The 33rd annual JP Morgan Healthcare Conference was held in San Francisco, CA from January 12-15, 2015. A comprehensive list of events and catalysts that were announced or updated at the conference is included in this report. For the first time, this report includes coverage of the device and diagnostic presentations at the conference, provided by the newly launched medtech platform, MedDeviceTracker.

We also provide some key highlights from the presentations, as well as a summary of the biggest stock movements that occurred during the conference. Additionally, a review of last year’s dealmaking activity for the companies that presented at the 2014 JPM conference is provided by Strategic Transactions and MedTrack.

Key highlights from the week of presentations:

• AG-120 for Hematologic Cancer (AGIO, CELG). Agios and Celgene announced that Celgene has exercised its option to license the isocitrate dehydrogenase (IDH) inhibitor AG-120 outside the U.S. The companies have been involved in a global strategic collaboration agreement, and Celgene exercised its licensing option for AG-221 (also an IDH inhibitor) last year. Both of these IDH inhibitors may alter cell metabolism to induce differentiation in cancer cells, thus reducing levels of aberrant proliferation.
• Rociletinib for NSCLC; Rucaparib for Ovarian Cancer (CLVS). Clovis provided updated FDA filing timelines and development plans for rociletinib in non-small cell lung cancer and rucaparib in ovarian cancer. The company reiterated plans to file for FDA and EMA approval for rociletinib in T790M mutation-positive NSCLC patients in mid-2015, based on data from the TIGER-2 and TIGER-X (Phase II expansion) trials. Qiagen (QGEN) is working with Clovis to develop a companion diagnostic to test for T790M mutations. Furthermore, the company is now developing rociletinib in the larger population of T790M-negative patients, cohorts of whom are being added to the TIGER-2 and TIGER-3 trials. Patients without biopsied T790M mutations may benefit from rociletinib treatment due to tumor heterogeneity or due to the agent’s inhibition of IGF-1R, since the IGF pathway may play a pro-proliferative role in patients with acquired resistance to EGFR TKIs.
  
  In addition, the company now plans to file for FDA approval of rucaparib in ovarian cancer with a “BRCAness” signature in 2016, based on results from the expanded Phase II ARIEL2 trial. Clovis is working with Foundation Medical (FMI) to develop a companion diagnostic to test for BRCA and other DNA repair mutations.
  
  
• CAR-T Therapies for Cancer (JUNO). Juno provided updates for its early-stage development pipeline, including two preclinical CAR-T products targeting solid tumors. One CAR-T product for ovarian cancer targets MUC-16 (which is cleaved to generate CA-125) and utilizes the company’s “armored CAR” technology to express the immunostimulatory cytokine IL-12; a Phase I trial will begin in 2015. A second CAR-T program set to start a Phase I trial in 2015 targets ROR-1, an embryonic antigen expressed on some lymphomas and epithelial cancers.
• KTE-C19 for DLBCL (KITE). In one of the final sessions, KITE rounded out a slew of interest in CAR-T therapies at JPM. The presentation itself was fairly uneventful. Perhaps the most notable aspect was the strong public commitment to developing KTE-C19 and Kite’s other CAR-T therapies for larger-market solid tumors. Indeed, Kite’s CEO suggested that the promising results so far in DLBCL, especially bone metastases associated with DLBCL, apply to solid tumors. Regardless, Kite’s short-term focus remains on developing KTE-C19 for DLBCL. In the longer-term, Kite’s CEO sounding optimistic that the collaboration to leverage Amgen’s tumor targets and antibodies would fuel Kite’s pipeline for the next 5-10 years.
• MM-398 for Pancreatic Cancer (MACK). Merrimack highlighted its lead pipeline asset, MM-398 including an announcement that a rolling NDA had been initiated in pancreatic cancer with a completed filing anticipated in late Q1 or early Q2 2015. The presentation emphasized that approval is being sought for all post-gemcitabine use, not just second-line or later since gemcitabine can be used as adjuvant therapy. A partnership with Baxter is also specifically evaluating MM-398 in the front-line pancreatic cancer setting. Finally, evaluation of MM-398 as a front-line gastric cancer therapy has also restarted after an apparent halt to development, and a Phase II study in gastric cancer is anticipated in to initiate in 2015.
• Keytruda for Non-Small Cell Lung Cancer (MRK). Merck added more pressure to the hotly-contested race to develop PD-1 immunotherapies by announcing plans to accelerate their timeline for Keytruda (lambrolizumab) in NSCLC. A BLA filing is now planned for mid-2015 which would put a potential approval much closer the anticipated approval for Opdivo near year end 2015. Additionally, Merck and Eli Lilly announced a collaboration to conduct several oncology trials evaluating Keytruda in combination with LLY’s compounds including pemetrexed, ramucirumab, and necitumumab.
• PF-06463922 for NSCLC (PFE). Pfizer provided a fairly informative overview including the guidance that a pivotal NSCLC trial for their promising ALK-inhibitor, PF-06463922, is expected to start in 2015. Little information has been publicly released for this drug, but a fairly large Phase I/II study initiated nearly a year ago. The presentation also revealed broad plans to evaluate PFE’s immuno-oncology agents in combination with Merck KGaA’s PD-1 inhibitor, avelumab, after progress evaluating avelumab as a monotherapy has been made.
• PCSK9 Programs for Hypercholesterolemia (PFE). A Pfizer official said they are developing an oral pill and vaccine targeting PCSK9, and expects to begin clinical trials this year for the small molecule pill. Preclinical studies reportedly showed a substantial reduction in cholesterol.
• REGN2810 for Cancer (REGN). Regeneron is joining the checkpoint inhibitor race with the disclosure of an IND filing for its PD-1 inhibitor, REGN2810.
• VX-661 for Cystic Fibrosis (VRTX). Vertex released additional details about the VX-661 + ivacaftor Phase III program and announced it is to start in February. The program will include only one F508del homozygous study, but also additional heterozygous trials in patients with a gating mutation, residual function mutations, and minimal CFTR function. Phase IIb data from the double combination in F508del homozygous patients is expected to be released early this year, and officials additionally said a much anticipated trial with a next-generation corrector added to the double combination will also start this year.
• Non-viral CAR-T Program (XON). Intrexon, Ziopharm (ZIOP), and MD Anderson announced a collaboration to develop a new CAR-T therapy. The program licenses technology from MD Anderson to generate CAR-T cells through a non-viral DNA/transposon-based genetic engineering approach as opposed to the more common viral gene transfer techniques. The non-viral approach could lead to differentiation from the increasingly crowded CAR-T landscape by reducing or eliminating the T-cell expansion time with directly infused gene-transferred cells expanding in the patient. This could potentially reduce the treatment delay to hours instead of weeks. The JPM presenter also suggested it could lead to off-the-shelf (allogeneic) products, but it was unclear how those would be differentiated from any generated by traditional viral gene transfer. The collaboration is expected to generate up to five CARs entering the clinic in 2015 with off-the-shelf programs starting in 2016.
• ZS-9 for Hyperkalemia (ZSPH). ZS Pharma continues to focus on its lead asset, ZS-9, which binds potassium to treat hyperkalemia. Positive pivotal trial data have already been presented, and regulatory filings are anticipated in the first half of 2015. The JPM presentation suggested pricing for ZS-9 could be in-line with other drugs used to treat electrolyte imbalances including Renvela ($750-800/mo.) and Sensipar. A tablet formulation, rather than the powder being used at launch, should be available approximately 1 year after initial approval.

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