The FDA calendar tracks upcoming PDUFA, Phase 2, and Phase 3 events. Phase 1 events are not included because they are too speculative and dangerous to trade. The idea is to only speculate in drugs that have at least passed Phase 1 trials.
Use the Search Events box at the top of the calendar to search for a company’s ticker, name of drug or trial. Dates with a blue box mark events. Hover over the blue box to see the number of events for that day. Clicking on an event day will show all events below the calendar for that day. Clicking on an event below the calendar will load an events detail page where you can read more about the event and see a stock chart through TradingView and a news feed through Google News Search if they are available.
Click the SUBSCRIBE TO THIS CALENDAR button and enter your email address to be alerted by email when important events are added to the calendar.
Catalyst swing trading is one of many tools used to “crack the code” of profitable stock market endeavors. The most basic aspect of catalyst trading is to foresee coming “hype” within a stock or industry, buy in advance, and sell into the exponential rise. If you plan to hold through the event, only hold onto what you can afford to lose; even when the news is positive, the hype is over and price action can turn negative. Aka “sell the news.”
Trading Biotech Stocks With Upcoming Catalysts
Catalyst events that move stocks are:
-Presentations/Conferences – (Any event tickers are hyping into)
The idea is to start tracking a stock’s price performance about a month before a PDUFA, Phase 2, or Phase 3 event. Ideally, you want the biotech stock to meet the following criteria:
-50%+ Analyst Price Target
-Action Heating Up
-Float Short 10%+
-Good Technical Setup
-Institutional Ownership 20%+
– Low Chance of Dilution/Cash on Hand
– Low Float/Small Cap
Please keep in mind that the goal is to play the run up or the news “hype” of the PDUFA, Phase 2, or Phase 3 event, NOT hold through it. As soon as the biotech stock runs up, you sell and book profits. Do not hold through the event unless there is a very high probability that the event news will be positive.SUBSCRIBE TO THIS CALENDAR
Seladelpar for Primary biliary cholangitis (PBC). Phase 2 top-line data released late May 2016. 52-week data due at AASLD November 12, 2018. CymaBay Announces the Initiation of the Seladelpar Global Phase
Seladelpar for Primary biliary cholangitis (PBC). Phase 2 top-line data released late May 2016. 52-week data due at AASLD November 12, 2018.
CymaBay Announces the Initiation of the Seladelpar Global Phase 3 Registration Study (ENHANCE) for the Treatment of Primary Biliary Cholangitis and Additional Corporate Updates
NEWARK, Calif., Oct. 30, 2018 (GLOBE NEWSWIRE) — CymaBay Therapeutics, Inc. (NASDAQ: CBAY), today announced that the company has initiated the seladelpar Phase 3 registration study for the treatment of Primary Biliary Cholangitis (PBC). The study is a 52-week, placEbo-coNtrolled, randomized, pHAse 3 study to evaluate the safety aNd effiCacy of sEladelpar (ENHANCE) that will be conducted in more than 20 countries spanning five continents. The study is intended to establish the efficacy and safety of seladelpar for the treatment of PBC to support the submission of a global registration dossier with health authorities to obtain approval of seladelpar. Seladelpar is a drug candidate for PBC patients who are inadequate responders to ursodeoxycholic acid (UDCA), the first line treatment of PBC, or who are intolerant to UDCA.
Pol Boudes, MD, Chief Medical Officer of CymaBay, commented, “The initiation of this Phase 3 is truly an exciting moment. I want to warmly thank all of the PBC patients who participate in our clinical studies, as well as their families and their medical teams. The data we have collected since the first PBC patient received seladelpar in December 2015 are encouraging and we believe support the potential for seladelpar to significantly improve the lives of patients with PBC. I also want to thank the CymaBay team, our advisers, and the regulators around the world who made this moment possible.”
Organizational Update – Development team expanded with key senior hires
In addition to the initiation of ENHANCE, CymaBay announced the appointment of key individuals to expand and strengthen the development organization in order to execute the seladelpar Phase 3 program and deliver a high-quality registration package.
Dr. Patricia Rohane, M.D., has joined CymaBay as Vice President, Clinical Development. Patricia brings extensive Phase 3 and global registration experience and will lead the seladelpar clinical team. Patricia is specialized in immunology/allergy and internal medicine and spent more than 15 years at Sanofi-Aventis and Celgene.
Dr. Stephen Rossi, Pharm. D., joins as Vice President, Early Clinical Development. Steve brings more than 15 years of experience in liver diseases, both in academia and industry. Steve most recently led the development of NGM282 for nonalcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC), and PBC at NGM Biopharmaceuticals. He also held leadership roles at Gilead Sciences and Roche.
Kamal Sigel, M.S., has been named Vice President, Quality. Kamal is a Certified Quality Auditor and brings substantial experience ranging from Phase 3 to commercial in the areas of manufacturing, compliance and quality control. Kamal comes to CymaBay most recently after more than 10 years at Anthera Pharmaceuticals and Hyperion Therapeutics.
Sujal Shah, President and CEO of CymaBay stated, “We are particularly excited to welcome Patricia, Steve, and Kamal to CymaBay as we continue to build the organization to achieve our goal of delivering seladelpar to PBC patients. The initiation of ENHANCE is a transformative event for the company. As we look to take this pivotal step forward in the development of seladelpar for PBC, new results from our ongoing Phase 2 study will be highlighted in two late-breaking presentations on November 12 at The Liver Meeting® 2018 of the American Association for the Study of Liver Diseases. These data give us confidence that we can target a potential label for seladelpar that reflects a strong efficacy profile without drug-induced pruritus.”
Pipeline Update – Kowa returns rights to arhalofenate; resources fully focused on liver programs
On October 24, 2018, Kowa Pharmaceuticals America, Inc. terminated the Exclusive License Agreement for the rights to develop and commercialize arhalofenate in the U.S. Arhalofenate is a Phase 3-ready drug candidate with a dual-acting anti-inflammatory and uricosuric activity. Arhalofenate had been in development as a combination with febuxostat, a xanthine oxidase inhibitor, to deliver greater urate lowering and anti-flare activity for patients with gout.
“We are disappointed in Kowa’s decision to end our development and commercialization agreement for arhalofenate for the treatment of gout,” said Sujal Shah. “However, over the past two years, we have been fully committed to redirecting our efforts and resources toward our current focus in liver disease and are extremely proud of the progress we’ve made advancing seladelpar in PBC and NASH. We believe seladelpar has the potential to significantly improve patient care and look forward to providing further updates in the weeks ahead.”
Primary biliary cholangitis (PBC) is a serious and potentially life-threatening autoimmune disease of the liver characterized by impaired bile flow (cholestasis) and accumulation of toxic bile acids. There is an accompanying inflammation and destruction of the intrahepatic bile ducts, which can progress to fibrosis, cirrhosis and liver failure. Other clinical symptoms of PBC include fatigue and pruritus, which can be quite disabling in some patients. PBC is primarily a disease of women: 1 in 1000 women over the age of 40 lives with PBC.
Seladelpar is a potent, selective, orally active PPARδ agonist that is in development for the treatment of the liver diseases PBC and NASH. For PBC, seladelpar has received an orphan designation from the US Food and Drug Administration and the European Medicine Agency. Seladelpar also received the PRIority MEdicine (PRIME) status from the European Medicine Agency.
ENHANCE (NCT03602560) is a 52-week, placebo-controlled, randomized, Phase 3 study to evaluate the safety and efficacy of seladelpar. It will be conducted in more than 20 countries over five continents (North America, South America, Europe, Australasia and Asia). Approximately 240 PBC patients will be randomized to seladelpar 10 mg/day, seladelpar 5/10 mg/day (starting treatment at 5 mg with the possibility to escalate dose to 10 mg after 6 months), or placebo. Patients must experience an inadequate response to UDCA (defined as a serum alkaline phosphatase level ≥ 1.67 x the upper limit of normal after at least 12 months of treatment) or an intolerance to UDCA to be eligible for the study. Patients who are inadequate responders to UDCA will continue their treatment during the study, and UDCA will be provided free of charge. The primary outcome measure is the responder rate after 52 weeks. A responder is defined as a patient who achieves an alkaline phosphatase level
CymaBay Therapeutics News
All Day (Monday)
TRC102 and Temodar for Glioblastoma, cancer. Phase 2 data at Society for Neuro-Oncology annual meeting in November 14 - 15 2018. Third Quarter 2018 and Recent Corporate Highlights In
TRC102 and Temodar for Glioblastoma, cancer. Phase 2 data at Society for Neuro-Oncology annual meeting in November 14 – 15 2018.
Third Quarter 2018 and Recent Corporate Highlights
- In September, we announced the publication of results from a Phase 1b clinical trial combining TRC105 with Inlyta® (axitinib) in patients with advanced or metastatic renal cell carcinoma (RCC) in The Oncologist. These data were previously presented at the European Society for Medical Oncology (ESMO) 2016 Congress in Copenhagen, Denmark. The open-label dose escalation and expansion Phase 1b study enrolled a total of 18 patients (17 of whom were evaluable for response) who had received at least one prior line of therapy with a VEGF receptor tyrosine kinase inhibitor. Patients in the trial received a combination of TRC105 and Inlyta and demonstrated an objective response rate (ORR) of 29%, an 88% overall disease control rate, and a median progression-free survival (PFS) of 11.3 months. For comparison, in a separate trial, the ORR seen in the large subgroup of VEGFR TKI-refractory patients treated with Inlyta (n=194) in the Inlyta AXIS Phase 3 study in second-line clear cell RCC patients was 11.3%, and median PFS was 4.8 months. The publication also noted that plasma levels of TGF-β receptor 3 (betaglycan) at baseline were significantly higher in patients who experienced a confirmed partial response, while levels of osteopontin were significantly lower at baseline for patients that achieved a confirmed partial response. Both markers correlated with time on study and their potential prognostic value will be investigated in the ongoing Phase 2b TRAXAR study. TRACON’s Phase 2b TRAXAR clinical trial of TRC105 in combination with Inlyta completed enrollment of 150 patients with advanced or metastatic RCC in Q3 2017 and top-line data are expected in December 2018.
- In September, we announced that the sample size of the Phase 3 TAPPAS clinical trial of TRC105 in combination with Votrient was amended to increase the sample size to account for a higher than expected rate of withdrawal for progressive disease not confirmed by central review. The amended protocol was reviewed and accepted by the FDA, with retention of the special protocol assessment (SPA) agreement. We expect the interim analysis to determine the final sample size and eligible patient population of the trial in Q1 2019. The trial design was published in Annals of Oncology in October 2018 and was recognized as the Most Innovative Trial of 2017 by the Clinical and Research Excellence Awards.
- In September, we submitted updated data from the Phase 1/2 trial of TRC105 and Nexavar in patients with hepatocellular carcinoma (HCC) to the Gastrointestinal Cancers Symposia of ASCO, which meets in January 2019 in San Francisco. The trial completed Phase 1 enrollment and is currently enrolling up to 21 patients in the Phase 2 portion with overall response rate as the primary endpoint. Two of nine evaluable patients in the Phase 1 portion of the trial had durable confirmed partial responses (22%). For comparison, in separate Phase 3 trials, the durable confirmed partial responses seen with Nexavar in patients with HCC was 2% and 3%.
- In October, we completed dose escalation of the Phase 1 trial of TRC105 in combination with Opdivo in lung cancer patients. We are currently enrolling two expansion cohorts of 12 patients each, one with patients naïve to PD-1/PD/L1 inhibitor therapy and one with patients who have relapsed following prior PD-1/PD-L1 therapy. We expect to provide top line safety and efficacy data from patients in the Phase 1 portion of the trial in December 2018.
- In July, we completed the Phase 1 portion of a Phase 1/2 trial of TRC253 in patients with metastatic prostate cancer and are now enrolling patients in the Phase 2 portion of the trial at approximately 20 sites in the US. In the Phase 2 portion of the trial, we are incorporating circulating tumor DNA testing in order to allow for biomarker-directed therapy of prostate cancer patients who have progressed following treatment with an androgen receptor inhibitor. We now expect top line Phase 2 data in 2020, rather than 2019, due to slower than expected enrollment resulting from a lower than anticipated frequency of a specific tumor mutation targeted by TRC253 among metastatic prostate cancer patients. As a reminder, we licensed TRC253 from Janssen and if they exercise their right to reacquire the asset following Phase 2 proof of concept data, TRACON will be entitled to receive a $45M payment, up to $137.5M in additional potential milestones, and a low single digit royalty on sales.
“We anticipate significant potential catalysts over the next two quarters, including two randomized data points: top-line Phase 2 data from TRAXAR in renal cell carcinoma and interim Phase 3 results from TAPPAS in angiosarcoma” said Charles Theuer, M.D., Ph.D., President and CEO of TRACON. “Of note, we continue to be encouraged by the rapid rate of accrual into the Phase 3 TAPPAS angiosarcoma trial.”
Expected Upcoming Milestones
- Announcement of safety and efficacy data from the Phase 2 trial of TRC102 in combination with Temodar in patients with glioblastoma at the Society for Neuro-Oncology annual meeting in November 2018.
- Announcement of top-line data, including biomarker correlations, from the randomized Phase 2 TRAXAR trial of TRC105 in combination with Inlyta for patients with advanced or metastatic RCC is expected in December 2018.
- Announcement of safety and efficacy data from the Phase 1b trial of TRC105 in combination with Opdivo in patients with non-small cell lung cancer is expected in December 2018.
- Announcement of the results of the interim analysis from the Phase 3 pivotal TAPPAS trial of TRC105 in angiosarcoma is expected in Q1 2019.
Third Quarter 2018 Financial Results
- Cash, cash equivalents and short-term investments were $47.2 million at September 30, 2018, compared to $34.5 million at December 31, 2017. We expect our current cash, cash equivalents and short-term investments to fund operations into Q4 2019.
- Research and development expenses for the third quarter of 2018 were $7.0 million compared to $4.3 million for the third quarter of 2017. The increase was primarily attributable to increased TRC105 drug manufacturing activities and direct clinical trial expenses in the third quarter of 2018 as compared to the 2017 period.
- General and administrative expenses for the third quarter of 2018 were $2.1 million compared to $1.8 million for the third quarter of 2017.
