There are no major FDA or trial data releases scheduled for Thanksgiving week. This is a shorter trading week with markets closed on Thursday, November 22, 2018, and opened half-day on Friday, November 23, 2018.
There are some events that we are waiting for in Q4 2018. We don’t know when these data releases will come. They could come next week, next month, or even pushed into Q1 2019. Below are the Q4 2018 events.
Achillion Pharmaceuticals (ACHN) ACH-4471 for paroxysmal nocturnal hemoglobinuria (PNH). Phase 2 interim data due December 17, 2018.
ACH-4471, Complement Factor D Inhibitor for PNH and C3G
Achillion’s first generation oral factor D inhibitor, ACH-4471 is being evaluated for safety and efficacy with Phase 2 clinical programs in both paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G).
The PNH program consists of two trials: A Phase 2 clinical trial in untreated PNH patients where ACH-4471 is being assessed as a monotherapy; the second trial is Phase 2 clinical trial evaluating ACH-4471 in patients who are inadequately controlled or sub-optimally responding to eculizumab, which is a therapy for patients with PNH.
The C3G program consists of three currently recruiting Phase 2 clinical trials: a 14-day biomarker study, a six-month blinded, placebo-controlled study, and a 12-month open label study.
More information is available at http://www.achillion.com/patients-and-clinicians/
“In the third quarter we completed enrollment in our Phase 2 PNH monotherapy trial of ACH-4471, initiated ten additional clinical trial sites to support our C3G development program, and commenced development of a comprehensive portfolio strategy that we plan to communicate to investors in December 2018. We also plan to provide interim data on all open-label ACH-4471 clinical trials as well as Phase 1 safety and pharmacokinetics interim data from the single ascending dose trials with our second-generation factor D compounds, ACH-5228 and ACH-5548,” Truitt noted. “We believe our innovative and differentiated factor D inhibitors have the potential to address unmet medical needs in patients with PNH and C3G as well as additional complement mediated diseases, together representing significant value-creation opportunities.”
ALKS – Alkermes ALKS 3831 – ENLIGHTEN-2 for schizophrenia. Phase 3 data due 4Q 2018.
Topline results for ENLIGHTEN-2, a six-month weight study of ALKS 3831 compared to olanzapine in patients with stable schizophrenia in Q4 2018.
“Our solid results in the quarter were in-line with expectations, driven by the growth of our proprietary commercial products, the continued strength of our royalty and manufacturing business, and the important investments we are making in our late-stage pipeline and commercial organization,” commented James Frates, Chief Financial Officer of Alkermes. “Our diverse business is financially strong and we are well positioned to execute on our strategy to drive value and long-term growth. Based on our outlook for the remainder of the year, today we are raising our financial expectations for 2018, primarily driven by upside from AMPYRA® revenues.”
Celgene Corporation’s REVLIMID – ROBUST for first-line ABC Diffuse large B-cell lymphoma (DLBCL). Phase 3 data due 4Q 2018. You can read more about what Celgene has planned in this company PDF.
ContraFect’s CF-301 for serious infections caused byStaph aureus including MRSA. Phase 2 data due 4Q 2018.
“We made meaningful steps this past quarter in both the clinical and corporate sides of our business, including completing enrollment in our ongoing Phase 2 clinical trial of exebacase and securing funding that extends our cash runway into the first quarter of 2020,” said Steven C. Gilman, Ph.D., Chairman and Chief Executive Officer of ContraFect. “In addition to these key milestones, our R&D team presented data on our lysins at scientific meetings and on a panel at a recent FDA-sponsored workshop during the quarter. This progress across our business gives us strong momentum as we look towards topline data from our study of exebacase later this year.”
Exebacase (CF-301) is a recombinant bacteriophage-derived lysin with potent bactericidal activity against Staph aureus, a major cause of blood stream infections, or bacteremia. Exebacase has the potential to be a first-in-class treatment for Staph aureus bacteremia. It has a novel, rapid, and specific mechanism of bactericidal action against Staph aureus and does not impact the body’s natural bacterial flora. By targeting a conserved region of the cell wall that is vital to bacteria, resistance is less likely to develop to exebacase. Combinations of exebacase with standard of care antibiotics significantly increased bacterial killing and survival in animal models of disease when compared to treatment with antibiotics or exebacase alone. In addition, in vitro and in vivo experiments have shown that exebacase is highly active against biofilm infections. Exebacase was licensed from The Rockefeller University and is being developed at ContraFect. It is the first lysin to enter clinical studies in the U.S.
