Analyst Conference Summary

biotechnology

conference date: August 6, 2020 @ 5:30 AM Pacific Time
for quarter ending: June 30, 2020 (second quarter, Q2)

Forward-looking statements

Overview:

Basic data (GAAP):

Revenue was $104.0 million, up 5% sequentially from $99.5 million, and up 133% from $44.7 million year-earlier.

Net income was negative $179.2 million, down sequentially from negative $178 million, and up from negative $219.5 million year-earlier.

Diluted EPS was negative $1.56, up sequentially from negative $1.62, and up from negative $2.02 year-earlier.

Guidance:

Increased 2020 revenue guidance to $280 to $300 million. Slightly decreased 2020 GAAP expenses to $1.13 to $1.225 billion, but non-GAAP expense steady at $1.0 to $1.075 billion.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "In the quarter, we also continued advancing our robust late-stage pipeline of investigational RNAi therapeutics, where, notably, we presented full results from the ILLUMINATE-A Phase 3 study and completed regulatory filings for lumasiran, and continued enrollment in our APOLLO-B and HELIOS-B Phase 3 studies of patisiran and vutrisiran, respectively. Further, a key business highlight in the second quarter was our completion of a landmark strategic financing collaboration with Blackstone, which we believe will secure our ability to achieve a self-sustainable financial profile without the need for future equity financing. These and other achievements position us to realize our Alnylam 2020 vision of building a multi-product, global biopharma company with a deep clinical pipeline to fuel continued growth and a robust, organic product engine to drive sustainable innovation and further value creation." Against most favored nation executive order idea, thinks insurance should prevent high out of pocket charges to patients.

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.

Givlaari (Givosiran) for AHP (actue hepatic porphyria) revenue in Q2 2020 was $11.0 million, up sequentially from $5.3 million. In Q1 2020 received approval in the EU.

Revenue from collaborators: $26.4 million from Genzyme/Sanofi.

Onpattro (patisiran) revenue was $66.5 million, down slightly sequentially from $66.7 million and up from $38.2 million year-earlier. Making progress ex-US with new launches or getting reimbursement. On August 6 Alnylam announced that it achieved agreement on pricing and reimbursement in France, completing patient access for Onpattro in all major European markets in under 2 years following approval

Cash and equivalents balance at the end of the quarter was $1.95 billion, up sequentially from $1.37 billion. No debt. Received $600 million from Blackstone for stock and from sale of future royalties.

Non-GAAP net income negative $191 million, down sequentially from negative $172 million, and up from negative $198 million year-earlier. EPS negative $1.67, down sequentially from negative $1.52, and up from negative $1.83 year-earlier.

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 results from the ILLUMINATE-A Phase 3 study of lumasiran at ERA-EDTA,, June 7, 2020 as a virtual event. Filed an NDA with the FDA, received an action date of December 3, 2020.

Inclisiran for hypercholesterolemia moved over to Novartis, which acquired The Medicines Company in January 2020. 2 Phase 3 results published in New England Journal of Medicine. NDA was submitted to the FDA and the EU.

In Q2 2020 filed a Clinical Trial Application for ALN-HSD, an investigational RNAi therapeutic targeting HSD17B13 in development for the treatment of nonalcoholic steatohepatitis (NASH). ALN-HSD is being advanced in collaboration with Regeneron.

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 $302 million consisted of: $20 million for cost of goods sold; $155 million for research and development; and $128 million for general and administrative expense. Operating loss $199 million. Interest & other income was $21 million. $1 million income tax benefit.

Q&A Summary:

The fact that healthcare providers themselves have had to get online has made them more receptive to virtual intorduction campaigns. We have had really good participation in virtual presentations, better than prior real-world local ones.

Haemophilia perspective? It is a multiproduct, multicompany market. Vitusiran fits in as the only once-monthly subcutaneous medicine with good resulting levels and safety. It could offer a very competitive profile. Gene therapy has benefits, but also questions. We eagerly await Phase 3 data early next year.

Regeneron option for APP? On track to file an IND in mid 2021. C5 program had already been opted into. They are also doing combination studies for complement mediated diseases.

In CEMEA region, home use doubled during the pandemic, in the U.S. went from 9% home infusion to about 20%. Most patients are managing without dose interuption. But we see them going back to infusion centers as the pandemic ebbs.

Givlaari experience, thoughts on lumasiran launch? Ultrarare disease PH1, underdiagnosis is well known. Playbook for introduction is well-developed. We have also gotten good at using virtual launch methods.

Givlaari start form trajectory? We are pleased with Q2 performance, despite pandemic. Q1 had the initial bolus. 25% of US patients are from outside the hub. We will not provide start form metrics because we are starting globally.

OpenIcon Alnylam page

OpenIcon Analyst Conference Summaries Main Page

Search

More Analyst Conference Pages: