Alnylam price target raised to $183 at BMO Capital on early Oxlumo approval » 11:5111/2411/24/20
BMO Capital analyst Do…
BMO Capital analyst Do Kim raised the firm's price target on Alnylam to $183 from $182 and keeps an Outperform rating on the shares following EMA and FDA approvals of Oxlumo ahead of the December 3 PDUFA date. The analyst's probability of success for Oxlumo has been raised to 100%, which is partly offset by a lower annual gross price of $490K and net price of $380K. The analyst says Oxlumo will be available by the end of the year and Kim expects meaningful revenue contribution beginning in 1Q21.
Piper reiterates Overweight rating on Alnylam after Oxlumo approved » 10:2711/2411/24/20
Piper Sandler analyst…
Piper Sandler analyst Edward Tenthoff noted that the FDA approved Alnylam's Oxlumo nearly two weeks ahead of the December 3 PDUFA date and following the EU approval of the drug last week. The analyst, who now projects net Oxlumo sales of $526,000 in Q4, growing to $48M in 2021, reiterates an Overweight rating and $169 price target on Alnylam shares.
Alnylam announces new framework for VBAs to help PH1 patients get Oxlumo » 07:1711/2411/24/20
Alnylam announced today a…
Alnylam announced today a new framework for value-based agreements, or VBAs, designed to help people with primary hyperoxaluria type 1, or PH1, gain access to Oxlumo. Now approved by the FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients, Oxlumo is the first-ever approved targeted therapeutic that substantially curbs oxalate production in patients living with PH1, an ultra-rare genetic disease characterized by oxalate overproduction. Alnylam is in active discussions with payers and has reached an agreement in principle with Express Scripts, Harvard Pilgrim, and Highmark to pursue VBAs for Oxlumo. The company has worked with payers on a new and enhanced VBA framework. Since Oxlumo is indicated for both pediatric and adult patients, and is dosed by weight, related costs can vary relative to each patient and use over time. As such, Alnylam has structured a new VBA component that specifically addresses many payers' concerns for budget predictability and value, particularly for ultra-rare orphan disease therapies that are administered across a wide spectrum of ages from infants to adults. The new VBA component, called a patient need adjustment, or PNA, is now being added to Alnylam's overall VBA offering for Oxlumo. Participating payers will qualify for the PNA rebate if the average number of vials utilized by a plan member exceeds an established threshold, providing payers with greater short-term and long-term predictability. The PNA was designed to mitigate the risk of escalating or varying costs associated with dosing requirements, thereby accelerating access for people diagnosed with PH1. To further address budget predictability, Alnylam is also making available its prevalence based adjustment, or PBA, component, which was first introduced last year for Alnylam's second-approved therapy, Givlaari. There are often uncertainties in diagnosis rates and disease prevalence estimates in ultra-rare, poorly diagnosed orphan diseases, making it challenging for payers to predict the number of patients who will be covered within their plans. This feature triggers a rebate to participating payers if the number of diagnosed patients they cover exceeds current epidemiologic estimates for PH1.
Alnylam announces FDA approval for Oxlumo injection for PH1 treatment » 07:1111/2411/24/20
Alnylam announced that…
Alnylam announced that the FDA approved Oxlumo injection for subcutaneous use, the first-ever therapy available for the treatment of primary hyperoxaluria type 1, or PH1, to lower urinary oxalate levels in pediatric and adult patients. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction. The excess production of oxalate results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis, progression to kidney failure, and systemic organ dysfunction. In ILLUMINATE-A - the largest controlled Phase 3 study ever conducted in PH1 - Oxlumo was shown to significantly reduce levels of urinary oxalate relative to placebo, with the majority of patients achieving normal or near-normal levels.