Catalyst Pharmaceutical’ Firdapse for Lambert-Easton Myasthenic Syndrome (LEMS). Refusal to file letter reported on February 17, 2016. NDA resubmitted with new PDUFA date under priority review of November 28, 2018.
Priority Review is granted by the FDA to drugs with the potential to address a serious condition and, if approved, would provide a significant improvement in safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of November 28, 2018. The submission is supported by positive results from two Phase 3 studies.
“We are delighted to have received Priority Review status for Firdapse for the treatment of LEMS and look forward to continuing to work closely with the FDA during the review process,” said Patrick J. McEnany, Chairman and CEO of Catalyst. “Together with the previous grant of Breakthrough Therapy Designation, the Priority Review underscores the robust potential of Firdapse and the need for a safe and effective FDA-approved treatment for LEMS.”
Evofem Biosciences’ Amphora for contraceptive vaginal gel. Phase 3 data due late-2018.
With the third quarter behind us and completion of Evofem’s Phase 3 clinical trial of Amphora® for the prevention of pregnancy (AMP002) rapidly approaching, I wanted to pause and take a moment to let you know how grateful we are for your ongoing support of Evofem. Our optimism and enthusiasm for our mission to develop and commercialize innovative products to address unmet needs in women’s sexual and reproductive health is unwavering.
Our research and development programs center on advancing Multipurpose Vaginal pH Regulator (MVP-R) product candidates for critical areas in women’s health where we believe current options are either insufficient or absent entirely.
We remain on track to complete AMP002 by year-end 2018. We over-enrolled this pivotal trial ahead of schedule due to strong demand; an indicator of this is that 500 additional women were in the screening progress when we closed enrollment in February 2018. The last patients in this trial have completed their last cycles and are in the process of completing their last office visits. This will enable us to lock our database and move forward with data analyses. We continue to expect top-line data from AMP002 by the end of this year.
Assuming positive data from AMP002, we plan to re-submit the Amphora New Drug Application (NDA) in the second quarter of 2019. Subject to timely FDA approval of this NDA, we will launch Amphora in January of 2020 as the first and only hormone-free, woman-controlled, on-demand birth control drug product in the U.S.
When and if Amphora is approved, we plan to focus initially on driving uptake of the product among the 16.5 million women in the U.S. who say they do not want to get pregnant, yet they are not doing anything to prevent pregnancy. According to the National Institutes of Health, these women have an 85% risk of becoming pregnant within one year if they continue this behavior. These statistics are staggering! “One size definitely does not fit all” when it comes to women and their bodies.
Equally important is the rise of sexually transmitted diseases (STDs) in our country. Under-reported STDs are in the national spotlight. In August 2018 the CDC released preliminary data showing that in 2017, reported cases of chlamydia, gonorrhea and syphilis increased for the fourth consecutive year. Amphora has vaginal pH regulating properties which may enable it to prevent the transmission of certain STDs including chlamydia and gonorrhea.
The standard of care for chlamydia and gonorrhea involves treatment with antibiotics. The current consensus in the medical community is that preventive measures are greatly needed, but there is no approved drug to prevent transmission of these vexing and rising STDs.
Evofem’s pioneering research in STD prevention includes our ongoing Phase 2b clinical trial evaluating Amphora to prevent urogenital transmission of chlamydia and gonorrhea in women.
FibroGen and Astrazeneca’s Roxadustat – ANDES for anaemia in Chronic Kidney Disease. Phase 3 completion of enrollment announced June 7, 2018 with data due 4Q 2018. Pooled MACE safety data 1H 2019.
“With new drug applications for roxadustat in anemia associated with chronic kidney disease supported by positive Phase 3 results and under review in China and Japan, we look forward to the upcoming reporting of topline clinical results, pooled safety data and submitting our U.S. NDA,” said Thomas B. Neff, FibroGen’s Chief Executive Officer. “For pamrevlumab, our proprietary anti-fibrotic and anti-fibroproliferative therapeutic candidate, we are initiating Phase 3 studies in idiopathic pulmonary fibrosis, and in unresectable locally advanced pancreatic cancer, in the first quarter of 2019.”
