Tuesday, September 25, 2018
Jefferies boosts Amarin target to $15, sees ‘meaningful potential’ for buyout
Jefferies analyst Roger Song raised his price target for Amarin to $15 saying the 25% MACE reduction in the REDUCE-IT “clearly gives” the company an opportunity to grow sales to $2B-$3B. His model now assumes peak adjusted sales of $3B and he keeps a Buy rating on Amarin. Song thinks the shares “have room to climb higher” after yesterday’s 300% rally. The analyst also sees “meaningful potential” for a takeover event since Amarin is now “significantly de-risked.”
https://thefly.com/landingPageNews.php?id=2795083
Galapagos starts global Phase 2 for osteoarthritis med
Galapagos NV (Euronext & NASDAQ:GLPG) reports first dosing in the global ROCCELLA Phase 2 trial with GLPG1972/S201086 in knee osteoarthritis patients. Galapagos receives a 9 million milestone payment from its collaboration partner Servierfor this achievement.
ROCCELLA is a multiregional, randomized, double-blind, placebo-controlled, dose ranging trial evaluating the efficacy and safety of three different once-daily doses of GLPG1972/S201086 in patients with knee osteoarthritis (OA). ROCCELLA is planned to recruit approximately 850 patients in countries in Europe, Asia, North America and South America. Galapagos is responsible for ROCCELLA in the United States, where 300 patients are targeted to be recruited. Servier will run the trial in all other countries.
The primary objective of ROCCELLA is to demonstrate the efficacy of at least one dose of GLPG1972/S201086 compared to placebo in reducing cartilage loss after 52 weeks of treatment. This cartilage loss will be measured precisely by magnetic resonance imaging (MRI). Secondary objectives include safety and tolerability, several additional measures of structural progression, improvement in pain, function, stiffness, and patient global assessment.
GLPG1972/S201086 is a disease-modifying osteoarthritis drug (DMOAD) candidate that, in two animal models, has been shown to efficiently target a cartilage degrading enzyme called ADAMTS-5. A Phase 1 trial in healthy volunteers met all of its safety and pharmacokinetic targets and also demonstrated that GLPG1972/S201086 reduced the blood level of the ARGS neoepitope by approximately 50% within two weeks. ARGS neoepitope is a biomarker for ADAMTS-5 activity and, as such, serves as a reflection of cartilage breakdown. In a more recent Phase 1b trial in OA patients in the United States, similar findings were seen over a four-week period. Specifically, GLPG1972/S201086 was well tolerated and reduced ARGS neoepitope blood levels by up to 50%.
OA is a highly prevalent and disabling pathology. There are no treatments available today that counteract disease progression. Patients are left with only symptomatic treatments. As a result, OA represents an important unmet medical need. Galapagos developed investigational molecule GLPG1972/S201086 with the potential of becoming a first-in-class DMOAD as part of a collaboration with Servier that began in 2010. Galapagos has full U.S. commercial rights to GLPG1972/S201086 and is eligible to receive development, regulatory and other milestone payments plus royalties from Servier upon commercialization outside the United States.
Reata Has Positive Phase 2 Data for Nephropathy, Kidney Disease Med
Statistically Significant Improvement in Kidney Function Observed in Both Diseases After 12 Weeks of Treatment
Conference Call with Management Scheduled for Today at 8:00 am ET
Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage biopharmaceutical company, today announced positive, final results from the IgA nephropathy and type 1 diabetic chronic kidney disease (T1D CKD) cohorts of PHOENIX, a Phase 2 study of bardoxolone methyl (bardoxolone) in patients with rare forms of CKD. Compared to baseline, bardoxolone significantly improved kidney function as measured by patients’ estimated glomerular filtration rate (eGFR) at Week 12 in both cohorts, which was the primary endpoint of the PHOENIX study.
In the IgA nephropathy cohort of PHOENIX, patients treated with bardoxolone experienced a significant increase in eGFR of 8.0 mL/min/1.73 m2 (n=26; p<0.0001) at Week 12 compared to baseline. Reata collected historical eGFR data for 23 of these patients, which demonstrated that these patients’ kidney function was declining at an average annual rate of 1.2 mL/min/1.73 m2 prior to study entry. The observed 8.0 mL/min/1.73 m2 improvement after 12 weeks of treatment with bardoxolone represents a recovery of approximately six years of average eGFR loss.
In the T1D CKD cohort of PHOENIX, patients treated with bardoxolone experienced a significant increase in eGFR of 5.5 mL/min/1.73 m2 (n=28; p=0.02) at Week 12 compared to baseline. Reata collected historical eGFR data for 22 of these patients, which demonstrated that these patients’ kidney function was declining at an average annual rate of 1.9 mL/min/1.73 m2 prior to study entry. The observed 5.5 mL/min/1.73 m2 improvement after 12 weeks of treatment with bardoxolone represents a recovery of approximately three years of average eGFR loss.
