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Ultragenyx Pharmaceutical Inc. (RARE)

Shares of this biopharma were just initiated at Credit Suisse with an “Outperform” rating, and analysts’ mean price target is $118.20.

Ultragenyx Pharmaceutical Inc. (RARE)
From Cannaccord Genuity

After meeting with management of Ultragenyx Pharmaceutical Inc. (RARE), we believe first regulatory approval and launch for AceER are on track with commercial launch preparations well underway. Second conditional MAA filing for KRN23 in pediatric X-linked hypophosphatemia (XLH) is expected in 2H16 following additional 40-week and 64-week data.

With a pipeline well-diversified across therapeutic areas and modalities on track to deliver steady data flow over the next 2-3 years, we continue to view RARE shares as attractive and reiterate our BUY rating.

Following the EU Conditional Marketing Authorization (CMA) filing for Ace-ER (aceneuramic acid extended release) for GNE myopathy in 4Q15, we expect conditional approval in 2H16 and positive Ph3 results in 1H17 to provide the basis of full approval in 2018. Ace-ER provides sialic acid that ameliorates the sialic acid deficiency resulting from GNE mutations, thereby attenuating the progressive muscle wasting of GNEM.

Interim KRN23 TIO data in 1H; conditional KRN23 MAA filing on track for 2H16 based on Ph2 pediatric X-linked hypophosphatemia (XLH) data. As anti-FGF23 monoclonal antibody KRN23 has shown benefit in 36 patients across different rickets and musculoskeletal measures at 40 weeks, we expect 64-week data and data in 52 patients, including 16 with more severe rickets, in mid-year to strengthen the available dataset and to be followed by CMA filing. For another disease of excess FGF23 levels, tumor-induced osteomalacia (TIO), we expect interim Ph2 results in 1H.

For enzyme replacement therapy recombinant human beta-glucuronidase (rhGUS) for mucopolysaccharidosis 7 (MPS7), we expect positive Ph3 data mid-2016 to support regulatory approvals 2017-18.

Substrate replacement therapy UX007 (triheptanoin) previously showed reduction in paroxysmal episodes in glucose transporter type 1 deficiency syndrome (Glut1 DS) patients and promising results in musculoskeletal manifestations of long-chain fatty acid oxidation disorders (LC-FAOD). We expect Ph2 52-week Glut1 DS results in 2H and 78-week LC-FAOD data in mid-year to guide Ph3 clinical trials.

RARE is building out its own global commercialization infrastructure to identify and diagnose patients, enable physicians to better identify and treat rare diseases, and ensure eligible patients receive the treatments they need. For example, in the US where Glut1 DS genotyping is available but generally not reimbursed, RARE has initiated a program to facilitate genetic testing and educate Glut1 DS families about the disease. We think this type of field work in the US and Europe will facilitate identification of patients with rare disease.

In 2016, we expect Ph3 rhGUS data in MPS7 (mid-16); Ace-ER CMA in GNEM (2H) Ph2 pediatric XLH KRN23 40-week data in 52 patients and 64-week data in 36 patients (mid-16), interim Ph2 TIO data (1H); Ph2 UX007 52-week data in Glut1D (2H) and LC-FAOD 78-week data (mid-16).

In 2017, we expect regulatory approvals and launches for rhGUS and Ace-ER, EMA CMA for KRN23 in pediatric XLH; Ph3 data for Ace-ER in GNEM and adult XLH data, and entry of fifth drug candidate, rhPCCA for galactosialidosis, into the clinic.

Arlinda Lee, Ph.D., Canaccord Genuity Research,, 617-371-3711, January 19, 2016