Recent Developments and Highlights
Roxadustat for Anemia in Chronic Kidney Disease (CKD) in the U.S./EU
- Topline Phase 3 clinical results anticipated for the fourth quarter of this year
- Pooled MACE analysis results and submission of New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) anticipated in the first half of 2019
- Astellas reported positive topline efficacy and safety results from the first global trial, ALPS, a Phase 3 placebo-controlled study evaluating roxadustat in non-dialysis-dependent CKD anemia patients in September 2018
- Astellas also completed its global trial, PYRENEES, a Phase 3 study in dialysis patients
Roxadustat for Anemia in CKD in China
- Roxadustat NDA approval for the treatment of dialysis-dependent CKD is anticipated by year-end 2018, followed by approval in CKD non dialysis
- Clinical results from two Phase 3 studies conducted in China were presented at the American Society of Nephrology (ASN) Kidney Week 2018 annual meeting in October
Roxadustat for Anemia in CKD in Japan
- Astellas NDA for roxadustat was filed with the Pharmaceuticals and Medical Devices Agency(PMDA) for anemia associated with dialysis-dependent CKD, triggering a $15 million milestone payment to FibroGen
- Astellas announced positive topline results from the four Phase 3 dialysis-dependent studies that support the Japan NDA
- Clinical results from two of the four Japan Phase 3 trials in dialysis-dependent patients were presented at the ASN Kidney Week 2018:
- A Phase 3 trial in peritoneal dialysis patients was presented in an oral session
- A Phase 3 darbepoetin alfa controlled study in stable hemodialysis patients previously treated with ESA was presented in a clinical late-breaking poster session
- One of two non-dialysis-dependent Phase 3 studies supporting approval for non-dialysis is now completed
Marinus Pharmaceuticals’ Ganaxolone for postpartum depression. Phase 2 data due 4Q 2018.
“This is an exciting time for Marinus,” commented Christopher M. Cashman, chairman and chief executive officer of Marinus. “With enrollment complete in our Magnolia study last quarter, we are on-track to report data shortly. This is our first data readout for ganaxolone in women suffering from postpartum depression, a devastating condition with few treatment options. We will learn how ganaxolone performs in this patient population at various doses and apply these learnings to ongoing and future development as we lay the foundation for later-stage studies.”
The Company has completed enrollment in the IV only portion of the Magnolia study, a Phase 2 double-blind, placebo-controlled, dose-optimization clinical trial to evaluate ganaxolone in women diagnosed with PPD. The efficacy endpoint is change from baseline in the Hamilton Depression Rating Scale (HAMD17) score. The Company expects to provide top-line data this quarter and announce next steps in our PPD program.
Enrollment is on-going in the Company’s Amaryllis study, a Phase 2 clinical trial to evaluate the safety, tolerability and efficacy of oral ganaxolone in women with PPD. Patients enrolled in the initial open label phase of the study receive one of multiple treatment regimens with oral ganaxolone. The efficacy endpoint is change from baseline in the HAMD17 score. The Company will provide an update on timing for data from the open label phase of the Amaryllis study after release of data from the Magnolia study, as the Magnolia study data may provide dosing insights that inform further oral dose optimization. Upon successful completion of the open label phase, the Amaryllis study is planned to continue as a double-blind placebo-controlled trial.
Vertex Pharmaceuticals’ VX-659 in combination with tezacaftor and ivacaftor for cystic fibrosis (CF) who have one F508del mutation and one minimal function mutation (F508del/Min). Phase 3 data due late 2018.
“We continue to make significant progress toward our goal of developing medicines for all people with CF as marked by the recent approvals of KALYDECO and ORKAMBI in younger children, the launch of SYMDEKO in the U.S. and the rapid progress we have made with our triple combination pivotal studies,” said Jeffrey Leiden, M.D., Ph.D., Chairman, President and Chief Executive Officer of Vertex. “In our development pipeline, we are advancing potential medicines for pain, sickle cell disease and beta thalassemia and are also beginning to advance additional compounds from research into early clinical development that could fundamentally change the treatment of other serious diseases in the future.”
Dr. Leiden continued, “Our ability to treat more and more people with CF is driving revenue and earnings growth and significant cash flow generation, which enables the company to invest to discover and develop transformative future medicines for the treatment of CF and other life-threatening diseases.”