With respect to safety, no treatment-related serious adverse events were reported in either cohort, and the reported adverse events were generally mild to moderate in intensity.
“With these data, bardoxolone has improved kidney function in multiple rare forms of CKD, including Alport syndrome, autosomal dominant polycystic kidney disease, IgA nephropathy, and type 1 diabetic CKD,” said Colin Meyer, M.D., Reata’s Chief Medical Officer. “The absence of drug-related serious adverse events and the eGFR improvements observed in the rare forms of CKD that we have studied suggest that bardoxolone has the potential to become an effective therapy for multiple rare forms of CKD.”
Reata management will host a call to discuss these results today, September 25th, 2018 at 8:00 a.m. ET.
| CONFERENCE CALL INFORMATION | |
| Date: | Tuesday, September 25th, 2018 |
| Time: | 8:00 a.m. ET |
| Audience Dial-in (toll-free): | (844) 348-3946 |
| Audience Dial-in (international): | (213) 358-0892 |
| Passcode: | 9899458 |
| Webcast Link: | https://edge.media-server.com/m6/p/qos423s5 |
Cerecor to acquire Ichorion Therapeutics in $26.6M transaction
Cerecor announced that it has agreed to acquire Ichorion Therapeutics. Ichorion is a privately-held biopharmaceutical company focused on developing treatments and increasing awareness of inherited metabolic disorders known as Inborn Errors of Metabolism. The terms of the agreement include the issuance of approximately 5,800,000 shares of Cerecor common stock at closing, subject to an end of 2019 lock-up, and development milestones worth up to an additional $15M, payable either in Cerecor stock or in cash in certain circumstances. The transaction was unanimously approved by the Board of Directors of both Cerecor and Ichorion and is expected to close later today.
https://thefly.com/landingPageNews.php?id=2794871
Amgen-Astellas BLINCYTO OKd In Japan For Acute Lymphoblastic Leukemia
First Approved Oncology Treatment From Amgen Astellas Joint Venture
BLINCYTO is the First-and-Only Approved CD19-Directed CD3 Bispecific T Cell Engager (BiTE®) Immunotherapy
Amgen (NASDAQ:AMGN) today announced that the Japanese Ministry of Health, Labour and Welfare has granted marketing approval for BLINCYTO® (blinatumomab) for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia (ALL). BLINCYTO was developed in Japan by Amgen Astellas BioPharma K.K. (AABP), a joint venture between Amgen and Astellas Pharma Inc., a pharmaceutical company headquartered in Tokyo.
“As proof-of-concept for our bispecific T cell engager technology, BLINCYTO has laid the groundwork for Amgen to deliver on our passion of addressing cancer by exploring numerous biologic pathways and therapeutic modalities,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “This innovation is a good example of how we provide new options to patients with serious illnesses like cancer. In bringing BLINCYTO to Japanese patients, we reinforce our commitment to deliver novel cancer therapies on behalf of patients worldwide.”
BLINCYTO is the first-and-only bispecific T cell engager (BiTE®) immunotherapy construct approved globally. It is also the first approved immunotherapy from Amgen’s BiTE® platform, an innovative approach that helps the body’s immune system target cancer cells.
“Today’s approval of BLINCYTO marks a significant milestone that reinforces our commitment to addressing unmet medical needs of patients in Japan,” said Steve Sugino, president and representative director, AABP. “As our first oncology treatment approved in the region, we are proud to provide a much-needed innovative treatment option for adults and children with relapsed or refractory B-cell ALL, one of the most aggressive B-cell malignancies.”
Hitoshi Kiyoi, M.D., Ph.D., professor of internal medicine, Hematology and Oncology, Nagoya University Graduate School of Medicine said, “The standard therapy for relapsed or refractory B-cell ALL has not been established in Japan and therefore different chemotherapy regimens have been selected, depending on the condition and background of each patient. BLINCYTO is a much-needed and important new treatment option for patients with relapsed or refractory B-cell ALL, as demonstrated by the efficacy and survival benefit seen in the TOWER study.”
The approval is based on data from multiple global studies, including the Phase 3 TOWER study and Japan Phase 1b/2 Horai study. In the TOWER study, BLINCYTO demonstrated a superior improvement in median overall survival (OS) versus standard of care (SOC) chemotherapy. Median OS was 7.7 months (95 percent CI: 5.6, 9.6) for BLINCYTO versus 4.0 months (95 percent CI: 2.9, 5.3) for SOC (HR for death=0.71; p=0.012). Safety results among subjects who received BLINCYTO were comparable to those seen in the previous Phase 2 studies of BLINCYTO in adult patients with Philadelphia chromosome-negative (Ph-) relapsed or refractory B-cell precursor ALL. In the TOWER study, major adverse reactions were pyrexia (39.0 percent), decrease in white blood cell count (14.6 percent), cytokine release syndrome (13.5 percent), febrile neutropenia (10.9 percent), headache (10.1 percent), elevated liver enzyme (10.1 percent) and decrease in platelet count (10.1 percent). In the Phase 1b/2 Horai study, BLINCYTO was administered to 35 Japanese adult and pediatric patients with relapsed or refractory B-cell precursor ALL. The safety results from the Horai study were comparable to those seen in the global studies, including TOWER. In the Horai study, major adverse reactions in adult patients were cytokine release syndrome (46.2 percent), pyrexia (46.2 percent), decrease in white blood cell count (38.5 percent) and decrease in platelet count (34.6 percent), and major adverse reactions in pediatric patients were elevated liver enzyme (66.7 percent), pyrexia (66.7 percent), cytokine release syndrome (55.6 percent) and abdominal pain (44.4 percent).
BLINCYTO is now approved in 57 countries, including the United States (U.S.), all member countries in the European Union (EU) and the European Economic Area, Canada and Australia.
Pharma Firms React to Employers, Plans Excluding New Therapies Based on Cost
The high cost of prescription medications has been a common subject of discussion in the news as of late. Many studies have been done regarding this, including one that shows that, on average, the US pays more than 10 other wealthy nations combined for medication.
There have been a lot of ideas proposed to help combat these costs, but few can agree on what plan would work best. Recently, however, a few companies have decided to enact their own plans that they believe will help bring down the cost of new therapies.
Insurance companies’ New Programs
CVS Caremark has launched a new program that will allow its clients to exclude any medication that launches at a price greater than $100,000 per quality-adjusted life-year (QALY), which is a benchmark created by the Institute for Clinical and Economic Review (ICER). This is intended to force pharmaceutical companies to drop the price of their medications by denying patients from purchasing them if they cost more than the threshold CVS has set.
But CVS isn’t the only one attempting to implement new policies to try and bring drug prices down. New York state’s Medicaid program has decided to exclude a new cystic fibrosis medication, citing that it costs too much for how effective it is. The drug in question, Orkambi, costs $272,000 a year but hasn’t shown to be very effective at helping patients.
Other companies like Express Scripts are following suit as well, using their own exclusionary plans that they hope will force pharma companies to give them better prices.
Whether or not these new programs will have the desired effect remains to be seen and with all of these health plans enacting these new programs, pharmaceutical companies have begun asserting their positions on the topic.
Reactions from Pharma Companies
Not long after CVS and the state of New York made their announcements for their new programs, Dr. Robert W. Dubois, the chief science officer and executive vice president of the National Pharmaceutical Council, spoke out against them.
Much of Dr. Dubois’s argument against these kinds of programs hinges on the idea that patients won’t be able to access the medication that they require. He also believes that this plan focuses too much on cost-effectiveness when deciding the value of a new therapy.
According to Dubois, to fully determine the correct value of a medication, many other considerations need to be taken into account. A few of the examples he gave include the medication’s ability to treat illnesses that were previously insufficiently treated, the possibility of a cure for the illness and the hope that it creates, and the ease of the regimen compared to other treatments.
Dubois also criticizes CVS’s implementation of the plan, noting that ICER’s framework has a variable economic threshold while CVS will be using a single threshold set at $100,000. He believes that such a dichotomous decision is a mistake and will end up severely limiting the kinds of new therapies patients have access to as, under CVS’s plan, a medication that is even slightly above their set threshold will be denied.
After Dr. Dubois made his statement against CVS and New York State’s new plans, CVS wrote their own article to fire back at him and the pharmaceutical companies he speaks for. They start off by pointing out that Dubois represents various pharmaceutical companies and, thus, his words should be seen in that light. They follow that up by defending their usage of ICER’s benchmark and how they’ve implemented their new plan.
If one is to take Dr. Dubois’s words as representative of the pharmaceutical industry as a whole, then it would seem as though pharma companies are very much against these new plans that companies like CVS are implementing.
However, not all pharma companies are completely against adjusting the cost of medications according to ICER’s benchmarks. Both Sanofi and Regeneron have announced that they will make their cholesterol medication more affordable to be in line with ICER’s analysis. But it remains to be seen if they will follow this plan for more medications later on.
In Conclusion
It would appear that pharmaceutical companies are still divided on the best method to go about lowering the cost of medications for patients. Insurance companies and other healthcare programs are also uncertain about the best way to force pharma companies to lower their prices, with each one attempting to enact their own plans to solve this issue.
The one thing that they all seem to agree on, however, is that the costs of new medications are currently too high for most patients and something needs to be done about it.
