Nick Morrison, M.D., Center for Vein Restoration, Mesa, AZ, presented at the 2019 Charing Cross Symposium in London. The new data demonstrate durable and consistent long-term outcomes of the Medtronic VenaSeal Closure System in treating patients with chronic venous disease. The VeClose Extension Study is a follow-on study with the purpose of evaluating long-term outcomes at five years post-treatment with the VenaSeal Closure System. The aggregate complete closure rate of the great saphenous vein, or GSV, was 94.6% in the VenaSeal subjects who completed five-year follow-up, including 47 randomized subjects and nine roll-in subjects. In the original VeClose Study, patients were randomized to receive treatment with VenaSeal or the Medtronic ClosureFast radiofrequency ablation, or RFA, device. By Kaplan-Meier analysis of the randomized cohorts to evaluate success over the life of the study through five years, vein closure estimates were 91.4% for VenaSeal and 85.2% for RFA, demonstrating continued non-inferiority of VenaSeal to RFA through five years. Patient improvement was rated on three assessments: Venous Clinical Severity Score, or VCSS, a clinical venous disease assessment; and the Aberdeen Varicose Vein Questionnaire, or AVVQ, and EQ-5D – both of which provide patient-reported quality of life, or QoL, outcomes. By all measurements, subjects sustained or maintained improvements in QoL after treatment with either VenaSeal or RFA. These first prospective five-year data for VenaSeal demonstrate a strong clinical portfolio with sustained long-term outcomes. “The five-year data support the safety, effectiveness, and quality of life-enhancing capability of both VenaSeal and ClosureFast in treating patients with chronic venous disease,” said Dr. Morrison. “Furthermore, the data demonstrate long-term, strong, and consistent outcomes. The industry will benefit from long-term data like this so that physicians and patients can be confident in their treatment choice.”
Wednesday, April 17, 2019
Ionis’ Tegsedi gets a U.K. boost after NICE reverses initial guidance
The path for Ionis’ Tegsedi in the U.K. looked rocky after a preliminary “no” last year from England’s drug-price watchdog. Fortunately for the drugmaker, that decision was reversed.
Tuesday, the National Institute for Health and Care Excellence (NICE) released (PDF) final guidance recommending Tegsedi’s placement on England’s National Health Service for treatment of nerve damage from hereditary transthyretin-mediated amyloidosis (ATTR), an ultra-rare disease that affects about 150 U.K. citizens, NICE said in an email.
NICE reversed its early guidance from October 2018 not recommending Tegsedi, which is in a head-to-head battle with Alnylam Pharmceuticals’ Onpattro, saying the drug’s cost efficiency over palliative care had improved after the drugmaker agreed to a steeper discount for its £5,925 ($7,737)-per-week treatment.
In its final guidance, NICE said the drug slowed the progression of ATTR, which causes the production of abnormal transthyretin protein in the liver, but had unclear long-term effects on top of an “uncertain” economic model. However, the price break Ionis served up was enough for a vote of confidence.
“Some assumptions in the economic modelling are also uncertain, particularly around the utility values and the healthcare costs,” the guidance said. “Despite the uncertainties, (Tegsedi) is likely to provide important clinical benefits for people with (ATTR) and value for money within the context of a highly specialized service.”
Prior to the introduction of Tegsedi and Onpattro, the only treatment for ATTR was supportive care.
While Tegsedi has had a bumpy ride into the English market, rival Onpattro’s path hasn’t been a cakewalk, either. Alnylam’s entry into the field received a similar “no” from NICE in October 2018 after the watchdog said the drug’s cost efficiency ratio per quality-adjusted life year gained—a metric NICE uses to establish cost-effectiveness—wasn’t up to snuff.
That knock in the U.K. compounded a rough first quarter of sales for Onpattro, which received FDA approval in August 2018. According to the company, Onpattro pulled in just $460,000 in revenue between its approval month and the end of the third quarter.
Tegsedi received its FDA approval in October 2018.
The two companies could soon see a third challenger enter the ring as Pfizer’s tafamidis seeks its own FDA approval to treat ATTR. In August, the heavyweight drugmaker announced positive phase 3 trial data for ATTR cardiomyopathy—a different form of the disease, but one in which existing competitors hope to eventually compete.
Precision BioSciences Starts Off-The-Shelf CAR T Cell Therapy Phase 1/2a Trial
Precision BioSciences (Nasdaq: DTIL) (“Precision”), a genome editing company dedicated to improving life (DTIL) through its proprietary ARCUS® genome editing platform, announced today it has dosed the first patient in the Phase 1/2a clinical trial of PBCAR0191, its first gene-edited allogeneic anti-CD19 chimeric antigen receptor (CAR) T cell product candidate. Precision is developing PBCAR0191 in collaboration with Servier, an international pharmaceutical company. PBCAR0191 is made from donor-derived T cells that are modified using Precision’s ARCUS genome editing technology. These edits are designed to generate CAR T cells that specifically recognize CD19, an important target in several B-cell cancers, and to prevent graft-versus-host disease, a significant complication associated with existing donor-derived cell-based therapies. This CAR T cell product candidate is being evaluated in adult patients with relapsed or refractory (“R/R”) non-Hodgkin lymphoma (“NHL”) or R/R B-cell precursor acute lymphoblastic leukemia (B-ALL) as an off-the-shelf cell therapy. The first patient dosed in this trial is being treated for R/R NHL, and Precision believes this is the first U.S.-based clinical trial to evaluate an allogeneic CAR T therapy for NHL.
