LGND partner SAGE Therapeutics reports positive Brexanolone PII trial results in post-partum depression: robust efficacy and safety demonstrated
Lancet. 2017 Jun 12. pii: S0140-6736(17)31264-3. doi: 10.1016/S0140-6736(17)31264-3. [Epub ahead of print] Brexanolone (SAGE-547 injection) in post-partum depression: a randomised controlled trial.
Kanes S1, Colquhoun H1, Gunduz-Bruce H1, Raines S2, Arnold R1, Schacterle A1, Doherty J1, Epperson CN3, Deligiannidis KM4, Riesenberg R5, Hoffmann E1, Rubinow D6, Jonas J1, Paul S1, Meltzer-Brody S7. Author information 1 Sage Therapeutics Inc, Cambridge, MA, USA. 2 2b Analytics, Wallingford, PA, USA. 3 Department of Psychiatry, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA. 4 University of Massachusetts Medical School, Worcester, MA, USA; Women's Behavioral Health, Zucker Hillside Hospital, New York, NY, USA. 5 Atlanta Center for Medical Research, Atlanta, GA, USA. 6 Department of Psychiatry, UNC School of Medicine, Chapel Hill, NC, USA. 7 Department of Psychiatry, UNC School of Medicine, Chapel Hill, NC, USA. Electronic address: firstname.lastname@example.org. Abstract BACKGROUND: Post-partum depression is a serious mood disorder in women that might be triggered by peripartum fluctuations in reproductive hormones. This phase 2 study investigated brexanolone (USAN; formerly SAGE-547 injection), an intravenous formulation of allopregnanolone, a positive allosteric modulator of γ-aminobutyric acid (GABAA) receptors, for the treatment of post-partum depression. METHODS: For this double-blind, randomised, placebo-controlled trial, we enrolled self-referred or physician-referred female inpatients (≤6 months post partum) with severe post-partum depression (Hamilton Rating Scale for Depression [HAM-D] total score ≥26) in four hospitals in the USA. Eligible women were randomly assigned (1:1), via a computer-generated randomisation program, to receive either a single, continuous intravenous dose of brexanolone or placebo for 60 h. Patients and investigators were masked to treatment assignments. The primary efficacy endpoint was the change from baseline in the 17-item HAM-D total score at 60 h, assessed in all randomised patients who started infusion of study drug or placebo and who had a completed baseline HAM-D assessment and at least one post-baseline HAM-D assessment. Patients were followed up until day 30. This trial is registered with ClinicalTrials.gov, number NCT02614547. FINDINGS: This trial was done between Dec 15, 2015 (first enrolment), and May 19, 2016 (final visit of the last enrolled patient). 21 women were randomly assigned to the brexanolone (n=10) and placebo (n=11) groups. At 60 h, mean reduction in HAM-D total score from baseline was 21·0 points (SE 2·9) in the brexanolone group compared with 8·8 points (SE 2·8) in the placebo group (difference -12·2, 95% CI -20·77 to -3·67; p=0·0075; effect size 1·2). No deaths, serious adverse events, or discontinuations because of adverse events were reported in either group. Four of ten patients in the brexanolone group had adverse events compared with eight of 11 in the placebo group. The most frequently reported adverse events in the brexanolone group were dizziness (two patients in the brexanolone group vs three patients in the placebo group) and somnolence (two vs none). Moderate treatment-emergent adverse events were reported in two patients in the brexanolone group (sinus tachycardia, n=1; somnolence, n=1) and in two patients in the placebo group (infusion site pain, n=1; tension headache, n=1); one patient in the placebo group had a severe treatment-emergent adverse event (insomnia). INTERPRETATION: In women with severe post-partum depression, infusion of brexanolone resulted in a significant and clinically meaningful reduction in HAM-D total score, compared with placebo. Our results support the rationale for targeting synaptic and extrasynaptic GABAA receptors in the development of therapies for patients with post-partum depression. A pivotal clinical programme for the investigation of brexanolone in patients with post-partum depression is in progress. FUNDING: Sage Therapeutics, Inc.
