Alzheimer’s disease research has taken another hit with the failure of AstraZeneca and Eli Lilly’s experimental (BACE) inhibitor lanabecestat.

The firms said they would discontinue two Phase III trials of the experimental drug after an independent data monitoring committee found that they were unlikely to meet their primary endpoints.

The AMARANTH trial randomised patients with early Alzheimer’s disease to receive lanabecestat, 20mg or 50mg, or placebo orally once daily for 104 weeks, to assess the drug’s effect on change from baseline on the 13-item Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog13).

Read more: http://www.pharmatimes.com/news/az,_lilly_pull_plug_on_alzheimers_disease_trials_1239521

RSP Systems, a Danish non-invasive diagnostics company, has completed the first of two rounds of a Series B fundraising to bring its diagnostic device for diabetes closer to regulatory approval and a market launch. If successful, RSP’s device could provide a non-invasive, more pleasant alternative to blood testing for diabetes patients by using light to measure the concentration of sugar in the blood.

RSP’s technology uses a phenomenon called Raman scattering to measure blood sugar levels. Raman scattering occurs when light is shone on a molecule and scattered, changing the energy of the light. RSP uses Raman scattering to measure the concentration of a given molecule, in this case blood sugar, within cells and in fluid between tissues. The company recently published a study showing the accuracy of its technology was comparable to invasive blood sugar monitors, tested in 35 patients.

Read more: https://labiotech.eu/rsp-systems-non-invasive-glucose-monitoring/

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the US Food and Drug Administration (FDA) has accepted the company's supplemental Biologics License Application (sBLA) and granted Priority Review for Hemlibra® (emicizumab-kxwh) for adults and children with haemophilia A without factor VIII inhibitors. The sBLA is based on data from the phase III HAVEN 3 study. The FDA is expected to make a decision on approval by 4 October 2018.

"People with haemophilia A can face significant challenges in managing their condition and may need to adapt their daily lives to avoid bleeds and accommodate treatment," said Sandra Horning, MD, Roche's Chief Medical Officer and Head of Global Product Development. "We believe the FDA’s decision to grant Priority Review to Hemlibra underscores its potential to improve the standard of care for people without factor VIII inhibitors and to help reduce treatment burden by offering more flexible subcutaneous dosing options. We look forward to working with the FDA to hopefully bring Hemlibra to all people with haemophilia A as quickly as possible."

Read more: http://www.worldpharmanews.com/roche/4436-fda-grants-priority-review-to-roche-s-hemlibra-for-people-with-haemophilia-a-without-factor-viii-inhibitors

AstraZeneca’s Tagrisso has been given a green light in Europe for first-line use to treat EGFR-mutated non-small cell lung cancer (NSCLC), offering a new line of attack at this stage of the disease.

The decision, which significantly expands the drug’s reach in the EU, was based on clinical data from the Phase III FLAURA trial, showing that progression free survival in patients taking Tagrisso (osimertinib) was 18.9 months compared to 10.2 months for those receiving the standard of care, while the objective response rate was 80 percent versus 76 percent, respectively.

A further recent analysis of FLAURA results - presented at the European Lung Cancer Conference (ELCC) in Geneva - also showed that the drug’s PFS benefit over EGFR tyrosine kinase inhibitors (TKIs) was sustained throughout post-progression outcomes.

Read more: http://www.pharmatimes.com/news/eu_approves_first-line_use_of_az_tagrisso_in_lung_cancer_1239303

Xconomy Boston —

At this rate, 2018 could be a record-breaking year for biotech IPOs. Two more Boston-area companies filed their IPO plans late last week. Neon Therapeutics is developing personalized cancer vaccines, one of which is in Phase 1 clinical testing. Translate Bio is a messenger RNA (mRNA) company that started human testing just last month.

Neon and Translate each set a preliminary $115 million target for their respective IPOs, but that number is likely to change after the companies set their share price. Cambridge, MA-based Neon plans to list on the Nasdaq under the symbol “NTGN.” Translate, headquartered in Lexington, MA, has applied for a Nasdaq listing under the symbol “TBIO.”

Read more: https://www.xconomy.com/boston/2018/06/04/neon-therapeutics-translate-bio-join-the-ranks-of-ipo-hopefuls/

An experimental HIV vaccine protected animals from dozens of strains of HIV. And a human trial of the vaccine is expected to begin in the second half of 2019, according to U.S. researchers.