TRIPLE COMBINATION REGIMENS
Bringing triple combination regimens to people with CF as quickly as possible
- Data are expected in late 2018 for the two Phase 3 studies of VX-659 in triple combination with tezacaftor and ivacaftor in people with CF who have one F508del mutation and one minimal function mutation and in people who have two F508del mutations.
- Enrollment is expected to be complete in the fourth quarter of 2018 for the two Phase 3 studies of VX-445 in triple combination with tezacaftor and ivacaftor in people with CF who have one F508del mutation and one minimal function mutation and in people who have two F508del mutations. The company plans to report data from these studies in the first quarter of 2019.
- Vertex plans to evaluate data from both the VX-659 and VX-445 Phase 3 triple combination programs to choose the best regimen to submit for potential regulatory approval. Together these data are expected to provide the basis for submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for people who have one F508del mutation and one minimal function mutation no later than mid-2019.
Evaluating VX-659 and VX-445 in children
- Studies are underway to evaluate both the VX-659 and VX-445 triple combination regimens in children with CF ages 6 through 11 who have one F508del mutation and one minimal function mutation and in children who have two F508del mutations. These studies are intended to support potential approval of a triple combination regimen in children ages 6 through 11.
Potential once-daily regimens
- In early 2018, the company reported positive safety and efficacy results for the once-daily potentiator VX-561 when dosed as part of a triple combination regimen including VX-659 or VX-445 and tezacaftor. The once-daily triple combination regimens were generally well tolerated, and the majority of adverse events were mild to moderate.
- Based on recent feedback from the FDA, Vertex plans to initiate a Phase 2 study in the first half of 2019 evaluating VX-561 as a monotherapy in people with CF who have a gating mutation.
SYMKEVI in the European Union
- On July 27, 2018, Vertex announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for SYMKEVI (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO) for the treatment of people with CF ages 12 and older who either have two copies of the F508del mutation or who have one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
- Approval for SYMKEVI in the European Union (EU) is expected in the fourth quarter of 2018.
APPROVED CF MEDICINES
Establishing long-term reimbursement outside of the U.S.
- On October 1, 2018, Vertex announced that it had entered into an innovative access contract with the Danish pharmaceutical and procurement organization, Amgros. This agreement provides eligible Danish CF patients access to all of Vertex’s current and future CF medicines. An agreement in Austria was also recently secured to provide access to ORKAMBI for all people with CF ages 6 through 11 who have two copies of the F508del mutation.
- In September 2018, Vertex announced a reimbursement agreement in Australia for the use of ORKAMBI in people with CF ages 6 and older who have two copies of F508del mutation. A pathway to access for future Vertex CF medicine, tezacaftor/ivacaftor, was also established as part of this process.
Treating patients at younger ages with CFTR modulators: The company continues to make significant progress toward gaining approval for its CF medicines to be used earlier in the course of disease progression. Recent highlights include:
- Vertex plans to submit a supplemental New Drug Application (sNDA) to the FDA in late 2018 based on positive data from a recently completed 24-week single-arm Phase 3 safety study of tezacaftor/ivacaftor in 70 children ages 6 through 11 who have two copies of the F508del mutation or who have one copy of the F508del mutation and one residual function mutation. The primary endpoint of the study was safety. The study met its primary safety endpoint, and safety data from the study showed that the treatment was generally well tolerated and safety data were consistent with those seen in previous Phase 3 studies of tezacaftor/ivacaftor in children ages 12 and older. To support approval in the EU, an eight-week, double-blind, placebo-controlled Phase 3 efficacy study is ongoing to evaluate tezacaftor/ivacaftor in approximately 65 children ages 6 through 11. The primary endpoint of this study is the absolute change in lung clearance index.
- KALYDECO was approved in August 2018 in the U.S. for children with CF ages 12 to <24 months. On October 19, 2018, Vertex announced that it received a positive opinion from the European CHMP for KALYDECO in this age group. The company expects a decision in the EU in late 2018.
- On October 18, 2018, Vertex announced positive data from a Phase 3 study evaluating ivacaftor in infants ages 6 to <12 months. These are the first data to demonstrate the potential to treat the underlying cause of CF in patients as young as six months old. The company plans to submit an sNDA to the FDA and a line extension to the EMA in late 2018.
- ORKAMBI was approved in August 2018 in the U.S. for children with CF ages 2 to 5 years old. A line extension has been submitted to the EMA with a decision anticipated in the first half of 2019.
