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JP Morgan 2017 - Day 2
January 10, 2017
The 35th annual JP Morgan Healthcare Conference is being held in San Francisco, CA from January 9-12, 2017. A list of events and
catalysts that were announced or updated at the conference today is included in this report.
Below are some key points from today’s company presentations:
For our disclosures, please read the Biomedtracker Research Standards.
Below are some key points from today’s company presentations:
- Sarepta (SRPT) recorded $5.4 million in sales of Exondys 51 in the fourth quarter of 2016 and has received over 250
genetically verified start forms, providing some reassurance to the market about the muscular dystrophy drug's launch, given questions
whether the FDA should have approved it in the first place. However, it is still quite early, and while only 8% of covered lives are in plans
outright denying coverage, only 13% are in plans with a favorable policy. 79% involve plans still pending a policy decision, reviewing
case-by-case, or approving with restrictions. Sarepta also presented initial data from their preclinical PPMO program (which involves
addition of a peptide to improve delivery and extend dosing), showing enhanced efficacy and good tolerability. IND-enabling GLP
toxicology studies expected to begin in early 2017 may lead to an IND before year-end 2017. The company also announced two gene
therapy collaborations with Nationwide Children’s Hospital, to expand their portfolio of treatment options.
- Alkermes (ALKS) gave an update on the development path for ALKS 5461 in major depressive disorder (MDD).
The Company is expecting a Type C meeting with FDA to discuss Phase III FORWARD-5 data in Q1 2017, a pre-New Drug Application
(NDA) meeting with FDA in Q2 2017 and submission of the NDA for ALKS 5461 as an adjunctive treatment in MDD in H2 2017. The
registration package will include data from all three Phase III trials, including the failed FORWARD-3 and FORWARD-4 trials. The
Company claims that the 2mg/2mg dose has shown consistent efficacy and safety across these trials in a treatment refractory patient
population and that FORWARD-5 and pooled analysis of FORWARD-3 and FORWARD-4 will support the NDA.
- 2017 is poised to be a deciding year for Axovant (AXON) as top-line results are expected from their Phase III
MINDSET study of intepirdine in Alzheimer's disease. Intepirdine is a selective 5-HT6 receptor antagonist that has shown modest yet
encouraging effects on cognition in a 684-patient Phase II study in mild-to-moderate Alzheimer's disease. Axovant also has data
readouts from three Phase II dementia studies of intepirdine and nelotanserin, a 5-HT2A receptor antagonist, slated for this year.
- Acadia (ACAD) announced that they are deferring the Nuplazid MAA filing based on the recent Alzheimer's
disease psychosis data, given the 10-year exclusivity period from approval of a first indication in the EU. If Acadia were to submit the
MAA for Parkinson’s disease psychosis, the exclusivity clock would start before any other indications could gain benefit. Acadia
announced that the pediatric plan (PIP) is not a factor, as it has been agreed upon at this time, and the Company will reevaluate the MAA
in 2018 as more data from the other Nuplazid indications are evaluated.
- AMAG Pharmaceuticals (AMAG) provided an update to their newly acquired Rekynda product being developed
for the treatment of hypoactive sexual desire disorder (HSDD). Rekynda has already completed two Phase III studies showing statistically
significant improvements in desire and decrease in distress associated with low sexual desire. AMAG is targeting an early 2018 NDA
submission. There is only one other approved product for HSDD (Addyi, VRX), and Rekynda has a more convenient on-demand dosing
as well as no interactions with alcohol, providing significant improvement over Addyi which requires a 30-day loading period and
contains a boxed warning to highlight the risks associated with alcohol.
- Esperion (ESPR) announced the initiation of the global cardiovascular outcomes trial (CVOT) to assess the
effects of ETC-1002 (bempedoic acid) on the occurrence of major cardiovascular events in statin intolerant patients with an expected
average LDL-C of ~135 mg/dl. The 12,600-patient study has 85% power to detect an ~14% relative risk reduction in the primary
endpoint. The study will be "well underway" when the company plans to initially file for approval of the drug for LDL-C lowering.