Announced today--Kyprolis approved by NICE for use in the UK. Another win by the Amgen team which will open an important door for growth. It is great news and a testament that Amgen is a partner who can perform well.
CHAMPION-2 trial results published; Carfilzomib in combination with Cyclophosphamide and Dexamethasone demonstrates robust efficacy in newly diagnosed multiple myeloma pts:
Clin Lymphoma Myeloma Leuk. 2017 May 10. pii: S2152-2650(16)30910-7. doi: 10.1016/j.clml.2017.05.009. [Epub ahead of print] A Multicenter, Open-Label, Phase 1b Study of Carfilzomib, Cyclophosphamide, and Dexamethasone in Newly Diagnosed Multiple Myeloma Patients (CHAMPION-2).
Boccia RV1, Bessudo A2, Agajanian R3, Conkling P4, Harb W5, Yang H6, Pinchasik D6, Kimball AS6, Berenson JR7. Author information 1 Center for Cancer and Blood Disorders, Bethesda, MD. Electronic address: RBoccia@ccbdmd.com. 2 Cancer Care Associates for Research and Excellence, Encinitas, CA. 3 The Oncology Institute of Hope and Innovation, Downey, CA. 4 US Oncology Research, Virginia Oncology Associates, Norfolk, VA. 5 Horizon Oncology Center, Lafayette, IN. 6 Amgen Inc, Thousand Oaks, CA. 7 Institute for Myeloma & Bone Cancer Research, West Hollywood, CA. Abstract INTRODUCTION: This phase 1b study evaluated the safety and efficacy of 3 dose levels of carfilzomib when provided with fixed dose oral cyclophosphamide and dexamethasone (KCyd) in patients with newly diagnosed multiple myeloma (MM). PATIENTS AND METHODS: CHAMPION-2 was a multicenter single-arm study. Patients with newly diagnosed secretory MM were enrolled and received KCyd treatment for up to 8 cycles. A 3 + 3 dose escalation scheme was used to evaluate twice-weekly carfilzomib at 36, 45, and 56 mg/m2 dose levels, followed by a dose expansion. RESULTS: No dose-limiting toxicities were observed in any of the dose evaluation cohorts. The KCyd regimen that included the maximum planned carfilzomib dose of 56 mg/m2 twice weekly was brought forward into dose expansion. A total of 16 patients were treated at this dose level. At 56 mg/m2 the overall response rate was 87.5% (95% confidence interval, 61.7-98.4), and the median time to response of 14 patients whose disease responded to therapy was 1 month. At this dose level, common adverse events of grade 3 or higher were anemia (25.0%), neutropenia (18.8%), acute kidney injury (12.5%), and decreased white blood cell count (12.5%). Ten of 16 patients who received carfilzomib at 56 mg/m2 completed all 8 cycles, 5 patients discontinued study therapy before cycle 8 as a result of adverse events, and 1 patient discontinued therapy as a result of progressive disease. CONCLUSION: Carfilzomib in combination with cyclophosphamide and dexamethasone is effective and has manageable toxicity for patients with newly diagnosed MM.
Daratumumab (DARA) in combination with carfilzomib, lenalidomide, and dexamethasone (KRd) in patients (pts) with newly diagnosed multiple myeloma (MMY1001): An open-label, phase 1b study. | 2017 ASCO Annual Meeting Abstracts
8000 Daratumumab (DARA) in combination with carfilzomib, lenalidomide, and dexamethasone (KRd) in patients (pts) with newly diagnosed multiple myeloma (MMY1001): An open-label, phase 1b study. Andrzej J. Jakubowiak, Ajai Chari, Sagar Lonial, Brendan
Ligand year to year comparisons will look much better when they are on the same tax basis. Picking up royalties a little sooner should also help. Its hard for me to see any negatives with this stock, but the low volume still bothers me. I expect thw price will explode one of these days. Ligands price movement today was not any more than market reaction. It was not worthy of the new.
LGND partner Novartis highlights Promacta at investor meeting:
SAN DIEGO--(BUSINESS WIRE)--Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) announces it will receive $10 million from a contractually specified royalty rate buy-down following the sale by CorMatrix Cardiovascular, Inc. of rights to its commercial pericardial repair and CanGaroo® Envelope extracellular matrix (ECM) products to Aziyo Biologics. In May 2016, Ligand acquired rights to royalties on these products and several pipeline ECM programs from CorMatrix for $17.5 million.