The vaccine targets a vulnerable site on HIV -- the virus that causes AIDS -- and triggered antibody production in mice, guinea pigs and monkeys, according to researchers with the U.S. National Institute of Allergy and Infectious Diseases (NIAID).

Read more: https://www.drugs.com/news/human-trials-set-experimental-hiv-vaccine-74828.html

An international team of researchers led by scientists at the University of Cambridge and MSD* has created the first detailed genetic map of human proteins, the key building blocks of biology. These discoveries promise to enhance our understanding of a wide range of diseases and aid development of new drugs.

The study, published in the journal Nature, characterised the genetic underpinnings of the human plasma 'proteome', identifying nearly 2,000 genetic associations with almost 1,500 proteins. Previously, there was only a small fraction of this knowledge, mainly because researchers could measure only a few blood proteins simultaneously in a robust manner.

The researchers used a new technology ("SOMAscan") developed by a company, SomaLogic, to measure 3,600 proteins in the blood of 3,300 people. They then analysed the DNA of these individuals to see which regions of their genomes were associated with protein levels, yielding a four-fold increase on previous knowledge.

Read more: https://www.sciencedaily.com/releases/2018/06/180606132715.htm

The most valuable drug in the pharma industry’s pipeline isn’t a new medicine for diabetes or cancer, but Vertex’s triple therapy for rare hereditary disease cystic fibrosis, according to EvaluatePharma.

That assertion ties in with its new report on worldwide prescription drug sales, which says that drugs for orphan diseases will outperform the overall pharma market, almost doubling to reach a whopping $262bn in 2024 when they will account for a fifth of all drug sales.
“This highlights the industry’s continued move to address small groups of neglected patients with high unmet need and to benefit from traditionally reduced payer scrutiny on orphan drugs, as well as regulatory and financial incentives,” says EP.

Read more: http://www.pmlive.com/pharma_news/drugs_for_rare_diseases_will_help_propel_pharma_sales_to_$1.2trn_by_2024_report_1238997

The U.S. Food and Drug Administration announced that it has warned nine online networks, operating a total of 53 websites, that they must stop illegally marketing potentially dangerous, unapproved and misbranded versions of opioid medications, including tramadol and oxycodone. Companies who fail to correct the violations, as outlined in the warning letters, may be subject to enforcement action, including product seizure or injunction.

"The FDA is taking additional steps to protect U.S. consumers from illicit opioids by targeting the websites that illegally market them and other illicit drugs. The internet is virtually awash in illegal narcotics and we’re going to be taking new steps to work with legitimate internet firms to voluntarily crack down on these sales. As part of that effort, we’re hosting a summit with internet stakeholders to find new ways to work collaboratively with them to address these issues. At the same time, we’ll be taking action against firms whose websites deliberately break the law," said FDA Commissioner Scott Gottlieb, M.D. "This illegal online marketing of unapproved opioids is contributing to the nation’s opioid crisis. Today’s warning letters go right to the source of this illegal activity to let online network operators know that marketing illegal and unapproved opioids directly to U.S. consumers will not go unchallenged by the FDA. Opioids bought online may be counterfeit and could contain other dangerous substances. Consumers who use these products take significant risk with their lives. The new warning letters are part of a comprehensive campaign to target illegal sales of unapproved opioids. We’ll be following these actions with additional steps in coming months to crack down on the flow of illegal, unapproved opioids sold online and shipped through the mail."

Read more: http://www.worldpharmanews.com/fda/4432-fda-takes-action-against-53-websites-marketing-unapproved-opioids-as-part-of-a-comprehensive-effort-to-target-illegal-online-sales

The U.S. Food and Drug Administration announced that it has warned nine online networks, operating a total of 53 websites, that they must stop illegally marketing potentially dangerous, unapproved and misbranded versions of opioid medications, including tramadol and oxycodone. Companies who fail to correct the violations, as outlined in the warning letters, may be subject to enforcement action, including product seizure or injunction.