- Net loss for the third quarter of 2018 was $9.1 million compared to net income of $1.2 million for the third quarter of 2017. The net income in the third quarter of 2017 was a result of receiving and recognizing as revenue a $7.0 million milestone payment from Santen.
Investor Conference Call
The Company will hold a conference call today at 4:30 p.m. EST / 1:30 p.m. PST to provide an update on corporate activities and to discuss the financial results of its third quarter of 2018. The dial-in numbers are (855) 779-9066 for domestic callers and (631) 485-4859 for international callers. Please use passcode 3189378. A live webcast of the conference call will be available online from the Investor/Events and Presentation page of the Company’s website at www.traconpharma.com.
TRACON Pharmaceuticals News
november 14 (Wednesday) - 15 (Thursday)
Advisory Committee Meeting November 14, 2018. PDUFA date November 16, 2018. U.S. FDA Announces Joint Advisory Committee Review of Mallinckrodt's Abuse-Deterrent, Immediate-Release Reformulation of Roxicodone® (Oxycodone Hydrochloride) -- Company Seeks Approval of
Advisory Committee Meeting November 14, 2018. PDUFA date November 16, 2018.
U.S. FDA Announces Joint Advisory Committee Review of Mallinckrodt’s Abuse-Deterrent, Immediate-Release Reformulation of Roxicodone® (Oxycodone Hydrochloride)
— Company Seeks Approval of New Drug Application for MNK-812 Through 505(b)2 Regulatory Pathway —
STAINES-UPON-THAMES, United Kingdom, Oct. 8, 2018 /PRNewswire/ — Mallinckrodt plc (NYSE: MNK), a leading global specialty pharmaceutical company, today announced the U.S. Food and Drug Administration (FDA) has confirmed it will hold a joint meeting of its Anesthetic and Analgesic Drug Products Advisory Committee and Drug Safety and Risk Management Advisory Committee on Nov. 14, 2018.
The Committees will assess the 505(b)2 new drug application submission for MNK-812, Mallinckrodt’s abuse-deterrent formulation of immediate-release, single-entity oxycodone tablets with a proposed indication for the management of pain severe enough to require an opioid analgesic and for which alternative treatments are inadequate. Mallinckrodt is advancing this reformulation of Roxicodone® (oxycodone hydrochloride tablets USP) with properties designed to deter intravenous and intranasal abuse.
“We look forward to engaging with the Advisory Committees to discuss the potential benefits our new abuse-deterrent formulation technology can bring in helping to mitigate opioid abuse and misuse,” said Matt Harbaugh, Executive Vice President, Chief Financial Officer and President, Specialty Generics for Mallinckrodt.
The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of Nov. 16, 2018, to complete its review of Mallinckrodt’s product.
All Day (Friday)
Varlilumab and nivolumab for solid tumors, cancer. Phase 1/2 data to be presented November 17, 2018 at the Society for Neuro-oncology (SNO) Meeting. Celldex Provides Corporate
Varlilumab and nivolumab for solid tumors, cancer. Phase 1/2 data to be presented November 17, 2018 at the Society for Neuro-oncology (SNO) Meeting.
HAMPTON, N.J., Nov. 07, 2018 (GLOBE NEWSWIRE) — Celldex Therapeutics, Inc. (NASDAQ:CLDX) today reported business and financial highlights for the third quarter ended September 30, 2018.
“We made considerable progress in the third quarter, particularly in the development program for CDX-1140, our promising antibody targeted to CD40,” said Anthony Marucci, Co-founder, President and Chief Executive Officer of Celldex Therapeutics. “We have completed four of the potential eight monotherapy dose levels in the ongoing Phase 1 study and remain encouraged by the safety and biological profile we have observed to date. We look forward to sharing interim data at the SITC Annual Meeting later this week. During the third quarter, we also began enrolling patients in a combination cohort of CDX-1140 with our dendritic cell mobilizer, CDX-301. We are very interested to explore the potential of CDX-1140 in the presence of greater dendritic cell activity.”
“Additionally, we are nearing completion of enrollment to the first stage of the Phase 2 study of CDX-3379 in advanced head and neck squamous cell cancer and anticipate data from this portion of the study in the first quarter of 2019,” continued Marucci. “We are also advancing several preclinical programs that we believe can play an important role in enhancing the immune system’s response to cancer, including CDX-0159, our TAM program targeting Tyro3, AXL and MerTK, and our growing bispecific antibody program.”
