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JP Morgan 2017 - Day 3
January 11, 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:
- Enanta (ENTA) focused on its wholly-owned, non-alcoholic steatohepatitis (NASH) Phase I and primary biliary cholangitis
(PBC) preclinical candidate, and highly potent and selective farnesoid X receptor (FXR) agonist, EDP-305. The Company particularly
noted the lack of TGR5 (G protein-coupled bile acid receptor 1) activity of EDP-305 compared to Intercept Pharmaceutical’s FXR agonist,
Ocaliva, which is currently in Phase III development for NASH and FDA-approved (in May 2016) for PBC. TGR5 is associated with
pruritus which is an observed side effect of Ocaliva. The Company also alluded to future development of ‘next generation’ candidates in
NASH and PBC.
- Intercept (ICPT) focused on its NASH Phase III and primary sclerosing cholangitis (PSC) Phase II candidate, and
farnesoid X receptor (FXR) agonist, Ocaliva (obeticholic acid). The Company expects that the currently enrolling Phase III REGENERATE
trial, which will readout out in 2019, will form the basis of FDA-approval in NASH and noted that FDA and EMA pre-agreed on the co-
primary endpoints (i.e. fibrosis improvement with no worsening of NASH and NASH resolution with no worsening of fibrosis). Intercept
emphasized that these co-primary endpoints should not be considered a composite endpoint (i.e. as recognized by FDA and EMA,
fibrosis and NASH do not necessarily tract together).
- Cempra (CEMP) plans to meet with the FDA in February to discuss the complete response letter issued for
solithromycin in community acquired bacterial pneumonia (CABP). Management stated that resources would not be put towards the
9,000-patient safety study recommended by the FDA, given the lack of a clinically meaningful label regardless of the outcome of the
study. Learnings from the meeting will inform the Company's decisions on the next steps for solithromycin development. In the meantime,
Cempra awaits the European CHMP opinion expected in November and continues to enroll the pediatric CABP trial. The Company is
also advancing the drug in other disease areas including gonorrhea, NASH, and ophthalmology. Outside of solithromycin, Cempra's
other late-stage program, Taksta (fusidic acid), is due to report ABSSSI data in Q1 and an exploratory study in bone or joint infections is
enrolling.
- After conducting a retrospective analysis on Phase IIb results of OTO-104 for Meniere's disease, Otonomy (OTIC)
determined that by setting a floor of definitive vertigo days of 4 and a ceiling of 22, they can reach statistical significance from the Phase
IIb results at 3 months. The FDA agreed with the changes to the treatment criteria, allowing for a more homogenous population to be
treated. Otonomy expects Phase III results of OTO-104 in the second half of 2017 and an NDA submission in the first half of 2018. If
approved, this would be the only approved treatment for Meniere's disease in the U.S. market.
- In 2017 Chimerix (CMRX) will look to advance its pipeline on multiple fronts. Most notably will be the
advancement of IV Brincidofovir, for which the Company expects several new trials to begin including a Phase I multiple ascending dose
trial and a pair of Phase IIb trials. If successful, the Company could enter Phase III as soon as early 2018. IV Brincidofovir appears to be a
significant improvement over the oral formulation with an improved exposure profile, particularly in the GI tract. IV Brincidofovir prevents
“over-exposure” in the small intestine which was observed in the oral formulation causing GI toxicity.
- Ziopharm (ZIOP) provided updated results from the Phase I study of Ad-RTS IL-12 in combination with veledimex
in subjects with recurrent or progressive GBM, demonstrating median overall survival (mOS) of 12.7 months in the 20 mg dosing group.
This mOS compares favorably to historical controls in previous studies in recurrent GBM. Based on the Phase I results to date, Ziopharm
has scheduled an End-of-Phase II meeting with the FDA to discuss the regulatory pathway forward for Ad-RTS IL-12. Additionally, various
studies of Ad-RTS IL-12 in GBM are planned, including a pivotal Phase II/III study expected sometime in 2017, a Phase I pediatric study
as well as a combination study with veledimex and a checkpoint inhibitor, both in the first half 2017.
- With the recent sale of Onivyde, Merrimack (MACK) is entering 2017 with a drastically different approach to their
pipeline. Merrimack announced that seribantumab in NSCLC and breast cancer will now be Company’s top priority. Furthermore, the
Company amended the patient protocols in the Phase II SHERLOC trial of seribantumab in NSCLC to now include only half the number
of patients in an effort to push out the Company’s new “succeed fast, fail fast” strategy. With the sudden change in development plans,
Merrimack’s progress to initiate Phase II in breast cancer in 2017 combined with the expected data in NSCLC in 2018 will both play key
roles in the Company’s success in the near future.
