Leerink Partners Reiterates Outperform Rating on Ultragenyx Pharma (RARE)

Leerink Partners analyst Joseph Schwartz reiterated an Outperform rating and $85.00 price target on Ultragenyx Pharma (NASDAQ: RARE).

The analyst comments "Bottom Line: Ultragenyx provided an update on the Cyprus2+ study for UX701 in Wilson disease, with the company opting to add a higher dose cohort before moving to Stage 2; we do not view this program as a core focus for the stock (at least in the near term), with most attention set on the ORBIT readout with setrusumab. UX701 is an AAV9 gene therapy in development for the treatment of Wilson disease, a condition characterized by the dysregulation of copper trafficking. Standard-of-care/SoC involves a low copper diet, copper chelators, and zinc. UX701 is advancing in the Ph.1/2/3 Cyprus2+ study (NCT04884815), broken into three stages: Stage 1 (safety and efficacy of UX701 across multiple dose levels), Stage 2 (randomized portion with efficacy/safety measured at 52 weeks), and Stage 3 (long-term follow-up). RARE shared initial Stage 1 data last year (here) from 5 patients in Cohort 1 (5.0 x 10^12 GC/ kg), which showed 4/5 patients tapering SoC, including 2 completely off chelators and/or zinc therapy. In the update today (PR here), RARE shared data across 15 patients in Stage 1 (5.0 x 10^12 GC/kg, 1.0 x 10^13 GC/kg, and 2.0 x 10^13 GC/kg). 6/15 of the patients have completely tapered off SoC and a 7th patient has begun to taper as of the August cut-off date. Management noted that responses have been seen in all three dose cohorts; however, a more granular breakdown was not provided. Based on the update last year, we know that 2 patients in the lowest dose cohort completely tapered off SoC. Thus, based on the updated 6/15 figure provided today, we can assume that a clear dose response is not being seen at this point (likely 1-3 patients have tapered completely off SoC in each Cohort 2 and 3). Based on this assumption, we are not surprised to see that the company plans to enroll an additional cohort in Stage 1 at a moderately increased dose with an optimized immunomodulation regime to enhance the efficiency and efficacy of UX701. The objective is to have the majority of patients come off SoC before selecting a dose for Stage 2. Management plans to submit a protocol amendment, and we look forward to future updates. We continue to model the program with a 30% probability-of-success/PoS and gross peak sales of ~$900M (US + EU). Regarding the stock, the core focus for investors remains on the setrusumab program, with a potential interim Ph.3 ORBIT study (NCT05125809) readout by year-end or early 2025. Reiterate Outperform."

For an analyst ratings summary and ratings history on Ultragenyx Pharma click here. For more ratings news on Ultragenyx Pharma click here.

Shares of Ultragenyx Pharma closed at $53.66 yesterday.

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