“The royalty transaction entered into with CorMatrix last year has been successful in generating cash and has contributed quality shots on goal to our portfolio. CorMatrix made a strategic shift to focus on its pipeline assets, and we are encouraged by the experience and marketing strength of Aziyo’s management team” Tweet this Under the terms of the original transaction, Ligand was entitled to a $10 million payment if, upon acquisition, the new owner elected to buy-down the royalty rate. As a result of this transaction, Ligand will receive a 5% royalty on these commercial products from Aziyo, down from the original 20% royalty with CorMatrix. As part of the royalty rate reduction, Aziyo has agreed to pay Ligand up to $10 million of additional sales-based milestones tied to the commercial success of the currently-marketed products and to extend the term on these royalties by one year. Ligand’s rights to potential royalties from CorMatrix upon the successful development of its remaining pipeline ECM programs remain unchanged.
“The royalty transaction entered into with CorMatrix last year has been successful in generating cash and has contributed quality shots on goal to our portfolio. CorMatrix made a strategic shift to focus on its pipeline assets, and we are encouraged by the experience and marketing strength of Aziyo’s management team,” said John Higgins, Chief Executive Officer. “This transaction repays the majority of the initial royalty purchase price, adds $10 million of new potential milestones and extends the term for royalties to be earned from the underlying ECM products. Ligand also retains rights to royalties on pipeline products being developed by CorMatrix. Overall, this transaction improves the value of the deal and near-term cash generation.”
As a result of this transaction, Ligand will book an estimated $2 million of additional revenue in 2017, net of offsetting non-cash accounting expenses. Previous net revenue guidance for 2017 was at least $130 million, and Ligand now expects 2017 net revenue to increase to at least $132 million. The additional revenue is expected to be classified as contract revenue and royalty revenue is expected to be slightly lower for the full year due to the lower royalty rate on CorMatrix products beginning with the second half of 2017. Additional upside in contract revenue guidance is reduced from $24 million to $14 million, reflecting the revenue from the royalty rate buy-down and associated amortization.
This new revenue guidance implies a minimum adjusted diluted EPS for 2017 of $2.90, increased from original guidance of at least $2.70.Thanks
seems like mainly reporting deals where there is shared income.?
buying AXSM at $4 is like buying JAZZ at $1 a few years ago now trading at $151..PLEASE READ THIS ... (AXSM) Market Cap $90 M/Cash $55 M / 5x BIG Phase 3 programs in various indications targeting large Markets with first results in Q3 2017 =20 BAGGER ..FANTASTIC OPPORTUNITY !
Undiscovered and massive undervalued Biotech Stock with lots of Big News on the way.This Stock is brutally undervalued with a Market cap of just $90 million and $55 million in cash .Founder and Ceo is the larget shareholder holding over 7 million shares (30%) more than 50% of O/S is owned by Insider and Institutions which is a great sign .
This undiscovered stock could be the next 10 bagger gem if just one of their 5 ongoing Phase 3 programs is successful .GL
Market-Cap: $90 Million Cash: $55 Million(cash runway into the first quarter of 2019.) Price:$3.90
Shares Out: 23.5 Million
Aegis Capital has reiterated a ‘Buy’ rating and price target of $20 on Axsome Therapeutics (NASDAQ: AXSM) after the company reported financial results for the quarter ended March 31, 2017. The analyst noted that although Axsome reported revenue in-line with consensus estimates, the focus remains on upcoming catalysts such as the company’s ongoing clinical trials. Since February 2017, the company’s AXS-05 has received fast track designation from the FDA for the treatment of Alzheimer’s disease (AD) agitation and treatment resistant depression (TRD). Topline data from the TRD treatment study is expected in the first quarter of 2018. Additionally, Axsome is developing AXS-02 in three separate phase 3 clinical programs, including chronic low back pain associated with modic changes and complex regional pain syndrome, with interim data expected in the fourth quarter of 2017, as well as knee osteoarthritis associated with bone marrow lesions, with interim data expected in the third quarter of 2017.
