CHMP says no to Amgen’s osteoporosis drug Evenity

Amgen and UCB have suffered another setback as they try to build momentum for Evenity, their new antibody therapy for osteoporosis.

The EMA’s Committee for Medicinal Products for Human Use (CHMP) has decided it cannot approve Evenity (romosozumab), issuing a negative opinion on the drug for the treatment of severe osteoporosis in postmenopausal women who are at risk of fracture.

It’s a blow to Amgen and Belgian partner UCB, who have been battling to get Evenity approved and onto the market for several years in order to provide a replacement to Amgen’s blockbuster osteoporosis Prolia/Xgeva (denosumab).

Prolia, a RANK inhibitor, is one of Amgen’s top-selling drugs with sales of $2.3 billion last year, but will start losing patent protection in Europe in 2022 and in the US in 2025.

In April, the duo made a big breakthrough for Evenity when the US FDA approved the drug, having previously rejected it in 2017 on safety grounds. They also picked up a Japanese approval around the same time, but hopes that they could quickly add an EU license to Evenity’s tally have now been dashed.

Evenity was approved by the US regulator with a boxed warning that it may increase the risk of heart attack, stroke and cardiovascular death – a safety issue that was responsible for the FDA’s earlier rejection of the drug and also led to the CHMP’s negative opinion.

In its assessment, the advisory committee said “as it was unclear why the medicine appeared to increase the risk of heart and circulatory problems, and there was no obvious group of patients in whom the risk of these was lower, measures to reduce the risk could not readily be put in place.”

The CHMP was also not convinced about the efficacy of the drug in patients with less severe osteoporosis, saying the “benefit was not so convincing” in this group.

The companies say they are planning to submit a written notice to the regulatory agency, requesting a re-evaluation of Evenity for the given indication.

The new drug works in a different way to Prolia, blocking the effects of the protein sclerostin and as a consequence building new bone formation. However it has been approved with a narrower indication than Amgen’s older drug, aimed at high-risk rather than all postmenopausal osteoporosis patients.

It also works differently to other widely-prescribed drugs, including bisphosphonates like Merck & Co/MSD’s Fosamax (alendronate) which work only by blocking bone destruction. Evenity topped alendronate in a head-to-head trial when it came to cutting fracture rates.

CHMP says no to Amgen’s osteoporosis drug Evenity

AstraZeneca’s asthma, diabetes drugs get label boosts in EU

Two of AstraZeneca’s most important drugs have received a boost, after European regulators backed a pen allowing people to inject themselves with respiratory drug Fasenra, and agreed that cardiovascular benefits data could be added to the label of diabetes drug Forxiga.

The European Medicines Agency’s CHMP regulatory committee gave a positive opinion to add a self-administration option for asthma injection Fasenra (benralizumab), based on a new delivery method as a pre-filled, single-use auto-injector.

Fasenra already has a convenience advantage over two rivals from the same class, GlaxoSmithKline’s Nucala (mepolizumab) and Teva’s rival Cinqaero (reslizumab) as it is given every eight weeks, after the first three doses administered that are four weeks apart.

Both Nucala and Cinqaero, which are also interleukin-5 inhibitors, are injected every four weeks.

Following the CHMP’s nod, the drug’s label can be changed without need for confirmation from the European Commission.

AstraZeneca expects a regulatory decision by the FDA on self-administration and the new pre-filled, single-use auto-injector device in the second half of 2019.

Fasenra is currently approved as an add-on maintenance treatment for severe eosinophilic asthma in the US, EU, Japan and other countries.

However, in May last year AZ said Fasenra had failed in a phase 3 trial in chronic obstructive pulmonary disease (COPD), saying it did not provide a statistically significant reduction in exacerbations, a blow to its development in this other indication. NICE has also restricted its funding for NHS patients.

The positive opinion for self-administration and the Fasenra pen is supported by the phase 3 GREGALE and GRECO trials, and the phase 1 AMES trial, respectively. The safety and tolerability of Fasenra in these trials were consistent with the known profile of the medicine.

At the same meeting late last week the CHMP said that diabetes drug Forxiga (dapagliflozin) could have its label changed to include new cardiovascular outcomes data.

The data from the DECLARE-TIMI 58 trial showed the SGLT2 class drug achieved a statistically significant reduction in the composite endpoint for hospitalisation for heart failure or CV death versus placebo, one of the two primary efficacy endpoints.

There were fewer major adverse CV events observed with Forxiga for the other primary efficacy endpoint, but this did not reach statistical significance.

It’s becoming increasingly common for pharma companies to try and show that drugs used to control blood sugar levels in diabetes patients bring cardiovascular benefits.

Eli Lilly and Boehringer Ingelheim were the first to do this with Jardiance, and other companies with drugs from SGLT2 and GLP-1 classes such as Johnson & Johnson and Novo Nordisk have followed the trend.

AZ’s asthma and diabetes drugs get label boosts in EU

NYC Health + Hospitals Rejects Federal Funding Tied to New ‘Gag Rule’

Mayor Bill de Blasio, Deputy Mayor for Health and Human Services Dr. Herminia Palacio, and President and CEO of NYC Health + Hospitals Dr. Mitchell Katz today announced the City’s public health system will cease participating in the federal Title X program for as long as the “gag rule” is in effect. The rule prevents medical providers from sharing information and counseling about abortion to their patients. In a directive to all NYC Health + Hospitals personnel, Dr. Katz today instructed doctors and nurses to support patients on whatever pathway they choose, including providing referrals to abortion and reproductive health services.