This multi-center, open label study of PBCAR0191 is expected to enroll up to 80 patients and several dose levels of PBCAR0191 will be investigated. Clinical sites include City of Hope, Moffit Cancer Center, Dana-Farber Cancer Institute and MD Anderson Cancer Center. The primary objective of the trial is to evaluate the safety of PBCAR0191 and determine the maximum tolerated dose. Secondary objectives include evaluating the anti-tumor activity of PBCAR0191. Precision will also evaluate the expansion, trafficking and persistence of PBCAR0191 in treated patients. Lymphodepletion will be conducted several days prior to PBCAR0191 infusion. Patient outcomes will be collected for up to one year.
Roche ups outlook as sales growth offsets price hit
Swiss drugmaker Roche lifted its 2019 outlook on Wednesday, as sales growth in China and the United States outpaced price declines it has suffered amid President Donald Trump’s pressure on the industry to hold down charges.
Roche, the world’s biggest cancer drug maker, now sees mid single-digit revenue growth, up from the previous forecast of low- to mid-single digit growth. It sees core earnings per share mirroring sales growth.
Drugs division chief Bill Anderson said Roche’s drug revenue is soaring even as it honours a price-freeze pledge it made last year after Trump took to the phone and Twitter to urge companies to back off price hikes.
“We haven’t taken any price increases in the U.S. since July 1,” Anderson said on a call. “Overall in the world, our 10 percent sales growth in pharma was based on 15 percent volume growth and 5 percent average price declines across the portfolio.”
Chief Executive Severin Schwan is getting a lift as sales of newer drugs offset erosion of older cancer drugs that face fierce competition in Europe.
Multiple sclerosis drug Ocrevus’s sales rose by two-thirds to 836 million francs, while cancer immunotherapy Tecentriq jumped two-fold to 336 million. Breast cancer medicine Perjeta rose 41 percent to 870 million francs and Hemlibra, Roche’s new haemophilia A drug, hit 219 million francs.
“I’m very pleased that 90 percent, the vast majority of our growth, comes from our new medicines,” Schwan said.
Still, a big unknown for the Austrian CEO is how swiftly rivals’ soon-to-be-launched copies of Roche’s $22 billion-per-year cancer medicines Rituxan, Herceptin and Avastin will eat into U.S. sales this year and next.
“Roche can keep up the unbroken growth, as long as Ocrevus, Hemlibra and the entire (breast cancer) franchise maintain their momentum,” Zuercher Kantonalbank analyst Michael Nawrath said.
Schwan expects the $4.3 billion takeover of Philadelphia-based Spark Therapeutics to be completed by June, despite a regulatory hiccup with the U.S. Federal Trade Commission that required an extension.
“Our firm expectation is it will close at the conditions we have offered,” the CEO said.
First-quarter sales rose 8 percent to 14.8 billion Swiss francs (£11.3 billion), beating the 14.2 billion Infront Data poll average estimate. Drug sales rose 10 percent to 11.9 billion francs, besting the 11.2 billion poll average, while diagnostics sales edged higher.
April 2019 Life Science IPOs
| DATE | COMPANY NAME | AMOUNT (USD) | COMPANY HQ | RESEARCH FOCUS |
|---|---|---|---|---|
| 4/12/19 | NextCure | $86.25M Filed | Beltsville, MD | Immunomedicines to treat cancer and other immune-related diseases |
| 4/12/19 | Cortexyme | $86.25M Filed | South San Francisco, CA | Novel treatments for altering the course of neurodegenerative diseases |
| 4/12/19 | Applied Therapeutics | $86.25M Filed | New York City, NY | Cutting-edge technology to validated drug targets in areas of high unmet medical need |
| 4/12/19 | Milestone Pharmaceuticals | $86.25M Filed | Montreal, Quebec, Canada | Developing and commercializing etripamil for the treatment of cardiovascular indications |
| 4/5/19 | TransMedics | $86.25M Filed | Andover, MA | Organ transplant therapy for end-stage organ failure patients across multiple disease states |
| 4/5/19 | Trevi Therapeutics | $86.25M Filed | New Haven, CT | Nalbuphine ER to treat serious neurologically mediated conditions |
| 4/5/19 | Guardion Health Sciences, Inc. | $6M Priced | San Diego, CA | Targeted ocular nutrition and diagnostic technology for eye diseases |
| 4/4/19 | Silk Road Medical | $120M Priced | Sunnyvale, CA | Medical devices to improve the treatment of carotid artery disease |
| 4/4/19 | NGM Biopharmaceuticals | $106.7M Priced | South San Francisco, CA | Transformative therapeutics for cardio-metabolic, liver, oncologic, and ophthalmic diseases |
Tuesday, April 16, 2019
Agilent receives FDA approval for PD-L1 IHC 22C3 pharmDx assay
Agilent Technologies (A) announced that the FDA has updated its approval of the company’s PD-L1 IHC 22C3 pharmDx assay. The assay can now be used as a companion diagnostic to identify a broader range of patients with stage III or metastatic non-small cell lung cancer, or NSCLC, for first-line treatment with KEYTRUDA, a targeted anti-PD-1 therapy manufactured by Merck (MRK). The FDA previously approved the assay to identify metastatic NSCLC patients whose tumors express PD-L1 Tumor Proportion Score of 50% or more for first-line treatment with KEYTRUDA. Now, patients with stage III NSCLC who are not candidates for surgical resection or definitive chemoradiation, or metastatic NSCLC, and whose tumors express PD-L1 TPS of 1% or more are eligible for first-line treatment. This expanded indication enables pathologists to identify a larger population of previously untreated patients who are now eligible for treatment with KEYTRUDA.
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