23 April 2019

Ipsen closes $1bn Clementia acquisition

Ipsen has completed an acquisition of Canada-based Clementia Pharmaceuticals to expand its rare diseases portfolio.

The deal involves an initial aggregate consideration of $1.04bn as an upfront cash payment, which equals $25 per share.

Ipsen will pay an additional $263m ($6 per share) if Clementia’s investigational retinoic acid receptor gamma (RARγ) Palovarotene is approved in the US as a treatment for multiple osteochondromas (MO).

An application seeking US Food and Drug Administration (FDA) approval for Palovarotene to prevent heterotopic ossification (HO) in fibrodysplasia ossificans progressiva (FOP) patients is set to be filed in the second half of this year.

The submission will include data from a Phase II clinical trial demonstrating that Palovarotene led to a more than 70% decrease in new HO across all dosing levels.

Ipsen chief scientific officer Alexandre Lebeaut said: “We are encouraged by compelling and consistent clinical data from the extensive Phase II programme, as well as fast-track, breakthrough therapy, orphan drug and rare paediatric diseases designations from the FDA.

“We are focused on the successful regulatory submission of Palovarotene as a first-in-class therapeutic solution for patients with episodic flare-up treatment of fibrodysplasia ossificans progressiva in the second half of 2019.”

Clementia CEO Clarissa Desjardins noted that Ipsen’s development capabilities and commercial footprint will help provide patients worldwide with access to Palovarotene.

The deal was first announced in February this year and has been approved by the board of directors of both parties, Clementia’s shareholders, and the Quebec Superior Court.

18 April 2019

FDA grants breakthrough status to Genfit’s Elafibranor

The FDA has granted breakthrough therapy designation to Genfit’s Elafibranor to treat adults with primary biliary cholangitis (PBC) that experience an inadequate response to ursodeoxycholic acid (UDCA).

Elafibranor is a double peroxisome proliferator-activated receptor alpha and delta agonist that was originally developed to treat non-alcoholic steatohepatitis (NASH).

Results from a Phase II clinical trial showed that the therapeutic could also treat PBC, a rare, chronic live disease characterised by gradual damage to bile ducts. The condition hinders the liver’s ability to remove toxins from the body, which leads to cirrhosis.

Genfit deputy chief medical officer Pascal Birman said: “PBC is a severe liver disease that can lead to cirrhosis and liver failure, and is commonly associated with debilitating symptoms such as pruritus, which affect patients’ quality of life.

“Approximately 50% of patients have an inadequate response to existing therapies, either because they do not respond to treatment or because they experience intolerable side effects like aggravated pruritus (itching) or hepatic toxicity. Elafibranor has shown promising anticholestatic effects in a Phase II clinical trial, while showing a trend in reducing pruritus.”

The FDA’s designation is based on results from a 12-week, double-blind, randomised placebo-controlled Phase II trial that treated non-cirrhotic subjects with PBC that showed an inadequate response to UDCA.

According to the results, Genfit’s drug candidate demonstrated a significant reduction of alkaline phosphatase (ALP) levels, compared to placebo. This was the primary endpoint of the study.

The trial also found improvements in other PBC markers such as gamma-glutamyl transferase (GGT), lipid markers, low-density lipoprotein (LDL) and anti-inflammatory markers in patients treated with Elafibranor.

Genfit noted that the GGT, lipid and anti-inflammatory marker improvements are consistent with those observed in the Phase II trial for NASH.

Elafibranor was generally well-tolerated in the trial, and is set to be studied in another Phase III trial.

17 April 2019

Pulmatrix and Cipla collaborate to develop asthma drug

Pulmatrix and Cipla Technologies have signed a definitive agreement to jointly develop and commercialise Pulmazole to treat allergic bronchopulmonary aspergillosis (ABPA) in asthma patients.

Pulmazole is an inhaled iSPERSE (inhaled small particles easily respirable and emitted) formulation of itraconazole, an anti-fungal drug.

Patients with asthma or cystic fibrosis often suffer from ABPA, an infection which causes an exaggerated allergic hypersensitivity response from the immune system.

Currently, ABPA is treated with oral formulation of itraconazole in adjunction with corticosteroids. However, the drug is linked with poor bioavailability, variable pharmacokinetics and toxicity concerns.

The inhaled Pulmazole medication is intended to improve upon the established safety and efficacy profile of the oral formulation.

Cipla Technologies will pay $22m in upfront payment to Pulmatrix. In return, Cipla will get all rights to Pulmazole in pulmonary indications.

The partners intend to equally share costs for the future development and commercialisation of Pulmazole, as well as worldwide free cash flow from future sales of the medication.

Pulmatrix will carry out the clinical development of the therapeutic, while Cipla Technologies will be responsible for its commercialisation.

