Scrip: industry news and insights
26 Nov 2020
Executive appointments at MyoKardia, Scancell Holdings and Sutro Biopharma
Biomedtracker’s Best Of J.P. Morgan: 15 Companies To Watch
Highlights from Biomedtracker’s daily reports from the J.P. Morgan Healthcare Conference include products advancing in NASH, competition in CAR-T and more.
Incyte Corp. provided a roadmap for its main revenue driver, Jakafi. Specifically, officials seemed excited about advancing its development for graft-versus-host disease and essential thrombocytopenia. Baricitinib will be the next revenue driver, according to the CEO, with a focus on psoriatic arthritis. IDO inhibitors have also been a new target class of intense interest, and the company’s epacadostat appears to be advancing strongly. Positive new data from the Phase I/II ECHO-202 combination study with Merck & Co. Inc.’s Keytruda in solid tumors were released with the meeting, and development will advance to Phase III in bladder, head & neck, NSCLC, and renal cell cancer in addition to the already Phase III melanoma indication. New data are anticipated at ASCO 2017. Data collection is ongoing for DLBCL and MSI-high colorectal cancer and future studies in those indications has not been decided. In ovarian and triple-negative breast cancer, there are no current Phase III plans as data does not justify moving forward at this point in time. Finally, the company initiated a collaboration with Merus NV to develop bispecific antibodies.
Regeneron Pharmaceuticals Inc. announced upcoming milestones for its IL-4/IL-13 antibody Dupixent for the treatment of allergic disorders. Regeneron believes it has found the “control point” for a variety of allergic diseases and that this drug can be expanded to indications including food allergies. To date, there has been significant efficacy data presented from the Phase III SOLO 1 and 2 trials in atopic dermatitis with top-line data in other indications including esophagitis and asthma expected by the end of the year. An FDA action date has been set for the BLA in atopic dermatitis at the end of the first quarter of 2017, and Regeneron expects to submit regulatory filings in asthma by the end of the year. Throughout the presentation, the CEO expressed the most optimism towards the direction of Dupixent as a promising first-in-class treatment, if approved, for this significant unmet need.
AMAG Pharmaceuticals Inc. provided an update on their newly acquired Rekynda (bremelanotide) product being developed for the treatment of hypoactive sexual desire disorder (HSDD), licensed from Palatin Technologies Inc. Jan. 9. 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, Valeant Pharmaceuticals International Inc.’s Addyi (flibanserin), 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.
SAGE Therapeutics Inc. 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 I.V. 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 Oncology Inc. 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 rucaparib 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 Inc.’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.
Enanta Pharmaceuticals Inc. 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 Pharmaceuticals Inc.’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 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 track together).
In 2017 Chimeric Therapies Inc. will look to advance its pipeline on multiple fronts. Most notably will be the advancement of I.V. 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. I.V. brincidofovir appears to be a significant improvement over the oral formulation with an improved exposure profile, particularly in the GI tract. I.V. brincidofovir prevents “over-exposure” in the small intestine which was observed in the oral formulation causing GI toxicity.
With the recent sale of Onivyde, Merrimack Pharmaceuticals Inc. is entering 2017 with a drastically different approach to their pipeline. Merrimack announced that seribantumab in NSCLC and breast cancer will now be the company’s top priority. Furthermore, it 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 Pharmaceuticals Inc. 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.
bluebird bio Inc. 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 that 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 that 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 Inc. 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 timeframe 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.
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 Inc. 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 Pharmaceuticals Inc. enters 2017 focused on advanccng 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 Pharmaceuticals Inc. 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.
[Editor’s note: Full daily reports from the J.P. Morgan Healthcare Conference are available from Biomedtracker.]
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