Tesaro Touts Maintenance Therapy Zejula During Ovarian Cancer Awareness Month
In 2015 Anne was diagnosed with stage III ovarian cancer. The diagnosis was made after she wound up in the emergency room complaining of severe back pain.
The diagnosis came as a complete shock and Anne immediately began a treatment regimen that included surgery. Months later she relapsed and was forced to continue treatment. Typically, when a woman has a recurrence of ovarian cancer, it is considered incurable. But, last year, Anne was told she was a good candidate for a newly-approved maintenance therapy that could halt or delay progression of the disease – Tesaro’s Zejula.
Formerly known as Niraparib, Zejula is an oral, once-daily PARP inhibitor used as maintenance treatment for ovarian cancer. It was the first PARP inhibitor to be approved by the U.S. Food and Drug Administration (FDA) that does not require BRCA mutation or other biomarker testing.
Since being placed on Zejula, Anne said she has something of a new lease on life. Her illness is under control and she’s been able to return to work – something she did not think was going to be possible.
Anne, along with Dr. Robert Holloway, the medical director of the Gynecologic Oncology Program at Florida Hospital, was part of an ovarian cancer awareness media blitz Tesaro conducted to highlight National Ovarian Cancer Awareness month.
Speaking to BioSpace, Holloway said there are a number of new treatment options for women with ovarian cancer that are allowing them to live longer following initial diagnosis. Holloway specifically pointed to Tesaro’s Zejula and said the once-per-day therapy is helping women maintain remission and preventing recurrence of the disease. Until Zejula, Holloway said, many patients had to go through a “wait and see” approach to their treatment. About 85 percent of patients went through a recurrence or relapse of disease, which required more chemotherapy or other treatments, Holloway said. When Zejula was approved last year though, ovarian cancer patients were able to benefit from the maintenance therapy and keep their cancers at bay.
“This is something that really benefits patients in the long run,” Holloway said.
Ovarian cancer is the fifth leading cause of cancer-related deaths in the United States. It is estimated that this year there will be more than 22,000 women in the U.S. diagnosed with the disease and about 14,000 disease-related deaths. The vast majority of diagnoses, about 80 percent, are found at an advanced stage.
Since the approval of Zejula last year, there has been significant movement in the treatment of ovarian cancer. The following are a few highlights from select company pipelines.
In June, Genentech received the green light from the U.S. Food and Drug Administration for Avastin (bevacizumab) as a treatment for stage III or stage IV ovarian cancer. The FDA approved Avastin in combination with chemotherapy followed by Avastin alone for the treatment of those cancer patients who have undergone initial surgical resection. According to the trial data that led to the regulatory approval, women who received Avastin in combination with chemotherapy, and continued use of Avastin alone, had a median progression-free survival of 18.2 months compared to 12 months in women who received chemotherapy alone.
Also in June, AstraZeneca and Merck revealed positive Phase III results from a trial testing Lynparza (olaparib) in BRCA-mutated (BRCAm) advanced ovarian cancer. Lynparza, also a PARP inhibitor, is being studied as a first-line maintenance therapy. The data showed patients treated with Lynparza demonstrated statistically significant and clinically meaningful improvement in progression-free survival compared to those receiving placebo. Lynparza is also being tested in another Phase III trial in combination with bevacizumab as first-line maintenance therapy for women with newly diagnosed advanced ovarian cancer, regardless of BRCA status. Data from that trial is expected in 2019.
In April West Lafayette, Indiana-based On Target Laboratories dosed the first patient in the Phase III trial of OTL38 in detecting ovarian cancer. As BioSpace previously reported, OTL38 is a ligand that targets folate receptors that appear in various types of cancers. OTL38 is injected into patients and includes an infrared dye that allows surgeons to visually identify cancer cells that can guide them in removing the cancer tissue.
Massachusetts-based ImmunoGen in March announced positive results from its FORWARD IItrial that showed the combination of mirvetuximab soravtansine and Merck’s Keytruda yielded positive results in platinum-resistant epithelial ovarian cancer. Then in June, the FDA granted Fast Track designation to Immunogen’s mirvetuximab soravtansine for patients with medium to high folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer who received at least one, but no more than three prior systemic treatment regimens. On its own, mirvetuximab soravtansine is being evaluated in a Phase III trial as a stand-alone treatment.
While Holloway noted there has been a significant improvement in treatment options for ovarian cancer patients, he said little headway has been made in the diagnosis of the disease. There has been some new understanding that there is a genetic component to ovarian cancer, which means women who have a family member diagnosed with the disease should proactively work with their doctors to watch for signs and symptoms of the disease.
“We’re hopeful for a blood test or other kind of test, but that’s not been brought forth yet,” Holloway said.
Subscribe to:
Posts (Atom)