- Enrollment continues in the Phase 1 dose-escalation study of CDX-1140 in solid tumors. Interim data from the ongoing study have been accepted for presentation on Friday, November 9, 2018 at the Society for Immunotherapy of Cancer (SITC) Annual Meeting. This study is designed to enroll up to 150 patients with recurrent, locally advanced or metastatic solid tumors and was recently amended to also include B-cell lymphomas. CD40 has long been an important target for immunotherapy, as it plays a critical role in the activation of innate and adaptive immune responses; however, effectively balancing systemic dosing and safety has proven challenging to date for CD40-activating therapeutics. CDX-1140 is a unique, potent CD40 agonist that Celldex believes has the potential to successfully balance systemic doses for good tissue and tumor penetration with an acceptable safety profile. Data to date from the four completed dosing cohorts (0.01, 0.03, 0.09 and 0.18 mg/kg) suggest that CDX-1140 is exhibiting a desirable safety profile and demonstrating early signs of biological activity based on biomarker analysis. The fifth monotherapy cohort at 0.36 mg/kg is currently being enrolled, along with the combination therapy cohort of CDX-1140 (0.09 mg/kg) with CDX-301 which was initiated in late August. CDX-301 is a dendritic cell growth factor that will be used as a priming agent to potentially increase the number of cells available to respond to CDX-1140. In addition, Celldex is evaluating the potential for combination with varlilumab, especially in lymphomas which co-express CD40 and CD27 receptors.
- Enrollment is nearing completion in the first stage of the Phase 2 study (n=13) of CDX-3379 in advanced head and neck squamous cell cancer in combination with Erbitux® in Erbitux-resistant patients who have been previously treated with or are ineligible for checkpoint therapy. According to the study’s Simon two-stage design, if at least one patient achieves an objective response in the first stage, enrollment may progress to the second stage. While a confirmed partial response has been documented, Celldex will wait to review the full data set before making decisions on future development, as a number of patients are still undergoing treatment and are not yet eligible for response evaluation.
- Data from the glioblastoma cohort in the Phase 1/2 study of varlilumab and Opdivo® have been accepted for presentation on Saturday, November 17, 2018 at the Society for Neuro-oncology (SNO) Annual Meeting.
- As previously disclosed, on May 29, 2018, Celldex received written notice from the Listing Qualifications department of the Nasdaq Stock Market indicating that the Company was not in compliance with the $1.00 minimum bid price requirement for continued listing on the Nasdaq Global Market. As is standard, the Company was afforded 180 days to regain compliance. Unless Celldex regains compliance with the minimum bid requirement by November 26, 2018 (the 180th day), the Company plans to apply to transfer to the Nasdaq Capital Market. Assuming Celldex’s application is accepted, the proposed transition should be seamless for Celldex shareholders and should allow the company an additional 180-day period in which to regain compliance. If Celldex is unable to regain compliance with the minimum bid price requirement, the Company may implement a reverse stock split to maintain its listing, a measure that was approved by shareholders at the Company’s 2018 Annual Meeting.
Celldex Therapeutics News
All Day (Saturday)
AQST-119 for erectile dysfunction. PDUFA date set for November 18, 2018. "Aquestive is the worldwide leader in delivering therapeutics on oral film with our PharmFilm® technology. Over the past quarter, we
AQST-119 for erectile dysfunction. PDUFA date set for November 18, 2018.
“Aquestive is the worldwide leader in delivering therapeutics on oral film with our PharmFilm® technology. Over the past quarter, we have met a number of key business milestones that position us well to achieve our long-term vision for growth,” said Keith J. Kendall, Chief Executive Officer of Aquestive. “We are excited to continue advancing our pipeline and building our CNS commercial franchise with the launch of SYMPAZAN™. Late stage development of Libervant is also advancing with the recent completion of the adult EMU study.”
Pipeline Overview, Upcoming Milestones and Business Update
Aquestive received U.S. FDA approval for SYMPAZAN (clobazam) oral film for the adjunctive treatment of seizures associated with Lennox-Gastaut syndrome (LGS) in patients 2 years of age or older. SYMPAZAN previously received tentative FDA approval on August 31, 2018 and has now been approved based on the expiration of the orphan drug exclusivity period for ONFI®.
Aquestive completed a pharmacokinetic adult epilepsy monitoring unit (EMU) study demonstrating that its investigational diazepam buccal film (DBF) product candidate, tentatively named Libervant, provides comparable bioavailability whether administered between seizures (interictal) or during and shortly after seizures (ictal/peri-ictal) in adult patients with poorly controlled tonic-clonic seizures or focal seizures with impaired awareness. DBF, a novel formulation of diazepam as a small, thin film strip placed inside the cheek, is under development for the management of selected patients with refractory epilepsy who require intermittent use of diazepam to control episodes of increased seizure activity.
The topline data from the adult EMU study will be presented as two late-breaking poster presentations at the American Epilepsy Society’s Annual Meeting, being held from November 30 to December 4, 2018.
Aquestive recently reached agreement with the FDA to end the swallowing study for AQST-117 (riluzole), an oral soluble film formulation for the treatment of Amyotrophic Lateral Sclerosis, the third late-stage CNS asset in its proprietary portfolio, sooner than previously planned based on an analysis of the study’s interim data. Topline results are expected to be reported in late 2018 and an NDA submission is expected in the first quarter of 2019.
