|
|
Biotech
|
|
||
Re: Trade - EIGRReport from which previous message was responding to: Takeaways from our Sickle Cell Disease MEDACorp KOL Call Takeaways from our Sickle Cell Disease MEDACorp KOL Call https://www.investorvillage.com/smbd.asp?mb=1933&mn=133078&pt=msg&mid=19760949 (thanks to Sangamo) • Bottom Line: Yesterday, we hosted a call with two MEDACorp KOLs to discuss the sickle cell disease (SCD) treatment landscape and emerging therapies – one KOL is director of a large adult SCD treatment center and the other treats over 150 SCD patients at a pediatric treatment center. While both were enthusiastic for new treatment options with differentiated mechanisms of action coming to market, they were more tempered about potential uptake in the SCD population, particularly for medicines that appear to provide limited benefit beyond that of standard-of-care hydroxyurea (HU). New treatments are likely to see most use in the segment of patients with renal insufficiency and in patients who have at least 2 vaso-occlusive crises (VOCs) per year while on hydroxyurea. KOLs also see gene therapy as a promising approach for a potentially curative therapy, though they do not believe sufficient data has been presented to determine how the clinical risk/reward of LentiGlobin stacks up vs allogeneic stem cell transplant, and open questions remain regarding whether the long-term efficacy profile of LentiGlobin is adequately robust to outweigh risks of ablative preconditioning. Though small molecule and antibody competitors are entering the SCD market as well, KOLs see limited overlap between these agents and LentiGlobin, and we continue to see ~$850M in peak unadjusted WW sales. Reiterate Market Perform rating and $121 PT for BLUE shares. • Gene therapy is seen as the most promising approach for SCD in the long term, but it is early to assess potential clinical profile of LentiGlobin. The KOLs see gene therapy as an attractive approach as a potentially curative SCD treatment, but are uncertain as to whether current iterations will live up to the enthusiasm in real world practice. Increases in hemoglobin and reductions in VOCs with LentiGlobin have been promising, though our KOLs believe more long-term data are needed to assess risk/benefit adequately – VOC’s don’t necessarily predict end organ damage in SCD, making longer term benefit on mortality and organ system failure difficult to extrapolate from VOC reduction or total hemoglobin benefit. The KOL who specializes in adult SCD noted that end organ damage sharply increases for many patients during their 30s and prevention of this damage with autologous gene therapy could greatly improve these patient outcomes vs available options – these patients often cannot receive stem cell transplant due to lack of HLA-matched donors. However, the two largest issues that dissuade patients and families from treatment with stem cell transplant - risk of secondary malignancies and loss of fertility due to the use of preconditioning - are present with LentiGlobin as well, and these concerns could hinder adoption. • Hydroxyurea (HU) is the standard-of-care treatment for SCD, though use in the real world is uneven between different treatment settings. Though some physicians recommend HU for all patients, many are more conservative, recommending HU only for more severe patients. Both KOLs agreed HU should be recommended for patients with 2 or more VOCs per year, though the treatment burden and required monitoring doesn’t justify treatment in most patients with only 1 VOC per year (~30% of SCD patients). One KOL also noted that in his experience, the most significant factor for patients who decline treatment with HU is risk to fertility – male patients can develop low sperm count, and women who are trying to become pregnant should not take HU. • Real-world use of voxelotor is expected to be narrow despite beneficial effect on hemoglobin, impact on hemolysis. KOLs believe it will be difficult to convince many patients that a 1g/dL increase in hemoglobin is meaningful – this modest increase might incrementally improve exercise tolerance, but patients care most about reduction in VOCs, prevention of end organ damage, and life extension. KOLs see voxelotor as best used in patients with renal insufficiency and other patients that cannot be treated with HU. In the SCD population at large, however, our KOLs believe that neither physicians nor payers will be motivated to treat hydroxyurea-responsive patients with voxelotor due to the modest absolute hemoglobin benefit and unanswered questions for long-term efficacy on other endpoints. • Patients who experience at least 2 crises a year on HU would be ideal candidates for crizanlizumab, but utilization will likely be limited by treatment burden of frequent IV administration. Our KOLs were intrigued and excited by the efficacy of crizanlizumab, with Phase 2 clinical data demonstrating 37% of patients VOC-free in one year from commencement of treatment seen as very encouraging. 20-30% of our KOL’s patients would make good candidates for treatment with crizanlizumab, but only an estimated 10% would be likely to receive commercial crizanlizumab after presumed approval next year. KOLs noted that a q4week IV infusion would be a major treatment burden for most SCD patients, likely to forestall widespread uptake in real world clinical practice. KOLs also noted that cost could potentially pose a barrier to uptake, as it has for Endari (despite a very different mechanism of action and degree of treatment burden). A KOL noted that of five patients prescribed Endari, only one received insurance coverage. That being said, the benefit of L-glutamine is relatively small compared to HU and pricing of crizanlizumab has yet to be disclosed, and the intensity of potential reimbursement challenges will depend (among other factors) on the granted label and on NVS’ pricing strategy for crizanlizumab. |
return to message board, top of board |
Msg # | Subject | Author | Recs | Date Posted |
28273 | Re: Trade - EIGR | arthurs1 | 2 | 9/11/2019 12:46:07 PM |