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| Lastly, we are seeing strong success in reauthorizations as the vast majority of reauths have been approved |
| We continue to see solid commercial execution this quarter with new prescriptions, prescribers and reimbursement |
| In addition, we continue to see gains in the depth and breadth of prescribers and positive reauthorization decisions |
| In Spain, the recently announced positive reimbursement decision covers both BBS and POMC LEPR biallelic patients |
| In addition, our Rhythm@Home services program, through which we provide patient support for each patient and their caregivers, has been quite successful |
| We feel very good about the level that we're at, both from Rx growth and reimbursed patients on therapy perspective |
| So we remain positive in terms of the opportunities for growth |
| Overall, the fourth quarter and the first full year of BBS commercialization has been strong |
| Within this 85%, we have stronger coverage for IMCIVREE as we have increased the number of states with a specific IMCIVREE policy in place |
| We continue to see incremental improvement in securing access and reimbursement with regard to overall coverage by state Medicaid programs |
| What is important is that the feedback from the HEPs and the feedback from the patients through the HEPs is very good, and the discontinuation rate that we observe is low |
| So again, in terms of field activity interactions, I think we are doing a very good job |
| Execution in that trial remains strong with a high level of sight and patient engagement |
| And that has remained consistent quarter-over-quarter, as well as the ones that have already prescribed and had a good experience with their patients |
| Third, we had another solid quarter commercially with $24.2 million in revenue, over 100 new prescriptions and more than 70 approvals reimbursement |
| Beginning on Slide 15, we remain pleased with the growth and consistent demand for IMCIVREE since launching in June of 2022, marking six full quarters and our first full calendar year |
| story continues to grow as expected, and we finished the quarter in a very good place, and Jennifer will, of course, provide more color in her section |
| Over 70% of approvals for reimbursement during the quarter came at the time of prior authorization or sooner, a trend that has shown incremental improvement each quarter since launch |
| Both countries are single-payer systems and have a national negotiated price, which is very good |
| The proportion of revenue generated by our International region increased from 20% to 24% quarter-over-quarter, reflecting strong growth in BBS patients on therapy in Germany |
| Another good aspect from a patient point of view in Japan, as you know, there are very good data, our hospital data |
| That sounds very promising for us |
| As previously described, we were impressed by the robust preclinical package and by the data in their Phase 1 study in healthy volunteers with obesity, which showed the expected dose dependent decrease in BMI at four weeks |
| We introduced this concept at the end of the first quarter when we reported that approximately 75% of Medicaid-covered lives were in states with either a positive IMCIVREE policy or in a state, where we had been able to gain positive coverage in at least one instance in the absence of an IMCIVREE policy |
| We are pleased to see this source of growth continue as we find new prescribers through our non-personal promotion efforts |
| I find these results quite remarkable for a couple of reasons |
| And Jennifer and I had a call with their leadership, which was incredibly constructive |
| 2023 was a very successful year for the international organization |
| 2024 will be a year focused on execution, setting up an exciting year of milestone achievements in 2025 |
| We are excited about our recent reimbursement approvals for BBS in Spain and Italy |
| Statement |
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| And in the worst cases, the parents are blamed for their inability to control their child's food intake |
| This pattern is consistent with our belief that revenue growth in rare diseases is difficult to trend quarter-over-quarter, but in the long term and globally, slow and steady growth continues |
| Therefore, during Q4, the total number of reimbursed patients dipped below where we exited Q3 |
| But we do think it's going to be consistently difficult to model if you will |
| One is that, despite the extremely young age, patients between the ages of two and five with either POMC LEPR deficiency or BBS, they were severely affected |
| But, you know, particularly non-Caucasian populations, it's an issue |
| Quarterly net loss per share was $0.70 |
| As David mentioned earlier, these positive trends were offset by a challenge associated with one payer |
| R&D expenses were $29.9 million for the fourth quarter of '23 compared to $23.5 million during Q4 '22, and an 11% decrease compared to Q3 '23 |
| In the meantime, incentive coverage for these patients was rescinded |
| So that's a huge issue for some patients, not all |
| For the first two groups starting from left to right, POMC and LEPR on the left hand side of the graph, these children are experiencing a five point to seven point decrease in their BMI Z scores from a starting point as high as 12 |
| First one on just looking at the [ENRx] (ph) so it seemed to be down slightly the last two quarters |
| And that's why it will be slow and steady |
| We basically just, you know, overwhelmed them to a certain extent with -- relative to what they had expected to see |
| Revenues in the quarter were impacted by a change in policy made by a single Medicaid program in one state in response to a disproportionately high volume of prescriptions |
| Expanding the label to children two and above will not open up a significant additional market opportunity |
| So, to be honest with you, I don't expect it to be better than setmelanotide |
| In the US, revenues were relatively flat quarter-over-quarter due to the shift of 30 patients to our bridge program early in the quarter, as David and Jennifer mentioned, Given that the 30 patients lost reimbursement early in the quarter, that change represented approximately a little more than $2 million in foregone potential revenue in the fourth quarter |
| So one, we don't expect them to drop back in next quarter |
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