New Captisol program with International Analgesix Inc., a private firm. It has an upfront payment, milestones and a royalty of 5-10% of net sales.
5/24/17 MarketRealist report: Kyprolis to drive revenue growth at AMGN in 2017:
Report: positive PII trial results with Melphalan/Lenalidomide/Dexamethasone in newly diagnosed AL amyloidosis patients:
Haematologica. 2017 May 18. pii: haematol.2016.163246. doi: 10.3324/haematol.2016.163246. [Epub ahead of print] Lenalidomide/Melphalan/Dexamethasone in newly diagnosed patients with AL Amyloidosis: results of a prospective phase 2 study with long-term follow-up.
Hegenbart U1, Bochtler T1, Benner A2, Becker N2, Kimmich C1, Kristen AV3, Beimler J4, Hund E5, Zorn M6, Freiberger A7, Gawlik M8, Goldschmidt H9, Hose D9, Jauch A10, Ho AD8, Schönland SO11. Author information Abstract Chemotherapy in light chain amyloidosis aims to normalize the involved free light chain in serum, which leads to an improvement or at least stabilization of organ function in most responding patients. We performed a prospective single center phase 2 trial with 50 untreated patients not eligible for high-dose treatment. The treatment schedule comprised 6 cycles of oral lenalidomide, melphalan and dexamethasone every 4 weeks. After 6 months complete remission was achieved in 9 patients (18%), very good partial remission in 16 (32%) and partial response in 9 (18%). Overall, organ response was observed in 24 patients (48%). Hematologic and cardiac toxicities were predominant adverse events. Mortality at 3 months was low with 4% (n=2) despite inclusion of 36% of patients (n=18) with cardiac stage Mayo 3. After a median follow-up of 50 months, median overall and event-free survival were 67.5 months and 25.1 months, respectively. We conclude that the treatment of lenalidomide, melphalan and dexamethasone is very effective to achieve a hematologic remission, organ response and consecutively a long survival in transplant ineligible patients with light-chain amyloidosis. However, as toxicity and tolerability are the major problems of 3 drug regimens a strict surveillance program is necessary and sufficient to avoid severe toxicities. This study is registered at www.clinicaltrials.gov as #NCT00883623 (Eudract2008-001405-41).
Ligand to Participate in Two Upcoming Investor Conferences
Business Wire Business WireMay 22, 2017 SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (LGND) announces that company executives are scheduled to participate in the following upcoming investor conferences: 14th Annual Craig-Hallum Institutional Investor Conference in Minneapolis. Conference takes place on Wednesday, May 31, 2017 with one-on-one meetings only. John Higgins, CEO; Matt Foehr, President and COO; and Matt Korenberg, CFO, will attend for Ligand. Jefferies Healthcare Conference in New York City. Presentation takes place on Thursday, June 8, 2017 at 4:00 p.m. Eastern time (1:00 p.m. Pacific time). John Higgins, CEO; Matt Foehr, President and COO; and Matt Korenberg, CFO, will attend for Ligand. A live webcast of the presentation will be available on Ligand’s website at www.ligand.com. A replay of the presentation will be archived on the website for 30 days.
Ligand Pharmaceuticals, Inc. (LGND)
I am not an accountant. I do not think enough effort was made on the last two calls to emphasize the changed accounting and the phantom impact of the change to the EPS. Or, to think about it differently, to emphasize the opportunity of having such a large cash flow. I'm sure they are looking at more acquisitions.
Ligand is unique, and the management team is conservative in its messaging. Getting the messaging right is hard. They don't want to forecast events that are uncertain. They are trying to do more. For example, they are talking about how many revenue-generating programs they are likely to have in 2020. But, they aren't giving any guidance to how those successes impact Captisol material sales, for example.
Bottom line - They have 155 funded programs. They have 90+ partners and stupendous deal flow. They have key IP. They have an internal GRA program with a novel mechanism of action in diabetes. This compound, if successful in Phase II and III, is a future blockbuster that can do billions in annual sales. They have an uber lean SG&A structure. No other companies have what Ligand has. Patience.