- Dosing is underway in Phase 3 study evaluating lumacaftor/ivacaftor in children ages 12 to <24 months.
Supernus Pharmaceuticals’ SPN-812 for ADHD. Phase 3 data from P301 trial due December 2018 with data from other three trials due 1Q 2019.
- Total revenue of $103.0 million, a 28% increase over 2017
- Net product sales of $100.2 million, a 28% increase over 2017
- Operating earnings of $37.5 million, a 68% increase over 2017
- Diluted earnings per share (GAAP) of $0.52, a 79% increase over 2017
- Data from first three Phase III SPN-812 trials expected in December 2018
- Acquisition of Biscayne Neurotherapeutics, Inc. closed in October 2018
- Ranked #1 fastest growing pharmaceutical company worldwide per Fortune 100 fastest growing companies
ROCKVILLE, Md., Nov. 06, 2018 (GLOBE NEWSWIRE) — Supernus Pharmaceuticals, Inc. (NASDAQ: SUPN), a pharmaceutical company focused on developing and commercializing products for the treatment of central nervous system (CNS) diseases, today reported record financial results for the third quarter of 2018 and related Company developments.
Third quarter 2018 product prescriptions for Trokendi XR® and Oxtellar XR®, as reported by IQVIA, totaled 221,855, a 22.6% increase over the third quarter of 2017.
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Net product sales for the third quarter of 2018 were $100.2 million, a 28.3% increase over $78.1 million in the third quarter of 2017.
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“Supernus generated another strong quarter of growth, setting a new record for quarterly net product sales of $100 million,” said Jack Khattar, President and CEO of Supernus Pharmaceuticals. “Despite the market introduction of new competitive preventive treatments for migraine, Trokendi XR continued to capture a greater portion of the topiramate market. For Oxtellar XR, we continue to prepare for the potential launch of the monotherapy indication for partial seizures.”
Progress of Product Pipeline
Given the recently accelerated development timeline for SPN-812 that positions its potential regulatory approval and commercial launch ahead of SPN-810, the Company has directed its resources to prioritize filing of the New Drug Application (NDA) and potential commercial launch of SPN-812 in the United States.
As a result, the following are the updated plans and timelines for both product candidates:
SPN-812 – Novel non-stimulant for the treatment of ADHD
- The Phase III program consists of four three-arm, placebo-controlled trials: P301 and P303 trials in patients 6-11 years old and P302 and P304 trials in patients 12-17 years old.
- The Company expects to announce top-line data from P301 and P303 pediatric trials concurrently in early December 2018, and from P302, the first adolescent Phase III trial, by the end of December 2018. Top-line data from the second adolescent Phase III trial, P304, are expected by the end of the first quarter of 2019.
- The Company expects to submit an NDA for SPN-812 in the second half of 2019, and to launch it, pending U.S. Food and Drug Administration (FDA) approval, in the second half of 2020.
SPN-810 – Treatment of Impulsive Aggression in patients with ADHD
- As expected, the first Phase III trial (P301) has reached its original enrollment target. However, given the aforementioned prioritization of SPN-812 and that top-line data from the second Phase III trial (P302) is expected around mid-2019, the Company has decided to keep P301 enrollment active until data from both trials can be released concurrently instead of sequentially. This change does not impact the timing of submission of the NDA for SPN-810, given that the NDA submission is rate-limited by completion of the P302 trial and generation of data in the adolescent patient population.
- The Company continues to observe enrollment in the open label extension (OLE) study at 90% or higher. On average, a patient in the OLE study remains on SPN-810 treatment for 9.5 months, which we believe is an encouraging sign of the tolerability and efficacy of SPN-810.
- Patient dosing has been initiated in the Phase III trial in adolescent patients.
- The Company expects to submit an NDA for SPN-810 in the second half of 2020, and to launch it, pending FDA approval, in the second half of 2021.
SPN-604 (formerly known as Oxtellar XR for Bipolar)
- The Company continues to expect initiating pivotal Phase III studies for the treatment of bipolar disorder in the second half of 2019.
“We are pleased to announce the completion of enrollment in the first three Phase III trials for SPN-812,” said Jack Khattar. “We look forward to reporting top-line data from these trials during December 2018. If successful, SPN-812 has the potential to be a novel non-stimulant for the treatment of ADHD that compares favorably to existing medications.”