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JP Morgan 2017 - Day 4
January 12, 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:
- Heron (HRTX) announced the submission of the New Drug Application (NDA) for Cinvanti (HTX-019), a polysorbate 80-free,
intravenous formulation of aprepitant for the prevention of chemotherapy induced nausea and vomiting (CINV), to the FDA. Heron
anticipates an approval decision in the fourth quarter of 2017, and if approved, it would mark a significant improvement in safety over the
current standard of care, Emend (IV), and become the second approved product for Heron in this indication alongside Sustol.
- In 2017, aTyr (LIFE) seeks to advance its pipeline on multiple fronts and obtain potential partnerships in one or
more of its programs. The Resolaris program for rare muscle diseases will continue to advance in clinical trials, with results from early
onset FSHD patients (Study 003) expected in the first half of 2017 and a new clinical trial planned for the second half of 2017, post
partnership. As for its Stalaris program, aTyr is planning the first in human clinical trial in patients with interstitial lung disease in the
second half of 2017. Lastly, aTyr plans to declare a third IND candidate from its Physiocrine Discovery Engine sometime in 2017.
- Mesoblast (MESO) focused on its four Tier 1 candidates, including (but not limited to) MPC-100-IV in Phase III
development in steroid-refractory acute graft versus host disease (aGVHD) for which Phase III pediatric complete enrolment is expected
in H1 2017, Phase III pediatric read-out is expected in Q4 2017 and U.S. pediatric launch is planned for 2018, and MPC-06-ID in Phase
III development in chronic low back pain due to degenerative disc disease for which Phase III complete enrolment is expected in H2
2017 and interim Phase III results are expected in Q1 2017. The Company’s resources are currently committed only to Tier 1 programs.
The composite primary pain and function endpoint in the ongoing Phase III study of MPC-06-ID (i.e. 50% VAS lower back pain
improvement and 15 point ODI improvement at both 12 and 24 months with no intervention at the treatment level) has been accepted for
FDA-approval. 360 patients are currently being recruited into the Phase III trial in the U.S. and Australia and complete enrolment is
expected in H2 2017.
The Company noted that MSC-100-IV for steroid-refractory aGVHD treatment is its nearest-term potential revenue driver (i.e. closest to market drug). The Company commented that aGVHD is a “niche market opportunity” for MSC-100-IV and “very attractive” to Mesoblast. The Company has received FDA agreement on the ongoing 60-children, open-label Phase III registration study, which will complete enrolment in H1 2017 and, if consistent with previous results, will form the basis of accelerated approval of the product for the pediatric population and U.S. launch in 2018. Mesoblast noted that the opportunity in adult aGVHD is 4-5 fold higher than in pediatric aGVHD and that MSC-100-IV development for adult aGVHD, targeting high-risk, non-responsive to steroids and at risk of death due to liver/gut disease patients, is being conducted in parallel with U.S. launch expected in 2021. The Company also commented on the scalability of its manufacturing, particularly its ongoing regulatory activities to meet FDA’s requirements for commercial manufacturing. Mesoblast also noted its ongoing partnership negotiations with Mallinckrodt in chronic pain and aGVHD.
Additionally, Mesoblast expects to conduct an interim analysis of its Phase III heart failure trial of MPC-150-IM (Allogeneic Mesenchymal Precursor Cells) in the first quarter of this year, which based on the 21st Century Cures Act, it hopes could guide its discussions with the FDA to identify a potential pathway for accelerated approval. (The Act facilitates the accelerated process for "regenerative advanced therapies" but does not change the standards.) - Retrophin (RTRX) announced that the Company will hold a meeting with the FDA in January 2017 to discuss the
results from the Phase II DUET study along with the regulatory pathway for sparsentan in focal segmental glomerulosclerosis. Upon
receiving feedback, the Company will provide an update on the next steps for the program within the quarter.
- In lieu of the September 2016 Phase III RESOLVE trial failure from its RSV F-protein recombinant nanoparticle vaccine
candidate (RSV F Vaccine) in older adults, Novavax (NVAX) plans to initiate a Phase II multi-arm trial in 300 older adults to
address immunosenescence through use of adjuvant and multiple dose regimens in the first quarter of 2017. Results from this study are
expected in the third quarter of 2017. Novavax also asked the FDA for an informational unblinding of data later in 2017 for its ongoing
Phase III PREPARE trial for infants via maternal immunization.
- Infinity (INFI) enters 2017 focused on advancing IPI-549 which the Company claims to be the only selective PI3K-
gamma inhibitor in clinical development. An ongoing Phase I/Ib study evaluating IPI-549 as both a monotherapy and combination
therapy has enrolled the first dose-escalation cohort, and updated results will be presented at the PI3K Keystone Symposia Conference
this month. Infinity believes that IPI-549 in combination with immune checkpoint inhibitors may overcome resistance to checkpoint
blockade.
- Corvus (CRVS) will look to advance their promising immune-oncology candidate CPI-444 in 2017. Having shown
some early single agent activity in PD-(L)1 refractory patients across multiple tumor types, the Company will look to build upon those
results with data presentations expected at AACR and ASCO. If the Phase I/Ib single agent and combination study with atezolizumab
ultimately proves to be a success, the Company aims to begin a registrational study by the end of this year.
- TG Therapeutics (TGTX) is advancing TG-1303, the Company’s combination product of TG-1101 (ublituximab)
and TGR-1202, across various lymphoma indications. Interim results of single agent arms from the Phase III UNITY-CLL study in chronic
lymphocytic leukemia (CLL) are expected in the second half of 2017. In addition, a first interim analysis of data from the Phase IIb UNITY-
DLBCL study in diffuse large B-cell lymphoma (DLBCL) are expected in mid-2017. Pending the results of UNITY-DLBCL, the Company
expects to drop TGR-1202 as “futile” and continue the TGR-1303 arm in approximately 100 patients. TG Therapeutics would then replace the TGR-1202 arm with TGR-1303 in combination with Benda.
- Intra-Cellular Therapies (ITCI) provided an update on its expected meeting with FDA regarding Lumateperone (ITI-007) for the treatment of schizophrenia, but was surprisingly quiet on the Phase III development of ITI-007 for the treatment of agitation in patients with dementia including Alzheimer’s disease. The Company has requested a meeting with FDA to discuss the submission of a New Drug Application (NDA) for ITI-007 for the treatment of schizophrenia, despite the disappointing top-line results of the second Phase III trial (Study ‘302) of ITI-007 in schizophrenia. Intra-Cellular expects to provide an update on the status of discussions with FDA in Q1 2017.
For our disclosures, please read the Biomedtracker Research Standards.
| Disease Group Covered: |
Autoimmune/immunology
Cardiovascular Endocrine Infectious Disease Neurology Oncology Renal Respiratory |
| Indications Covered: |
Dysmenorrhea
Post-Traumatic Stress Disorder (PTSD) Premenstrual Dysphoric Disorder (PMDD) Smoking Cessation Substance Use Disorder Wound Healing |
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