- Rigel (RIGL) gave a detailed update on the progress of its fostamatinib product. The CEO started off with a review
of previously released data from the Phase III FIT trials for immune thrombocytopenic purpura (ITP). The presentation led to the release of
updated data from the Phase III 049 extension study which provided evidence that fostamatinib can provide enduring benefit to patients,
as seen by a lasting increase in median platelet levels. An NDA filing is planned for the first quarter of 2017. Furthermore, Rigel went into
detail about its plans to expand fostamatinib into other indications, notably with the presentation of preliminary data from the Phase II
study in patients with IgA nephropathy. Initial results from the first 100 mg BID cohort of fostamatinib showed a decrease of 54% in
proteinuria in patients, compared to a 36% decrease in the placebo population. The second 150mg BID cohort is currently enrolling
participants and further cohort 1 results are expected this month.
- PTC Therapeutics (PTCT) announced plans to file the Translarna NDA over protest with the FDA in the first
quarter of 2017. The FDA had refused the application for the nmDMD drug last year, but the company feels filing over protest is a more
promising strategy than continuing to appeal the decision.
- Bluebird bio (BLUE) expects initial data from their NorthStar-2 pivotal trial of LentiGlobin in transfusion-dependent
beta-thalassemia at this year's EHA meeting, though there will only be a limited number of patients and follow-up. The update will be
more about what the improved manufacturing process achieves in vivo. Further data in that indication and sickle cell disease, where
additional measures are being tried to improve efficacy, will be presented later at ASH. Given the gene therapy could be a one-time,
curative treatment in certain settings, officials reiterated that they have started early discussions with payers around innovative
reimbursement options, from annuity-based to pay-for-performance and other risk-sharing strategies.
In late 2016 Bluebird presented top-line data from a Phase I study of BB2121, their CAR-T product targeting BCMA in multiple myeloma. These early results showed strong efficacy and an excellent safety profile (no dose limiting toxicities, no neurotoxicities or cytokine release syndrome above grade 2). Bluebird plans to release updated data at ASCO which should provide insight into the durability of the response and the safety profile. Bluebird is also working on BB21217, a second generation CAR-T product which they plan to bring to the clinic in 2017. BB21217 incorporates a PI3K which promotes younger T cells in the product. Younger cells persist better in animal models and may translate into a more durable response. Bluebird advanced two hypotheses for why their CAR-T product showed a better safety profile than their competitors: (i) their manufacturing process is different from other companies; and (ii) they screened their CAR-T constructs until they found those that resulted in a low basal level of activation as measured by IFN-gamma release in the absence of BCMA. - Kite Pharma (KITE) announced that it anticipates releasing the primary 6-month analysis from its pivotal Phase I/II
ZUMA-1 trial of its CAR-T cell therapy KTE-C19 in patients with aggressive non-Hodgkin’s Lymphoma during the first quarter of 2017.
These data will support a rolling BLA submission in the same time frame and a regulatory filing in Europe later this year. With KTE-C19
primed for a product launch later this year, if approved, Kite has furthered development of this therapy into additional indications with top-
line data in several other lymphomas expected during 2017 and 2018. Kite also announced filing an IND for several new CAR and TCR
clinical programs in the near-term, along with details of a new anti-CD19 control CAR technology designed to allow for dialable, instant,
and reversible control of CAR activity.
- Aclaris (ACRS) provided further details regarding their JAK inhibitor program with Rigel that was originally
announced in September 2015. The Company today introduced ATI-50001, ATI-50002, and ATI-50003 as three potential candidates for
their alopecia and vitiligo program. Both ATI-50001 (oral) and ATI-50002 (topical) are expected to enter a Phase II clinical study in the
third quarter of 2017 for the treatment of alopecia.
- Advanced Accelerator Applications (AAAP) announced plans to meet with the FDA within the next month to address the issues in their December 2016 Complete Response Letter regarding format, traceability, uniformity, and completeness relating to the NETTER-1 and Erasmus clinical datasets. Following the meeting, the Company plans on updating the timeline for submission of the revised datasets and other requests of the FDA. AAAP does not believe any of these issues will affect their submission within the EU.
For our disclosures, please read the Biomedtracker Research Standards.
| Disease Group Covered: |
Allergy
Autoimmune/immunology Cardiovascular Dermatology Endocrine Infectious Disease Metabolic Neurology Not Specified Oncology Ophthalmology |
| Indications Covered: |
Dysmenorrhea
Post-Traumatic Stress Disorder (PTSD) Premenstrual Dysphoric Disorder (PMDD) Sleep Apnea Smoking Cessation Substance Use Disorder |
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