Aquestive continues to make progress on its early stage complex molecule development programs for anaphylaxis and acromegaly. The Company expects to commence a Phase 1 proof of concept study during 2019 for AQST-108, its sublingual film formulation of epinephrine in development for the treatment of anaphylaxis. During the third quarter 2018, Aquestive commenced a Phase 1 human proof of concept study for AQST-305, the Company’s sublingual formulation of octreotide in development for the treatment of acromegaly.
Aquestive’s collaborative partnership development activities continue to advance, and its year-to-date Suboxone volume produced and shipped has risen by 5%. In addition, AQST-119, an oral soluble film formulation of tadalafil, a vasodilator that is used to treat erectile dysfunction, or ED, has its Prescription Drug User Fee Act (PDUFA) date on November 18, 2018 for tentative approval.
Aquestive Therapeutics News
All Day (Sunday)
Larotrectinib (LOXO-101) for solid tumors that harbor a TRK fusion. PDUFA date under priority review November 26, 2018. Larotrectinib New Drug Application (NDA) PDUFA date is November 26, 2018 LOXO-292 Registrational
Larotrectinib (LOXO-101) for solid tumors that harbor a TRK fusion. PDUFA date under priority review November 26, 2018.
Larotrectinib New Drug Application (NDA) PDUFA date is November 26, 2018
LOXO-292 Registrational Data Expected in 2019
LOXO-292 NDA Submission Expected in Late 2019
Enrollment of First Patient in LOXO-305 Phase 1/2 Study on Track for Fourth Quarter 2018
STAMFORD, Conn., Nov. 08, 2018 (GLOBE NEWSWIRE) — Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company developing highly selective medicines for patients with genomically defined cancers, today reported third quarter 2018 financial results.
“In the third quarter we made significant progress across our pipeline,” said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. “At ESMO, larotrectinib investigators provided a comprehensive program update that included durability and additional response data in patients with TRK fusion cancers. Medical meeting updates for LOXO-292 in September and October showed encouraging ongoing durability of response for patients with RET-driven cancers. Taken together, these data have increased our conviction around a foundational company thesis—that selective, purpose-built medicines offer the best opportunity for durable efficacy and a manageable safety profile. In the fourth quarter, we look forward to LOXO-305, our fourth program, entering the clinic.”
- Conference presentations
- European Society for Medical Oncology (ESMO) 2018 Congress: On October 21, 2018, updated clinical data for larotrectinib were presented at ESMO. The oral presentation provided approximately one year of additional follow-up for the primary dataset, the 55 patients with TRK fusion cancer described in the larotrectinib New England Journal of Medicine publication from February 2018. In addition, the update included data for a supplementary dataset, an additional 67 patients with TRK fusion cancer who were subsequently enrolled across the larotrectinib development program. Response evaluations were based on investigator assessment. As of a data cut-off date of July 30, 2018, in the primary dataset (n=55), the overall response rate (ORR) was 80% (44/55) (95% CI: 67-90%) and in the supplementary dataset (n=67), the ORR was 81% (44/54) (95% CI: 69-91%). Across both datasets, the ORR was 81% (88/109) (95% CI: 72-88%). The ORR analyses for the supplementary and integrated datasets included nine patients with unconfirmed partial responses awaiting confirmatory response assessments, but did not include 13 patients who were awaiting an initial response assessment and continuing on study. Median duration of response (DOR) had not been reached in either the primary dataset or supplementary dataset, with median follow-up of 17.6 months and 7.4 months, respectively. Larotrectinib was well tolerated, with the majority of adverse events recorded as grade 1 or 2. The most common treatment-emergent adverse events occurring in 15% or more of patients in the trial were fatigue, dizziness, nausea, constipation, anemia, increased alanine aminotransferase (ALT), increased aspartate aminotransferase (AST), cough, diarrhea, vomiting, pyrexia, dyspnea, headache, myalgia and peripheral oedema. See the presented data here.
- Annual Meeting of the American Thyroid Association (ATA): On October 4, 2018, clinical data for patients with TRK fusion thyroid cancer enrolled in the larotrectinib development program were presented in an oral presentation at ATA. See the presented data here.
- International Association for the Study of Lung Cancer (IASLC) 19th World Conference on Lung Cancer: On September 24, 2018, clinical data for patients with TRK fusion non-small cell lung cancer (NSCLC) enrolled in the larotrectinib development program were presented in a poster presentation at the IASLC World Conference on Lung Cancer. The poster can be found here.
- Molecular Analysis for Personalised Therapy 2018 Congress: On September 15, 2018, Ventana Medical Systems, Inc., a member of the Roche Group, and Loxo Oncology, presented a co-authored poster on the analytical validation of Ventana’s pan-TRK IHC assay at the Molecular Analysis for Personalised Therapy 2018 Congress. These data, in addition to other recently published evidence, suggest an annual incidence of approximately 2,500 to 3,000 cases of TRK fusion cancer in the United States. The poster can be found here.
