Analyst Conference Summary

biotechnology

conference date: November 3, 2022, @ 5:00 AM Pacific Time
for quarter ending: September 30, 2022 (Q3, third quarter 2022)

Forward-looking statements

Overview: Revenue from Pyrukynd is not ramping much yet.

Basic data (GAAP):

Revenue was $3.5 million, down sequentially from $5.6 million, and up from $0 million year-earlier.

Net income was negative $82 million, up sequentially from negative $91.8 million, and up from negative $89 million year-earlier.

EPS (diluted GAAP) was negative $1.49, up sequentially from negative $1.68, and up from negative $1.56 year-earlier.

Guidance:

Believes has cash to reach cash-flow positive.

Conference Highlights:

Brian Goff, CEO of Agios said "Agios is on the cusp of changing the treatment landscape for people with rare and genetically defined diseases, with the potential of Pyrukynd to make a positive impact across multiple underserved diseases. I joined the company in August because of its differentiated portfolio, top-notch team and genuine dedication to patients, and my excitement for the future of Agios has only grown over the past few months. We are continuing to build our capabilities and connections in rare and genetically defined diseases through our U.S. launch of Pyrukynd, and we expect our learnings to support anticipated future expansion in related diseases where development efforts are ongoing. We will build on the tremendous accomplishments we’ve achieved in 2022 as we close out the year with a focus on continuing to execute the launch, enrolling our thalassemia and sickle cell disease pivotal trials and securing the approval of Pyrukynd for PK deficiency in the EU and Great Britain. I am honored to lead this team as we together drive long-term growth and value for patients, shareholders and all our stakeholders." Focus is on moving most promising programs forward while in-licensing potential therapies. Label expansion will be into larger indications with more revenue potential than PK deficiency.

On October 27, 2022, Agios announced that Sagard Healthcare bought Agios's rights to 5% royalties on U.S. sales of Tibsovo for a one-time payment of $131.8 million.

Pyrukynd revenue was $3.5 million. Second full quarter since FDA approval. Milestone revenue (from former oncology business) was $0 million. 56 patients on Pyrukynd and 84 have completed prescription enrollment forms. Discontinuations low so far, but label says check for efficacy at 6 months. More that 5,300 Anemia ID kits have been ordered and about 25% have been completed. So far positive test rates are about 40% for pediatric patients and 60% for adults.

Brian Goff became CEO on August 8, 2022. Jackie Fouse became Chair of the Board.

Over 20 Agios abstracts have been accepted for presentation at ASH; they go online today.

Pyrukynd (Mitapivat) in adults with PK deficiency, for treatment of hemolytic anemia, received FDA marketing authorization on February 17, 2022. Had previously launched myAgios patient support services for people living with PK deficiency and their caregivers, providing tailored support, educational resources and opportunities to connect with other patients and caregivers in the community.

Continuing to work on payer reimbursement. MyAgios program educates patients online. Will take some time to get reimbursements, perhaps a full year. Believes there could be 4,000 on-label patients in the U.S., which could lead to annual revenue of $200 to $225 million.

In April 2022 results from the Activate Phase 3 clinical study evaluating Pyrukynd in adults with PK deficiency who do not receive regular transfusions were publishing in the New England Journal of Medicine. In Q2 2022 Agios initiated Phase 3 studies of Pyrukynd in pediatric patients with PK deficiency who are not regularly transfused and who are regularly transfused, respectively. In Q2 2022 clinical and translational data were presented at EHA, including new data supporting the potential benefits of Pyrukynd treatment in adults with PK deficiency.

An EMA Pyrukynd decision for PK definiency is expected before the end of 2022. Received a positive CHMP opinion. Also applying in Britain.

Now working on extensive label expansion. In Q1 2022 the first patients were dosed in all three pivotal Phase 3 trials for not regularly transfused and regularly transfused adults with thalassemia, and for sickle cell disease.

In Q3 an AG-946 Phase 2a study in MDS is expected to start by year-end 2022; this will be open label. Phase 2b will be double-blind.

The BCAT2 preclinical program targets acidemias. Also working on a preclinical phenylketonuria (PKU) agent.

Cash (including equivalents & securities) ended at $1.03 billion, down sequentially from $1.1 billion. No debt.

GAAP operating expenses were $ million, consisting of: Cost of Sales <$1 million; $65 million for R&D and $29 million for SG&A. Loss from operations was $91 million. Interest income was $4 million. Other income $6 million.

Q&A summary:

Prescription conversions, ramp guidance? Prescription enrollment gets them into Agios support that guides them through the process. It takes 4 to 6 weeks to fill a new presciption. Too early to provide guidance on ramp.

Reauthorization criteria? Working on specifics, will see early patients hit six months this quarter. Looking at blood parameters, so far no hurdles from payers.

Anemia ID is not specific to PKD. Drives physician education. Drives diagnostic triage process.

Pyrukynd pricing in Europe? Expecting final approval, focus is on ensuring access. We see most revenue from the US. In EU will work on commercialization, but is mainly the pathway to label expansion. Looking for partners.

MDS competition? Luspatercept (Reblozyl) should have no impact on our clinical development plan. We have an oral formulation and a very different mechanism of action. Focus is on lower-risk MDS.

Genotypes? We have see characteristics across the board.

In clinical trial response rate was 40% to 45% at 6 months.

EPO for MDS? We are only enrolling naive patients at this point.

Thalasemia pricing, sickle cell? We will wait to see the data for pricing decisions.

Business development? We would like to diversify our pipeline. We have enlarged our BD team. Sweet spot is rare diseases.

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