Eagle Pharma set to refile Ryanodex NDA; shares ahead 5%

In a regulatory filing, Eagle Pharmaceuticals (EGRX +5.2%) discloses that it expects to resubmit its U.S. marketing application for Ryanodex (dantrolene sodium) for the treatment of exertional heat stroke “shortly.”

The company received a CRL in July 2017 citing the need for an additional clinical trial. In its Q3 earnings press release, it stated that it planned to seek FDA sign-off on the adequacy of data on 41 people who participated in Hajj pilgrimages in 2015, 2018 and 2019. Target enrollment for the Phase 3 study launched in August 2018 was 100. The estimated completion date was August of this year.

https://seekingalpha.com/news/3525189-eagle-pharma-set-to-refile-ryanodex-nda-shares-ahead-5

Sage to advance Huntington’s candidate SAGE-718; shares up 2%

Citing encouraging Phase 1 data, Sage Therapeutics (SAGE +1.6%) plans to advance allosteric NMDA receptor modulator SAGE-718 to Phase 2 for the treatment of Huntington’s disease (HD), an inherited disorder in which nerve cells in the brain progressively degrade resulting in uncontrollable movements in the limbs, face and upper body in addition to cognitive and psychiatric problems.

Results from a 14-day open-label Phase 1 showed a favorable safety, tolerability and pharmacokinetic profile and improved performance in six HD patients who received daily doses of SAGE-718. Additional data will be presented at a medical conference in 2020.

https://seekingalpha.com/news/3525202-sage-therapeutics-to-advance-huntingtons-candidate-sageminus-718-shares-up-2

Dermira’s lebrikizumab Fast Track’d for atopic dermatitis; shares up 12%

Dermira (DERM +12.3%) is up on modestly higher volume in reaction to Fast Track status in the U.S. for IL-13 inhibitor lebrikizumab for the treatment of atopic dermatitis.

Two Phase 3 studies, ADvocate 1 and ADvocate 2, are in process.

Fast Track provides for more frequent interaction with the FDA review team and a rolling review of the marketing application.

https://seekingalpha.com/news/3525169-dermiras-lebrikizumab-fast-trackd-for-atopic-dermatitis-shares-up-12

Intra-Cellular up 10% ahead of presentation of lumateperone data

Intra-Cellular Therapies (ITCI +10%) perks up on modestly higher volume ahead of a presentation of lumateperone data tomorrow at the American College of Neuropsychopharmacology Annual Meeting in Hollywood, FL.

Results from a Phase 3 study in bipolar depression, pooled data in schizophrenia and results from a 12-month safety study will be presented at 5:30 pm ET.

The FDA’s action date for the company’s application seeking approval of lumateperone for schizophrenia is Friday, December 27.

https://seekingalpha.com/news/3525176-intra-cellular-up-10-ahead-of-presentation-of-lumateperone-data

Bluebird bio up 3% on updated CAR T candidate bb21217 data

Bluebird bio (BLUE +3%) and collaboration partner Bristol-Myers Squibb (BMY +0.5%) (via its acquisition of Celgene) announce updated results from a two-part open-label Phase 1 clinical trial, CRB-402, evaluating BCMA-targeted CAR T candidate bb21217 in heavily pretreated patients with relapsed/refractory multiple myeloma (MM), a population with a poor prognosis. The data were presented at ASH in Orlando.

The dose-escalation portion (three dose levels) is finished. The dose-expansion phase is in process. Final enrollment should be 74 subjects.

12 patients in the 150 x 10⁶ CAR+ T cells arm (the lowest dose) were evaluable. Mean follow-up was 17.6 months. 83% (n=10/12) showed clinical response, including four complete responders and six with very good partial responses. Median duration of response was 11.1 months.

As of the data cutoff, follow-up was still early in the other dose arms (300 x 10⁶ CAR+ T cells and 450 x 10⁶ CAR+ T cells) but no confirmed responders have progressed. The response rate in the 300 group was 43% (n=6/14), none complete, and 57% (n=4/7) in the 450 group with one complete responder. Median follow-up periods were 4.0 months and 3.3 months, respectively.

On the safety front, the most frequent serious/life-threatening adverse events in 38 treated patients were neutropenia (82%), leukopenia (55%), thrombocytopenia (55%), anemia (50%), lymphopenia (34%), hypophosphatemia (21%), hyponatremia (13%) and febrile neutropenia (11%). Seven patients experienced serious/life-threatening infections.

66% (n=25/38) developed bb21217-related cytokine release syndrome (CRS), most mild or moderate. There was one case of serious CRS and one death (in the 450 arm after 15 days of follow-up). 24% (n=9/38) developed neurotoxicity, two serious and one life-threatening (encephalopathy).

The estimated completion date is January 2025.

#ASH19

https://seekingalpha.com/news/3525157-bluebird-bio-up-3-on-updated-car-t-candidate-bb21217-data

Amgen Phase 3 KYPROLIS-DARZALEX data is ASH19 late breaker

Amgen (NASDAQ:AMGN) today announced additional results from the primary analysis of the Phase 3 CANDOR study evaluating KYPROLIS^® (carfilzomib) in combination with dexamethasone and DARZALEX^® (daratumumab) (KdD) compared to KYPROLIS and dexamethasone alone (Kd) in patients with relapsed or refractory multiple myeloma. The data will be presented in a late-breaking abstract session at the 61^st American Society of Hematology (ASH) Annual Meeting & Exposition.

At a median follow up of 17 months, the study met its primary endpoint of progression-free survival (PFS), resulting in a 37% reduction in the risk of disease progression or death in patients receiving KdD (HR=0.63; 95% CI: 0.464, 0.854; p=0.0014). Median PFS was not reached for the KdD arm versus 15.8 months for the Kd arm.

“This primary analysis of the CANDOR study adds to the body of evidence supporting the combination of KYPROLIS and DARZALEX, two powerful targeted agents for multiple myeloma,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “KYPROLIS has demonstrated deep and sustained responses in treating patients with multiple myeloma that have relapsed. The CANDOR study now offers additional insight into the effectiveness of this combination as a potential new treatment option for relapsed myeloma patients.”

In addition to meeting the primary endpoint, the KdD combination demonstrated efficacy in key secondary endpoints, including overall response rate (ORR), minimal residual disease (MRD) negative-complete response at 12 months and overall survival (OS). The ORR was 84.3% versus 74.7% (p=0.0040), and the rate of complete response or better was 28.5% versus 10.4% for the KdD and Kd arms, respectively. The analysis found the MRD-negative complete response rate at 12 months was 12.5% for KdD versus 1.3% for Kd (p<0.0001), a nearly 10-times higher response rate versus Kd-treated patients. The median OS was not reached in either group (HR=0.75; 95% CI: 0.49, 1.13; p=0.08).

https://www.biospace.com/article/releases/amgen-data-from-phase-3-candor-study-combining-kyprolis-carfilzomib-and-darzalex-daratumumab-to-be-presented-during-late-breaking-session-at-american-society-of-hematology-annual-meeting/

Cantel Medical Q1 revenues up 14%

Cantel Medical (CMD) Q1 results: Revenues: $257.2M (+14.0%).

Net Income: $5.8M (-69.8%); EPS: $0.14 (-69.6%); non-GAAP Net Income: $27.2M (+5.0%); non-GAAP EPS: $0.65 (+4.8%); CF Ops: $8.9M (-72.4%).

Previously: Cantel Medical EPS beats by $0.06, beats on revenue (Dec. 10)

https://seekingalpha.com/news/3525055-cantel-medical-q1-revenues-up-14