AstraZeneca PLC (NASDAQ:AZN) Q2 2023 Earnings Call Transcript

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The possibility of curative treatment for rare genetic disease becomes achievable when combined with the Alexion rare disease expertise. With that, I’ll now hand over to Marc, who will cover the rare disease genomic strategy in more detail, along with rare disease highlights in the period. Please advance to the next slide.

Marc Dunoyer: Thank you, Mene. Can I see the next slide. In the first half, rare disease total revenue grew 12%, contributing $3.8 billion. Growth in the period was driven by increased demand and benefited from timing of tender market orders, slightly offset by the onetime pricing adjustment in the international region recognized in the second quarter of last year. Across the portfolio, our global patient numbers continue to grow. And notably, this is the first quarter Ultomiris patients exceeded those of Soliris. Ultomiris grew 60% in the second quarter driven by continued naive patient growth in generalized myasthenia gravis, new market launches and successful conversion from Soliris across the shared indications. As a consequence of this dynamic, Soliris declined 19%.

Thought I’m excited by our performance, as I mentioned in the first quarter, we expect some headwinds in the second half of the year. These include pricing pressure related to renegotiations as Ultomiris launches in large neurological indications and potential for Soliris biosimilar entry in Europe. Also, as a reminder, we benefited from tender market order timing in the second half of last year. Timing of these orders are variable throughout the course of the year, impacting growth versus prior periods. Beyond C5, both Strensiq and Koselugo grew 25% and 30%, respectively, reflecting underlying patient demand and expansion into new markets. Please advance to the next slide. During the quarter, ALXN2220 Phase I data were presented at the European Society of Cardiology in patients with transthyretin amyloid cardiomyopathy.

As a reminder, ATTR-CM is a progressive and fatal disease caused by misfolding transthyretin depositing in heart tissue. The safety and pharmacokinetic profiles of ALXN2220 were assessed and cardiac imaging studies were performed. As shown, the images detailed remarkable reduction in cardiac amyloid deposition over 4 and 12 months. The observations were supported by change in level of cardiac biomarkers as well as functional measures. ALXN2220 is the first and only medicine to clear amyloid deposition, and it has the potential to reverse the course of disease both as a complementary therapy with other modalities, but also as a monotherapy. We are excited by this Phase Ib data, and as previously shared, we plan to initiate a Phase III trial later this year.

Across AstraZeneca and Alexion, we are looking to transform the care of transthyretin amyloidosis through multiple therapy modalities. Our broader amyloidosis portfolio includes silencer eplontersen and stabilizer acoramidis for which we have rights in Japan. Recently, we saw positive 30 months Phase III data for acoramidis. We’ve demonstrated a 50% relative risk reduction in cardiovascular-related hospitalization building confidence in the meeting ability to stabilize disease progression. Eplontersen met its primary endpoint in regulatory ransthyretin-mediated amyloid polyneuropathy, and we have ongoing Phase III trials in ATTR-CM. We believe this portfolio of medicine this differentiating mechanism of action will address the full spectrum of disease severity.

Next slide, please. As you may have seen, we have done a series of small to medium-sized business development deals, expanding our technologies and platform in research. I wanted to take the opportunity to highlight some of these and affirm our long-term ambition to be an industry leader in genomic medicine. Approximately 80% of rare diseases are genetic, driven by inherited or acquired gene mutations. By leveraging AstraZeneca technologies in on-viral [ph] delivery systems and nucleases, such as CRISPR, LogicBio, GeneRide and sAAVy platforms, [Indiscernible] cargo for delivery to the central system or CNS, as well as our own operated platforms, we aim to address a number of genetic diseases across liver, kidney, heart, and muscle and CNS. The agreement announced this morning to acquire a portfolio of gene therapy programs for rare disease from Pfizer accelerates our timeframe to bring this potentially transformative and curative treatment to patients.

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