Alnylam gets FDA approval for Oxlumo, Bloomberg reports 18:5811/2311/23/20
VBI submits MAA to EMA for for 3-antigen prophylactic hepatitis B vaccine » 08:0711/2311/23/20
VBI Vaccine announced the…
VBI Vaccine announced the submission of a Marketing Authorization Application, MAA, to the European Medicines Agency, EMA, for the Company's 3-antigen prophylactic hepatitis B vaccine candidate, seeking approval for the prevention of infection caused by all known subtypes of the hepatitis B virus in adults. "We believe our 3-antigen prophylactic hepatitis B vaccine has the potential to be an important intervention in the fight to eliminate Hepatitis B infections and this MAA submission is a significant milestone for the product and VBI more generally," said Jeff Baxter, President & CEO. "We are committed to working collaboratively with the EMA throughout the regulatory process to provide access to this vaccine in Europe. As part of the global commercialization strategy for this vaccine, we also remain on track to submit the Biologics License Application to the U.S. Food and Drug Administration in the next couple of weeks."
|Over a week ago|
VBI Vaccines announces data from ongoing VBI-1901 study at SNO » 08:0511/1911/19/20
VBI Vaccines announced…
VBI Vaccines announced Phase 2a data from its ongoing Phase 1/2a study of VBI-1901, the company's cancer vaccine immunotherapeutic candidate designed to target cytomegalovirus, or CMV, as a foreign viral antigen in recurrent glioblastoma, or GBM. The data were presented in an e-poster at the Society for Neuro-Oncology, or SNO, annual meeting. Two partial responses, or PRs, and two stable disease, or SD, were observed in the VBI-1901 + GM-CSF vaccinated group, resulting in a disease control rate of 40%. A 56% disease control rate achieved in the group vaccinated with VBI-1901 + AS01, with five stable disease observations, tumor response data for the 10th patient enrolled is pending. Presumed pseudoprogression was observed in both vaccinated groups- defined as immune infiltration into the tumor which appears initially as tumor growth, but later subsides resulting in tumor growth stabilization and/or shrinkage. Based on the available data, VBI is exploring a randomized, controlled clinical study, including a potential registration study, for the next phase of development, which could begin in 2021, pending approval from regulatory bodies.
Alnylam gets EU approval for Oxlumo for hyperoxaluria type 1 treatment » 07:0711/1911/19/20
Alnylam Pharmaceuticals …
Alnylam Pharmaceuticals announced that the European Commission, EC, has granted marketing authorization for OXLUMO, lumasiran, an RNAi therapeutic, for the treatment of primary hyperoxaluria type 1, PH1, in all age groups. PH1 is an ultra-rare orphan disease characterized by excessive oxalate production, which can lead to life threatening end-stage renal disease, ESRD, and other systemic complications. Heterogeneity in disease manifestation often contributes to delays in diagnosis - particularly in adult PH1 patients, with a median time from symptoms onset to diagnosis of approximately six years. Untreated PH1 leads to progressive kidney damage; patients with advanced kidney disease require intensive dialysis to help filter waste products, including oxalate, from their blood until they are able and eligible to receive a dual or sequential liver/kidney transplant, an invasive procedure associated with a high risk of morbidity and mortality, and life-long immunosuppression. "Prior to now there have been no approved treatment options for PH1 in Europe, so this is a potentially life-changing milestone for people diagnosed with this ultra-rare, debilitating disease - many of whom are infants and children - and their families. Lumasiran will address the urgent unmet need that exists for patients with PH1 and its approval today marks our continued commitment to rare disease communities," said John Maraganore, Ph.D., Chief Executive Officer, Alnylam Pharmaceuticals. "Alnylam has taken lumasiran from identification of compound to regulatory approval in just six years and we will progress with the same sense of urgency as we work with national reimbursement bodies across Europe to bring lumasiran to patients."
VBI Vaccines announces results from VBI-2601 study » 08:1211/1811/18/20
VBI Vaccines announced…
VBI Vaccines announced interim clinical results from the ongoing Phase 1b/2a study of VBI-2601, a novel recombinant, protein-based immunotherapeutic candidate for the treatment of chronic hepatitis B virus, or HBV, infection, in development in collaboration with Brii Biosciences. The low-dose cohorts of the ongoing two-part Phase 1b/2a evaluated VBI-2601, unadjuvanted and adjuvanted, in combination with a nucleoside analogue, or NUC, therapy, intended to reduce HBV DNA, vs. NUC therapy only, in chronically-infected HBV patients. Objectives of this early-stage study were to elicit immunologic responses known to be associated with functional immunity against HBV infection, including the stimulation of T cell immunity and the induction of antibody responses to HBV surface antigens. The interim data showed potent re-stimulation of T cell responses to HBV surface antigens seen in 67% and 78% of evaluable patients in the low-dose VBI-2601 unadjuvanted and adjuvanted study arms, respectively. Antibody responses against HBV surface antigens were observed in 60% of evaluable patients in the unadjuvanted cohort and in 67% in the adjuvanted cohort. The low-dose, with and without the adjuvant, was well-tolerated with no safety signals observed. The Phase 1b/2a clinical study of VBI-2601 is a randomized, controlled study designed to assess the safety, tolerability, and antiviral activity of VBI-2601 in patients with chronic HBV infection. The study is designed as a two-part dose-escalation study assessing a low dose and a high dose of VBI-2601, with and without an undisclosed adjuvant, and has enrolled 46 patients. The HBV patients enrolled in the low-dose cohort of the study, including both E+ and E- patients, had baseline mean circulating levels of S-antigen of approximately 3 logs. This study was conducted by our partner Brii Biosciences. Based on the results from this study, Brii Biosciences is planning to conduct additional combinations studies of VBI-2601 within Asia-Pacific Economic Cooperation (APEC) and the greater China areas.
Arbutus Biopharma announces additional HBsAg decline data with AB-729 » 07:4911/1611/16/20
Arbutus Biopharma Corporation announced additional clinical data from an ongoing Phase 1a/1b clinical trial with AB-729, its proprietary GalNAc delivered RNAi compound. The new data described expands on the presentation entitled Safety and pharmacodynamics of the GalNAc-siRNA AB-729 in subjects with chronic hepatitis B infection, recorded on October 14, 2020 and presented on November 15, 2020 by Professor Man-Fung Yuen, D.Sc., M.D., Ph.D., from the University of Hong Kong at The Liver Meeting Digital ExperienceTM, The American Association for the Study of Liver Diseases Meeting. The new data summarized below include HBsAg data for the complete 60 mg every 4 weeks multi-dose cohort at week 20, and the first results for the AB-729 90 mg single-dose cohort of HBV DNA positive subjects. Repeat dosing of AB-729 60 mg every 4 weeks results in continuous declines in mean HBsAg through week 20. Dr. Gaston Picchio, Chief Development Officer at Arbutus stated, "Further follow up of the 60 mg every 4 weeks multi-dose cohort confirmed continuous reductions in mean HBsAg at week 20, and in a subset of subjectsbeyond this time point, while being generally safe and well tolerated. Additionally, the mean HBsAg declines and slopes of declines are similar between single doses and repeat doses of AB-729 up to week 12. Importantly, this suggests that dosing AB-729 as frequently as every 4 weeks may not be necessary, and that AB-729 has the potential to be dosed every 8 weeks or even every 12 weeks. This dosing strategy is being investigated in other cohorts of the trial with results from the 60 mg every 8 week cohort expected before the end of 2020." AB-729 90 mg single-dose reduces HBsAg and HBV DNA in HBV DNA positive chronic Hepatitis B subjects with mean HBsAg declines similar to those seen in HBV DNA negative subjects: Dr. Picchio added, "It is also encouraging to observe that a single 90 mg dose of AB-729 is capable of reducing HBsAg in HBV DNA positive subjects to the same extent achieved in other single-dose HBV DNA negative cohorts. Further, a single 90 mg AB-729 dose substantially reduced HBV DNA as well as HBV RNA and HBcrAg." AB-729 was safe and well tolerated after single and repeat doses, No serious adverse events or discontinuations due to adverse events. No treatment-related Grade 3 or 4 adverse events.