"The FDA is taking additional steps to protect U.S. consumers from illicit opioids by targeting the websites that illegally market them and other illicit drugs. The internet is virtually awash in illegal narcotics and we’re going to be taking new steps to work with legitimate internet firms to voluntarily crack down on these sales. As part of that effort, we’re hosting a summit with internet stakeholders to find new ways to work collaboratively with them to address these issues. At the same time, we’ll be taking action against firms whose websites deliberately break the law," said FDA Commissioner Scott Gottlieb, M.D. "This illegal online marketing of unapproved opioids is contributing to the nation’s opioid crisis. Today’s warning letters go right to the source of this illegal activity to let online network operators know that marketing illegal and unapproved opioids directly to U.S. consumers will not go unchallenged by the FDA. Opioids bought online may be counterfeit and could contain other dangerous substances. Consumers who use these products take significant risk with their lives. The new warning letters are part of a comprehensive campaign to target illegal sales of unapproved opioids. We’ll be following these actions with additional steps in coming months to crack down on the flow of illegal, unapproved opioids sold online and shipped through the mail."

Read more: http://www.worldpharmanews.com/fda/4432-fda-takes-action-against-53-websites-marketing-unapproved-opioids-as-part-of-a-comprehensive-effort-to-target-illegal-online-sales

A novel approach to immunotherapy developed by researchers at the National Cancer Institute (NCI) has led to the complete regression of breast cancer in a patient who was unresponsive to all other treatments. This patient received the treatment in a clinical trial led by Steven A. Rosenberg, M.D., Ph.D., chief of the Surgery Branch at NCI's Center for Cancer Research (CCR), and the findings were published June 4, 2018 in Nature Medicine. NCI is part of the National Institutes of Health.
"We've developed a high-throughput method to identify mutations present in a cancer that are recognized by the immune system," Dr. Rosenberg said. "This research is experimental right now. But because this new approach to immunotherapy is dependent on mutations, not on cancer type, it is in a sense a blueprint we can use for the treatment of many types of cancer."

The new immunotherapy approach is a modified form of adoptive cell transfer (ACT). ACT has been effective in treating melanoma, which has high levels of somatic, or acquired, mutations. However, it has been less effective with some common epithelial cancers, or cancers that start in the lining of organs, that have lower levels of mutations, such as stomach, esophageal, ovarian, and breast cancers.

In an ongoing phase 2 clinical trial, the investigators are developing a form of ACT that uses tumor-infiltrating lymphocytes (TILs) that specifically target tumor cell mutations to see if they can shrink tumors in patients with these common epithelial cancers. As with other forms of ACT, the selected TILs are grown to large numbers in the laboratory and are then infused back into the patient (who has in the meantime undergone treatment to deplete remaining lymphocytes) to create a stronger immune response against the tumor.

Read more: http://www.worldpharmanews.com/research/4433-new-approach-to-immunotherapy-leads-to-complete-response-in-breast-cancer-patient

Boehringer Ingelheim’s Spiriva Respimat is now licensed for use in the UK to treat people with asthma aged six years and older.

The therapy can be prescribed as add-on maintenance bronchodilator treatment in young patients with severe asthma who have experienced one or more severe asthma exacerbations in the past year.

The firm notes that the UK has one of the highest prevalence rates for paediatric asthma worldwide, with one in 11 children affected, while it is estimated that a child is admitted to hospital every 20 minutes because of an asthma attack.

Sprivia (triotropium respimat) is part of a class of medicines called long-acting muscarinic antagonists (LAMA), and works by preventing the air passages closing, improving airflow in and out of the lungs.

Read more: http://www.pharmatimes.com/news/boehingers_spiriva_gets_uk_license_for_young_asthma_patients_1238993

US regulators have approved a combination of Eli Lilly’s Alimta and MSD’s Keytruda as first-line treatment for patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), irrespective of PD-L1 expression status.

The combination was approved via the US Food and Drug Administration’s accelerated approval pathway based on Phase II tumour response rate and progression-free survival (PFS) data observed in a cohort of Merck's KEYNOTE-021 study, and so its clinical benefit may still need to be verified in confirmatory trials.

In the trial, the combination of Alimta (pemetrexed), carboplatin and Keytruda (pembrolizumab) showed a statistically significant improvement in objective response rate (ORR) versus Alimta plus carboplatin alone (55 percent vs 29 percent), while median progression free survival came in at 13.0 months and 8.9 months, respectively.

