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| Statement |
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| But we are very excited, most importantly, with the fact that as we advance our pipeline and as we've already noted, we have two back-to-back launch potentials with thalassemia next year followed by sickle cell disease in 2026 |
| We're well positioned with the differentiated mechanism of action that improves red blood cell health beyond hemoglobin increase and allows us to pursue large commercial opportunities with substantial value and the potential for two additional first-in-class and best-in-class indication for PYRUKYND as we build a multibillion dollar franchise in PK activation |
| Driven by this goal, we continue to generate consistent and compelling data across our industry-leading PK Activator franchise |
| I think we're inspired by the potential |
| And we're always very grateful to be able to have those conversations because I do think incorporating feedback from multiple stakeholders always leads to better design choices |
| We really saw an improvement on the FACIT-Fatigue there as well, which we believe is extremely meaningful because now we are adding to the hemoglobin plus story here |
| The things that we've observed in PKD now also have been observed in the non-transfusion dependent thalassemia population, and that continues to add to this consistent and compelling data story that we are continuing to generate |
| So we definitely remain excited about the opportunity in PKD, and we continue to expect those peak sales at $200 million to $225 million for the US |
| Together, the consistency of data generated across the mitapivat development program bolsters our conviction in the probability of success of our ongoing studies, including two additional Phase 3 readouts we expect by the end of this year, and this data highlights the potential of our PK activators to transform the course of multiple hematologic diseases |
| Importantly, we believe our PK activation pipeline is well positioned with multiple near-term catalysts to become a multi-billion dollar franchise and deliver significant value |
| Overall, I'm very proud of the tremendous progress made by our R&D organization in 2023 and look forward to continuing this momentum in 2024 |
| As we move toward additional potential value creating milestones in the near term, I am confident that our strong balance sheet will enable us to execute from a position of strength as we continue to pursue ways to create shareholder value |
| But the way PYRUKYND is performing is what we believe really puts us in a position of strength as we approach ENERGIZE, sorry, keep seeing ENERGIZE, as we approach thalassemia as well as sickle cell beyond that |
| And we have been delighted by the enthusiasm of the investigators |
| Led by mitapivat, the industry's most advanced PK activator, now with over eight years of clinical experience, the consistent and compelling data we have generated to date in PK deficiency, thalassemia and sickle cell disease to continue to de-risk our ongoing development program and highlight the potential for this molecule to transform patient's function and quality of life |
| We are also enthusiastic about the potential for the rest of our growing pipeline, and we are pleased to note that we remain on track to deliver on our milestones, including, enrolling the first patients in the Phase 2b trial for AG-946 in lower risk MDS, and for the Phase 1 trial for AG-181, the compounds named for our PAH stabilizer for phenylketonuria |
| And with seamless cross-functional collaboration of the Agios team, we made remarkable progress advancing this mission in 2023 |
| As you heard today, our team has great conviction in our potential to deliver transformative new therapies to patients and significant long-term value to shareholders |
| And that is allowing us to enter into very compelling commercial opportunities |
| And we believe firmly in mitapivat’s potential to deliver a best-in-class option for patients suffering from this devastating disease |
| We're increasingly convinced that the benefits on making the red blood cells healthier really position it as such |
| We were therefore very pleased to be able to announce positive results from the ENERGIZE study |
| I feel very good about the work the team has done and that'll be the next step |
| And in the case of low risk MDS, as I think folks know, we just reported out last year very encouraging proof-of-concept from our Phase 2a study |
| We believe that we'll be very well positioned with PYRUKYND to provide a treatment option, which will bring benefits to physicians, patients, and ultimately, payers as well by improving hemoglobin, reducing VOCs, and ultimately improving the way patients feel and function |
| This is therefore the first drug that not only improves hemoglobin but actually makes people with thalassemia feel better, consistent with what we observe in patients with PKD and what we hope to be able to deliver for patients with sickle cell disease as well |
| Similar to sickle cell disease, I think the thalassemia launch will be meaningfully differentiated in terms of market characteristics compared to PKD and that will position us well for adoption of PYRUKYND, assuming approval in 2025, including again, these patients are diagnosed, it's 6, 000 diagnosed patients in the US with thalassemia, and well-established ICD 10 own, a stronger concentration of the prescribed base and all of these elements gives us confidence on our ability to commercialize the product and drive adoption at launch |
| Beyond the excitement we have for the ENERGIZE data itself, the readout of the ENERGIZE trial also gives us further confidence towards the readout of ENERGIZE-T |
| Given this market dynamic and PYRUKYND target product profile, we believe we are well positioned to provide a potential foundational treatment option for patients with thalassemia regardless of subtype |
| So we are very excited about that opportunity to come |
| Statement |
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| Q4 2023 net PYRUKYND revenue was $7.1 million, a 4% reduction compared to the third quarter |
| It's a challenge |
| These patients suffer from a poor quality of life, a high rate of serious morbidities, including thrombosis and premature death |
| Based on our learnings to date, given the ultra-rare nature of the disease and the long lead times associated with initiating patients on therapy, we continue to expect slow and steady growth and quarter-to-quarter variability in 2024, similar to what we saw in 2023 |
| We think it's going to be slow and steady, continuing to see the trends we've seen in 2023 for the next few years |
| So slow and steady is the right phrase |
| SG&A expenses were $35 million for the fourth quarter and $120 million for the full year 2023, a decrease of $2 million compared to the full year 2022 |
| As we continue to stack successive positive data readouts for mitapivat, we are only growing more confident in the probability of success ahead |
| Patients endure high rates of morbidities and increase complications as they age |
| And so we'll take the slow and steady path and we're going to continue to expect that going forward |
| As a reminder, we anticipate low levels of inventory at any given time given our limited distribution network, which consists of one specialty pharmacy and one specialty distributor |
| We've gotten a few questions from investors around the sort of the transition from the Phase 2 data to the Phase 3 data |
| In regards to the waning, we don't see waning of our hemoglobin response |
| Despite not requiring regular transfusions, NTD-thalassemia patients experience significant impact on quality of life, a wide range of serious morbidities, and an elevated risk of premature death |
| But it's more than that |
| At the moment, the adult patient population with sickle cell disease has very limited treatment options available |
| Actual events and results could differ materially from those expressed or implied by any forward-looking statements as a result of various risks, uncertainties and other factors, including those set forth in our most recent filings with the SEC and any other future filings that we may make with the SEC |
| Brian Goff Yes, and Chris, I mean, that last point is the one that I would just emphasize, is that I know folks are trying to make comparisons, but in so many ways, they're incomparable because of the profound difference in the mechanisms |
| Thalassemia remains an area of high unmet needs with few treatment options |
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