In February 2019, the Trump Administration issued the federal “gag rule,” which bars health care providers who receive federal Title X funding from referring or counseling patients about their abortion options. A federal lawsuit – for which New York City filed an amicus brief – had been blocking the gag rule from taking effect. Last week, the Federal Court of Appeals in the Ninth Circuit vacated this injunction, allowing the gag rule to take effect immediately across the country. This would force doctors and medical providers who receive these federal funds for sexual and reproductive health to withhold information about legal abortions from their patients.

Rather than censor providers, NYC Health + Hospitals will reject $1.3 million in federal funding from the Title X program — which funds a range of family planning and related preventive health services — until the gag rule is lifted. The City will cover the lost Title X funds, and NYC Health + Hospitals will direct doctors and nurses to continue providing referrals to abortion and reproductive health services.

Mayor, NYC Health + Hospitals Reject Federal Funding Tied to New “Gag Rule”

Retrophin (RTRX) PT Raised to $41 at Canaccord

Canaccord Genuity analyst Michelle Gilson raised the price target on Retrophin (NASDAQ: RTRX) to $41.00 (from $37.00) while maintaining a Buy rating after the company announced the FDA has approved THIOLA EC 100mg and 300mg tablets for the treatment of cystinuria.

“While we are encouraged by the step forward in the regulatory process, an update on IP around the new formulation is anticipated 2H19 or later,” Gilson commented. “A positive update on IP would help to lift the overhang on generics development further, though would not prevent competition around the original formulation. We expect comparable pricing to original formulation, which should enable access to switching.”

https://www.streetinsider.com/Analyst+Comments/Retrophin+%28RTRX%29+PT+Raised+to+%2441+at+Canaccord+Genuity/15663433.html

Regeneron Libtayo OKed for Cutaneous Squamous Cell Carcinoma in EU

Libtayo is the only treatment approved in the EU for adult patients with metastatic or locally advanced cutaneous squamous cell carcinoma who are not candidates for curative surgery or curative radiation

CSCC is one of the most common skin cancers worldwide and is especially difficult to treat in advanced stages

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Commission (EC) has granted conditional marketing authorization for Libtayo^® (cemiplimab) for the treatment of adults with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or curative radiation. Libtayo is a fully-human monoclonal antibody targeting the immune checkpoint receptor PD-1 (programmed cell death protein-1) and is the only treatment approved in advanced CSCC in the European Union (EU).

“With no other medical treatments approved for advanced CSCC in the EU, Libtayo represents an important new option for patients affected with this advanced skin cancer who cannot be cured by surgery or radiation,” said Axel Hauschild, M.D., Ph.D., an investigator in the pivotal CSCC clinical program and Professor and Head of the Interdisciplinary Skin Cancer Center at the University Hospital Schleswig-Holstein in Kiel, Germany. “Results from the Libtayo pivotal trial are very encouraging and demonstrated substantial and durable responses following Libtayo treatment, including in the elderly and regardless of PD-L1 expression levels.”

Updated data from the registrational EMPOWER-CSCC-1 trial were recently shared at the 2019 American Society of Clinical Oncology Annual Meeting.

https://www.marketscreener.com/REGENERON-PHARMACEUTICALS-10649/news/Regeneron-Pharmaceuticals-Libtayo-cemiplimab-Approved-for-Advanced-Cutaneous-Squamous-Cell-Carc-28837939/

Lonza fills portfolio hole by buying Novartis drug bottling plant

Lonza Group is buying a drug bottling plant from Novartis in northern Switzerland as the Swiss drug ingredients maker fills a gap in its offering for drugmakers seeking to outsource production.

Novartis is selling the so-called “fill and finish” facility because its production lines had been underutilised.

Lonza has been building up its drug products services business for three years and has been weighing whether to buy a factory from a rival where it can put the finishing touches on injectible medicines, o
r build such a facility itself like it is doing now in Visp, Switzerland. [https://reut.rs/32272tu]

In buying Novartis’s 10-year-old plant in Stein, Lonza will be able to speed up work for customers seeking to take their injectible medicines quickly into the clinic and onto the market, in particular for smaller lots of medicines aimed at niche populations, a Lonza spokeswoman said.

“Buying rather than building also means we will be operational immediately with an experienced team,” said Hanns-Christian Mahler, Lonza’s head of drug product services who the company poached in 2016 from Roche to build up the business.

Lonza shares were up 0.7 percent at 0830 GMT, bringing their rise this year to 29 percent.

Lonza plans to keep the facility’s employees and will continue to produce for Novartis.

SEAMLESS FIT

“The acquisition of the sterile bottling plant in Stein fits seamlessly into Lonza’s strategy to expand its drug development, production and formulation business,” Zuercher Kantonalbank analyst Philipp Gamper wrote in a note to investors. Gamper has a “market weight” rating on Lonza shares.

Lonza previously expanded into packaging operations for drugs when it bought Capsugel in 2016 for $5.5 billion.

Novartis is selling the drug bottling plant that it built in 2009 after reviewing alternatives for its under-used production lines.

“The planned sale of the two buildings is the best option to ensure the further employment and development of our employees and the continuation” of the site, a Novartis spokesman said. “Lonza will produce drug product at the facility for Novartis as well as providing capacity for additional customers.”

This month, Lonza carved out its specialty ingredients operation which makes products such as anti-microbials for paint into a standalone business aiming to remedy problems there that have dragged on earnings.

Lonza has also sold a water care business as it seeks to expand its faster-growing biopharmaceuticals business.

https://www.marketscreener.com/LONZA-GROUP-2956013/news/Lonza-fills-portfolio-hole-by-buying-Novartis-drug-bottling-plant-28834841/

Principia Biopharma started at Buy by Wainwright

Target $55

https://www.benzinga.com/stock/PRNB/ratings