- Integrative Therapies Program for Children with Cancer (ITPCC): On September 13, 2018, clinical data for children and adolescents with TRK fusion metastatic thyroid carcinoma enrolled in the larotrectinib development program were presented at the ITPCC conference. The poster can be found here.
- Targeted Oncology Publication: On October 2, 2018, a manuscript was published online in Targeted Oncology detailing the potential effectiveness of TRK inhibition, including larotrectinib treatment, in patients with tumors harboring NTRK gene fusions, and the need for effective testing strategies. The publication can be found here.
- British Journal of Cancer Publication: On September 17, 2018, a case report was published in the British Journal of Cancer detailing a patient with TRK fusion high-grade glioma treated with larotrectinib. The publication can be found here.
- Cancer Publication: On September 11, 2018, a manuscript was published online in Cancer detailing the treatment of children with locally advanced TRK fusion sarcoma who were treated preoperatively with larotrectinib and underwent subsequent surgical resection. The publication can be found here.
- JCO Precision Oncology Publication: On August 2, 2018, a case report was published in JCO Precision Oncology detailing an adolescent patient with a TRK fusion undifferentiated sarcoma treated with larotrectinib. The publication can be found here.
- European Marketing Authorization Application (MAA): On August 27, 2018, Loxo Oncology and Bayer announced that Bayer had submitted an MAA for larotrectinib to the European Medicines Agency (EMA). More information can be found here.
- LOXO-195 Orphan Drug Designation (ODD): In October, the U.S. Food and Drug Administration (FDA) granted ODD to LOXO-195 for the treatment of solid tumors with neurotrophic tyrosine receptor kinase (NTRK)-fusion proteins that have developed acquired resistance to prior TRK inhibitor therapy. The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to Loxo Oncology certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
- Annual Meeting of the ATA: On October 6, 2018, updated interim clinical data for LOXO-292 from the global Phase 1/2 LIBRETTO-001 trial in patients with RET-mutant medullary thyroid cancer (MTC) and RET fusion-positive thyroid cancer were presented at the Annual Meeting of the ATA. The data presented were based on a July 19, 2018 data cut-off date and included the 29 patients with RET-mutant MTC and the nine patients with RET fusion-positive thyroid cancer who were included in the LOXO-292 presentation at the 2018 ASCO Annual Meeting. With 3.5 months of additional follow-up since the ASCO presentation, LOXO-292 demonstrated encouraging, early evidence of durable activity. Sixteen of 17 (94%) responding RET-mutant MTC patients remained on therapy and in response (median follow-up of 7.6 months for all 29 patients; median follow-up of 8.4 months for responding patients). Seven of seven (100%) responding RET fusion-positive thyroid remained on therapy and in response (median follow-up of 7.6 months for all nine patients; median follow-up of 8.5 months for responding patients). In RET-mutant MTC, the overall response rate was 59% (17/29) (95% CI: 39-77%) and the confirmed overall response rate was 56% (15/27) (95% CI: 35-75%). Of nine patients with RET fusion-positive thyroid cancer, the confirmed overall response rate was 78% (7/9) (95% CI: 40-97%). Of the 82 patients in the safety analysis, most treatment-emergent adverse events were Grade 1 in severity and judged by the investigator as not related to LOXO-292. See the presented data here.
- IASLC 19th World Conference on Lung Cancer: On September 25, 2018, updated interim clinical data for LOXO-292 from the global Phase 1/2 LIBRETTO-001 trial in patients with RET fusion-positive NSCLC were presented at the IASLC World Conference on Lung Cancer. The data presented were based on a July 19, 2018data cut-off date and included the 38 patients with RET fusion-positive NSCLC who were initially included in the LOXO-292 presentation at the 2018 ASCO Annual Meeting. With 3.5 months of additional follow-up since the ASCO presentation, LOXO-292 demonstrated encouraging, early evidence of durable activity, with 25 of 26 (96%) responding RET fusion-positive NSCLC patients remaining on therapy and 24 of 26 (92%) remaining in response (median follow-up of 8.5 months for all 38 patients; median follow-up of 9.5 months for responding patients). The overall response rate was 68% (26/38) (95% CI: 51-83%) and the confirmed overall response rate was 68% (25/37) (95% CI: 50-82%). Of the 82 patients in the safety analysis, most treatment-emergent adverse events were Grade 1 in severity and judged by the investigator as not related to LOXO-292. See the presented data here.
- LOXO-292 Breakthrough Therapy Designations: The FDA granted three Breakthrough Therapy Designations to LOXO-292:
- for the treatment of patients with metastatic RET fusion-positive non-small cell lung cancer who require systemic therapy and have progressed following platinum-based chemotherapy and an anti-PD-1 or anti-PD-L1 therapy;
- for the treatment of patients with RET-mutant medullary thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options; and for
- for the treatment of patients with advanced RET fusion-positive thyroid cancer who require systemic therapy, have progressed following prior treatment and have no acceptable alternative treatment options.