Read more: http://www.pharmatimes.com/news/us_nod_for_alimtakeytruda_lung_cancer_combo_1238721

Loxo Oncology and Bayer should hear from the FDA about their tumour biomarker-targeted cancer drug larotrectinib by 26 November, after picking up a priority review by the US regulator.
TRK inhibitor Larotrectinib is being reviewed by the US regulator for adult and paediatric patients with locally advanced or metastatic solid tumours with a neurotrophic tyrosine receptor kinase (NTRK) gene fusion biomarker, a departure from the usual regulatory route of seeking approval based on the organ or tissue where the cancer starts to grow.
In trials presented at last year’s American Society of Clinical Oncology (ASCO) meeting and since published in the New England Journal of Medicine (NEJM), larotrectinib achieved a 75% overall response rate (ORR) in TRK fusion cancers, including 13% complete responses and 62% partial responses.

Read more: http://www.pmlive.com/pharma_news/loxo,_bayer_get_quick_fda_review_for_precision_cancer_drug_1237511

Immunizing girls against human papillomavirus (HPV) doesn't increase their risk for autoimmune diseases, according to new research from Canada.

HPV is the world's most common sexually transmitted disease, affecting up to 75 percent of sexually active people and is the main cause of cervical cancer. Gardisil, the quadrivalent HPV4 vaccine, protects against up to 90 percent of strains that cause cervical and rectal cancer.

"Despite demonstrated effectiveness in real-world settings, concerns continue to persist regarding the safety of the HPV4 vaccine," said lead study author Dr. Jeffrey Kwong. He's a senior scientist at the Institute for Clinical Evaluative Sciences in Toronto and at Public Health Ontario.

Read more: https://www.drugs.com/news/no-link-between-hpv-vaccine-autoimmune-diseases-study-74593.html

Novartis announced today new real world evidence from the CHAMP-HF registry comparing Entresto® (sacubitril/valsartan) patients to patients not taking Entresto.[1] This pre-specified analysis of an interim data cut from the CHAMP-HF registry showed that chronic heart failure (HF) patients with reduced ejection fraction (HFrEF) taking Entresto reported early, statistically significant improvement in health status, as measured by the KCCQ-12 overall summary score (KCCQ-OS). [1] This finding was driven by statistically significant improvements in symptom frequency and quality of life domains of the KCCQ-12.[1] The study findings were presented today by lead investigator Yevgeniy Khariton, MD, MSc, Saint Luke's Hospital, Mid-America Heart Institute, University of Missouri-Kansas City, as a part of a late-breaking session at the European Society of Cardiology Heart Failure (ESC-HF) Congress in Vienna, Austria.

Read more: http://www.worldpharmanews.com/novartis/4420-patient-reported-outcomes-tool-revealed-significant-improvement-in-symptom-frequency-and-quality-of-life-domains-with-entresto

US regulators have expanded the reach of UCB’s Cimzia, allowing its use to treat adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy.

The anti-inflammatory is already on the market for use in rheumatoid arthritis, active psoriatic arthritis, ankylosing spondylitis and axial spondyloarthritis.

This latest approval makes Cimzia (certolizumab pegol) the first Fc-free, PEGylated anti-TNF treatment option for this indication and marks UCB’s entry into immuno-dermatology, “where significant unmet need currently exists,” the Beligian drugmaker noted.

The drug’s expanded label rides on the back of positive data from a Phase III clinical development programme consisting of CIMPASI-1, CIMPASI-2 and CIMPACT trials, which enrolled over 1,000 patients, of whom nearly one third had prior biologic exposure.

Read more: http://www.pharmatimes.com/news/us_approves_ucbs_cimzia_for_psoriasis_1237343

Abbott (NYSE:ABT) said yesterday that it won FDA approval for its Xience Sierra everolimus-eluting coronary stent.

The company’s device features a thin profile, improved flexibility and longer lengths than previous stents. The device and delivery system were designed to help clinicians tackle complex cases, like those involving multiple or totally blocked vessels, according to Abbott.

“We developed XIENCE Sierra so that physicians can more easily deliver the stent even in challenging cases,” Chuck Brynelsen, SVP of Abbott’s vascular unit, said in prepared remarks. “The updated design and improved deliverability mean doctors can access and unblock difficult-to-treat lesions with more flexibility and precision than other stents.”