- Sage (SAGE) gave an update on the development of SAGE 547 in post-partum depression (PPD). The Company
expects top-line data from the pivotal Phase III (HUMMINGBIRD) trial of SAGE 547 in 220 PPD patients in H2 2017. Sage also discussed
possible development of SAGE 547 for (lower dose) in-home overnight infusion as well as in-hospital infusion upon success of the
HUMMINGBIRD trial. The Company seems particularly excited about the development of SAGE 217 in multiple indications and expects
top-line data from Phase II open-label, proof-of-concept studies in major depressive disorder and Parkinson’s disease in H1 2017 and
from Phase II placebo-controlled studies in essential tremor and PPD in H2 2017. If FDA-approved, Sage anticipates premium pricing for
SAGE 217 given its novel mechanism and treatment paradigm shifting potential, well above routine SSRIs and antipsychotics. During the
Q&A session, Sage commented on the easier development path of the IV formulation SAGE 547 versus the oral (more potent and longer-
acting) formulation SAGE 217, particularly in PPD. If FDA-approved SAGE 547 will allow PPD patients to resume breast-feeding within
seven days, however, Sage is unsure if that will be the case for SAGE 217. Nevertheless, Sage anticipates SAGE 217, although taking
longer to develop since it is an oral formulation, will likely be used more broadly than SAGE 547. The Company also outlined its strategy
of not initiating placebo-controlled trials until clear positive signals are read out from open-label trials in well-define patient populations,
or leading “with human data.”
- Clovis (CLVS) discussed the future of Rubraca (rucaparib), recently approved for the treatment of patients with
BRCA mutation (germline and/or somatic) associated advanced ovarian cancer. The Company looks forward to expanding Rubraca into
multiple new indications and treatment populations in the coming year. Initially, Clovis expects data from the ARIEL3 maintenance study
of rudaparib versus placebo in mid-2017. The trial allows for the potential to reach a larger population of ovarian cancer patients within
the maintenance indication, beyond the original BRCA and HRD patient populations. Further development programs were outlined,
including a combination therapy with Genentech’s Tecentriq for solid tumors and gynecologic cancers (focusing on ovarian cancers), and
the TRITON studies of Rubraca for the treatment of prostate cancer associated with homologous recombination deficiency.
- GlaxoSmithKline (GSK) provided multiple updates regarding late stage drug candidates, including their closed
triple therapy FF/UMEC/VI and Nucala. Pending confirmatory Phase III results, GSK plans to file for regulatory approval of FF/UMEC/VI for
the treatment of patients with exacerbations and for the treatment of asthma before the end of 2018. Regulatory filings for Nucala for the
treatment of hypereosinophilic syndrome (HES), nasal polyps, and eosinophilic granulomatosis with polyangiitis (EPGA) are also
expected to occur before the end of 2018.
- Merck (MRK) released a surprising disclosure that it had filed an sBLA for Keytruda in combination
with chemotherapy for first-line NSCLC. The filing was surprising in its reliance on the Phase I/II KEYNOTE-021 study rather than the
larger Phase III ‘189 study already ongoing. The move is likely designed to move quickly in the face of increased competition from other
PD-1 therapies, and while it would only be a conditional approval, the move would likely help maintain the momentum for Keytruda
following the high-profile approval for front-line monotherapy treatment obtained in late 2016. If approved, this combination therapy
would allow Keytruda incorporation into an even larger patient population rather than just the high-PD-1 expressers on the current label.
The PDUFA date is set for May 10, 2017.
- Shire (SHPG) announced that they filed a Class II resubmission of the NDA for SHP465 for the treatment of ADHD
on December 20, 2016. SHP465 is a long acting amphetamine offering efficacy at 16 hours post dose, and is expected to receive a 6-
month review. Shire also expects to see many other catalysts in 2017, one of which is the filing of an MAA for Xiidra for drye eye disease
in the third quarter, along with its continued uptake in the U.S. market.
- Ultragenyx (RARE) presented updated data from its adult extension study of KRN23 for X-linked hypophosphatemia, showing maintained lowering in serum phosphorous to low normal levels through 48 weeks, along with improvements in pain, stiffness, and physical functioning scores. The company reaffirmed they expect Phase III study in adults in the first half of 2017 and a US submission in the second half, without the need for the Phase III pediatric study. For triheptanoin in fatty acid oxidation disorders (FAOD), the company is still considering an exercise based versus event based pivotal trial design, and will provide an update after meeting with the FDA this year.
For our disclosures, please read the Biomedtracker Research Standards.
| Disease Group Covered: |
Autoimmune/immunology
Cardiovascular Endocrine Metabolic Neurology Obstetrics/Gynecology Oncology Ophthalmology Respiratory |
| Indications Covered: |
Post-Traumatic Stress Disorder (PTSD)
Premenstrual Dysphoric Disorder (PMDD) Smoking Cessation Substance Use Disorder Wound Healing |
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