More information can be found here and here.
- LOXO-292 Orphan Drug Designation: In October, the FDA granted ODD to LOXO-292 for the treatment of pancreatic cancer. The FDA’s Office of Orphan Drug Products grants orphan drug designation to support the development of medicines for underserved patient populations, or rare disorders, that affect fewer than 200,000 people in the United States. Orphan drug designation provides to Loxo Oncology certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees and tax credits for qualified clinical trials.
- Society of Hematologic Oncology (SOHO) Annual Meeting: On September 12, 2018, preclinical characterization data for LOXO-305 were presented at the SOHO Annual Meeting. The poster can be found here.
Loxo Oncology News
All Day (Monday)
Firdapse for Lambert-Easton Myasthenic Syndrome (LEMS). NDA resubmitted with new PDUFA date under priority review of November 28, 2018. Catalyst Pharmaceuticals Announces FDA Acceptance of NDA
Firdapse for Lambert-Easton Myasthenic Syndrome (LEMS). 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.”
About Catalyst Pharmaceuticals
Catalyst Pharmaceuticals is a biopharmaceutical company focused on developing and commercializing innovative therapies for people with rare debilitating, chronic neuromuscular and neurological diseases, including Lambert-Eaton myasthenic syndrome (LEMS), congenital myasthenic syndromes (CMS), MuSK antibody positive myasthenia gravis, spinal muscular atrophy (SMA) type 3, and infantile spasms. Firdapse® (amifampridine phosphate) has received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the treatment of LEMS and Orphan Drug Designation for LEMS, CMS and myasthenia gravis. Firdapse is the first and only approved drug in Europe for symptomatic treatment in adults with LEMS.
Catalyst is also developing CPP-115 to treat refractory infantile spasms. CPP-115 has been granted U.S. Orphan Drug Designation for the treatment of infantile spasms by the FDA and has been granted E.U. Orphan Medicinal Product Designation for the treatment of West syndrome by the European Commission. In addition, Catalyst is developing a generic version of Sabril® (vigabatrin).
Catalyst Pharmaceuticals News
All Day (Wednesday)
OMS-100 for melanoma. Phase 2 data to be presented November 30, 2018. Dr. Alain Algazi to discuss key findings from OMS-100 study of monotherapy TAVO demonstrating local treatment with TAVO
OMS-100 for melanoma. Phase 2 data to be presented November 30, 2018.
Dr. Alain Algazi to discuss key findings from OMS-100 study of monotherapy TAVO demonstrating local treatment with TAVO led to the regression of untreated lesions in patients with metastatic melanoma
SAN DIEGO and PENNINGTON, N.J., Oct. 2, 2018 /PRNewswire/ — OncoSec Medical Incorporated (OncoSec) (NASDAQ:ONCS), a company developing intratumoral cancer immunotherapies, today announced that Dr. Alain Algazi, Associate Professor of Medicine at UCSF, will present data from OncoSec’s OMS-100 study of TAVO (intratumoral tavokinogene telseplasmid) as a monotherapy treatment for metastatic melanoma in an oral presentation during the Melanoma Bridge Conference being held on November 29 – December 1 in Naples, Italy.
Dr. Algazi’s recently accepted presentation, entitled Intratumoral tavokinogene telseplasmid induces abscopal clinical responses in metastatic melanoma patients, will describe the OMS-100 Phase 2 multicenter, single-group study which evaluated the efficacy and safety observed after repeat dosing and different intervals between TAVO cycles. The primary endpoint was overall response rate (ORR) by modified “skin” Response Evaluation Criteria In Solid Tumors (mRECIST). Dr. Algazi, the primary investigator, is a leading expert in oncology research as well as a Clinical Strategic Advisor and a Clinical Advisory Board member for OncoSec.
The new data will demonstrate that local treatment with TAVO alone led to whole-body immune responses associated with regression of untreated lesions in half of the 50 patients treated on the study.
“We were grateful to Dr. Algazi and the Melanoma Bridge Conference for the opportunity to share this important TAVO monotherapy data demonstrating abscopal clinical responses with the clinicians, biotechnology executives, and industry opinion leaders in attendance,” said Daniel J. O’Connor, OncoSec President and Chief Executive Officer. “Having Dr. Algazi present data from our OMS-100 study at the Melanoma Bridge Conference is an exciting opportunity for OncoSec as it serves to highlight our clinical work investigating TAVO as a monotherapy in the treatment of metastatic melanoma, as well as bring added visibility to our Phase 2b PISCES/KEYNOTE-695 clinical trial combining TAVO with pembrolizumab in metastatic melanoma for patients that have failed all available treatment options, including anti-PD-1 immunotherapy.”
OncoSec Medical News
All Day (Friday)