Abbott won CE Mark clearance for Xience Sierra in October last year and regulatory approval in Japan earlier this year. The device has been used in more than 8 million people worldwide, Abbott touted.

Read more: https://www.drugdeliverybusiness.com/fda-approves-abbotts-latest-drug-eluting-stent/

Bluebird Bio has claimed an FDA breakthrough designation for its Lenti-D gene therapy for cerebral adrenoleukodystrophy (CALD) – a rare neurological disease mainly affecting young males.
The new status – which could result in a speedy review by the US regulator and shorten the time to market for the therapy – comes after impressive data from a phase II/III trial suggesting it could stabilise disease for more than two years in boys with the highly progressive and life-threatening disorder.
Data from 17 patients in the STARBEAM study was published in the New England Journal of Medicine last October, and showed that Lenti-D (elivaldogene tavalentivec) stabilised disease in 15 of them, with one boy dying as a result of the disease and another progressing despite therapy.

Read more: http://www.pmlive.com/pharma_news/bluebird_bags_breakthrough_status_for_cald_gene_therapy_1236797

Pfizer Inc. (NYSE:PFE) announced that it has started a Phase 1/2 trial of its respiratory syncytial virus (RSV) vaccine candidate in healthy adult volunteers. RSV is a common respiratory virus that affects the lungs and airways, with significant impact on young children and older adults. RSV affects 33 million children globally and leads to approximately 120,000 childhood deaths every year. (1) In the United States approximately 177,000 older adults are hospitalized annually because of RSV.(2) The highest risk of severe outcome from RSV occurs in the first months of life.

Read more: http://www.worldpharmanews.com/pfizer/4414-pfizer-begins-a-phase-1-2-study-to-evaluate-respiratory-syncytial-virus-rsv-vaccine

Glad to be an official member of the PharmeMed group!

US regulators have approved a new indication for Amgen’s Prolia, allowing its use to treat glucocorticoid-induced osteoporosis (GIOP) in men and women at high risk of fracture.

The decision allows physicians to prescribe the drug to patients with a history of osteoporotic fracture, those with multiple risk factors for fracture, or patients who have failed or are intolerant to other available osteoporosis therapy.

The approval is based on Phase III data showing that patients who received Prolia (denosumab) had greater gains in bone mineral density (BMD) compared to those who received its Actonel (risedronate).

Read more: http://www.pharmatimes.com/news/fda_clears_amgens_prolia_for_glucocorticoid_osteoporosis_1236621

The U.S. Food and Drug Administration today approved Doptelet (avatrombopag) tablets to treat low blood platelet count (thrombocytopenia) in adults with chronic liver disease who are scheduled to undergo a medical or dental procedure. This is the first drug approved by the FDA for this use.

GlaxoSmithKline’s inhaled corticosteroid Arnuity Ellipta has been approved in the US for paediatric use.

US regulators have issued a green light for the once-daily inhaler as maintenance treatment of asthma in children from as young as five years.

The submission was supported by data from a pivotal study pitting the efficacy and safety of Arnuity Ellipta (fluticasone furoate) against placebo in 593 children aged 5 to 11 years (inclusive) with asthma.

Read more: http://www.pharmatimes.com/news/us_clears_paediatric_use_of_gsks_arnuity_ellipta_1236328

The New England Journal of Medicine:

The drug-abuse epidemic in the United States has led to an increased number of organ donors dying from drug intoxication.1,2 We investigated the effect of drug intoxication–related deaths on organ donation and outcomes after transplantation by analyzing data on donors and transplantation over a period of 17 years in the United States as compared with Eurotransplant (a collective of all transplantation centers in eight European countries). We ascertained the proportion of recovered organs that were from adult donors who died as a result of drug intoxication. We also examined survival after heart or lung transplantation among the recipients of such allografts, since these organs are especially sensitive to ischemic injury that results from hypoxia or hypotension, conditions that are typically noted in persons who die from drug intoxication.3-5 The Cochran–Armitage statistic was used to describe differences in organ recovery over time. A multivariable Cox regression model was constructed to assess the independent effect of donor cause of death on post-transplantation survival with the use of the log-rank test. Monthly survival was estimated by the Kaplan–Meier method and interpolated linearly.

Read more: https://www.nejm.org/doi/full/10.1056/NEJMc1802706