency-associated liver disease, and completed a non-exclusive cross-licensing agreement with Dicerna for Alnylam's lumasiran and Dicerna's nedosiran investigational programs for the treatment of primary hyperoxaluria.

In May 2020 announced positive initial topline results from the ongoing Phase 1 study (N=48) of ALN-AGT in hypertension, providing initial human proof of concept with over 90 percent mean knockdown of angiotensinogen (AGT) and an over 10 mmHg reduction of mean 24-hour systolic blood pressure at week 8 relative to placebo, with a durability that supports once quarterly or less frequent dose administration. In addition, ALN-AGT administration showed an encouraging safety and tolerability profile including no drug-related serious adverse events.

See also Alnylam pipeline.

Operating expenses of $310 million consisted of: $13 million for cost of goods sold; $170 million for research and development; and $127 million for general and administrative expense. Operating loss $210 million. Interest & other income was $29 million. $0.6 million income tax benefit.

Q&A Summary:

In guidance, new patients v. existing? We expect impact in Q2, then recovery beginning in Q3. Each country is different. Patients transition from academic centers towards home infusion. In U.S. had home use move from 9% to 16% in April.

Givlaari patients? All patients outside our clinical studies and EAP patients. So brand new patients.

Hypertension competitive landscape? ALN-AGT. The drop we have shown is impressive compared to most drugs. Another advantage is the long time between treatments, 3 or 6 months apart. Also our reduction is steadier than pills that give a seesaw effect.

ALN-AGT safety? Patients had to come off prior drugs to enter the study. Safety profile very encouraging, no serious adverse events. Our drug acts on liver alone, sparing the kidney.

In Europe they tend to move patients quickly from EAP to paid use. They are seeing patients who did not benefit enough from stabilizers.

We do expect some enrollment slowdown in Apollo B, but overall the data integrity and timeline should be good.

We believe stabilizer plus Onpattro is for patients who have both cardioneuropathy and polyneuropathy, especially in the US. In rest of world we are seeing patients switched off of stabilizer.

We have not yet taken debt down, expect to take $200 million down at the end of this year (using the Blackstone funds).

OpenIcon Alnylam page

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: