Sandy back to big mouth : $QURE dumped by BP (if any interest) , $ONCE FTC seeing GAMES THAT PLAYE | SGMO Message Board Posts

Sangamo Therapeutics, Inc.

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Msg  133078 of 135071  at  9/6/2019 9:50:53 AM  by

sangamo


 In response to msg 133077 by  StkTracker
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Re: Sandy back to big mouth : $QURE dumped by BP (if any interest) , $ONCE FTC seeing GAMES THAT PLAYED , BMRN mew construct , FDA no liky blocking tactics !?

 Voxelotor (previously called GBT440) is being developed as an oral, once-daily therapy
 
 this has nothing to do with our gene editing product
 

September is

NSC Awareness Month graphic
 
 
 
Takeaways from our Sickle Cell Disease MEDACorp KOL Call
 
 
 
• 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.
 
 
 


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