Cipla managing director and global CEO Umang Vohra said: “Pulmazole will be Cipla’s entry into the branded respiratory space and will serve a vital unmet medical need for the treatment of ABPA, a condition that possibly impacts over two million patients worldwide but has no labelled drug.”

The deal also provides Pulmatrix with the funds needed to conduct a Phase II clinical trial of the product.

During the randomised multi-centre trial, Pulmazole’s safety, tolerability and pharmacokinetics will be assessed in adult patients with asthma and allergic bronchopulmonary aspergillosis.

Pulmatrix CEO Robert Clarke said: “This is also an important financial milestone for the company, securing adequate funds to complete the Pulmazole Phase II study, along with 50% commitment from Cip Tec for future Pulmazole development and commercialisation costs while retaining worldwide rights to 50% of the free-cash-flow from future revenues.”

A joint steering committee will be established to supervise the collaboration.

16 April 2019

Catalent to buy gene therapy manufacturer Paragon for $1.2bn

Catalent, a provider of biologic drug development and manufacturing services, has signed a definitive agreement to acquire gene therapy manufacturing company Paragon Bioservices for $1.2bn.

Paragon offers development and manufacturing services for a variety of biopharmaceuticals, including recombinant viral vectors, vaccines, certain recombinant proteins and oncolytic viruses.

The company has partnered with multiple biotech and pharmaceutical businesses to develop and manufacture drug products.

Catalent expects the gene therapy manufacturing expertise of Paragon to bolster its business.

Catalent chair and CEO John Chiminski said: “Paragon’s unparalleled expertise in the rapidly growing market of gene therapy manufacturing will be a transformative addition to our business that we believe will accelerate our long-term growth.

“Paragon brings to Catalent a complementary capability that will fundamentally enhance our biologics business and our end-to-end integrated biopharmaceutical solutions for customers.”

Paragon president and CEO Pete Buzy added that the deal would help the company in expanding its capabilities and platform.

Catalent estimates that Paragon would generate more than $200m in revenue this year. The company added that approximately 90% of the expected revenue is already reflected in signed contracts.

In addition, Catalent believes that Paragon is positioned to cater to the emerging gene therapy market, which is anticipated to register growth of 25% in the medium term.

Apart from the purchase price and transaction costs, Catalent also plans to finance a part of the ongoing capital expansion projects at Paragon’s facilities located in Maryland, US.

Subject to customary closing conditions, the acquisition is set to be completed in the second quarter of this year.

After the closing of the deal, Pete Buzy will continue to lead Paragon. The company’s management team and around 380 employees will be transitioned to Catalent.

Catalent expects that the acquisition will be accretive to its adjusted net income per share in the second full fiscal year after completion and significantly accretive thereafter.

15 April 2019

Bristol-Myers $74bn Celgene takeover gets shareholders approvals

Bristol-Myers Squibb has secured shareholders’ approval for its $74bn acquisition of biotechnology company Celgene. More than 75% of the shareholders voted in favour of the merger agreement.

Celgene shareholders also voted to approve, with nearly 98% of the votes supporting the deal.

As per the terms of the agreement, Celgene stockholders will get one Bristol-Myers share and $50 in cash per share of the biotech’s common stock.

The merger is expected to create a speciality biopharma company. Subject to customary closing conditions and regulatory approvals, the acquisition is expected to close in the third quarter of this year.

The combined entity will have a portfolio of nine products with more than $1bn in annual sales and six launch prospects with more than $15bn revenue potential. It will also feature 50 assets in the early stage pipeline.

Celgene chairman and CEO Mark Alles said: “The combined company will create a leading focused specialty biopharma company well positioned to address the needs of patients with cancer, inflammatory and immunologic disease and cardiovascular disease through high-value innovative medicines and leading scientific capabilities.”

The deal, which is considered to be one of the largest in the industry, had faced opposition from investor groups Wellington Management and Starboard Value.

Wellington argued that the transaction is too risky for BMS shareholders and also raised concerns over the execution success, while Starboard Value called it ‘poorly conceived and ill-advised.’

Last month, Starboard Value withdrew its campaign against the merger after proxy advisory firms Institutional Shareholder Services and Glass Lewis offered their support in favour of the deal.

Announcing the advisory groups’ recommendation, Bristol-Myers said: “We are confident that combining Bristol-Myers Squibb and Celgene will create a leading focused specialty biopharma company that is well positioned to address the needs of patients across disease areas and generate meaningful financial benefits for all shareholders through 2025 and beyond.”

12 April 2019

Sanofi to slash costs of certain insulin products in US

Sanofi has said that it will reduce the cost of some of its insulin products to $99 per month in a bid to mitigate burden on diabetes patients in the US.

Patients who are not covered under the federal or state insurance programmes such as Medicare and Medicaid will have access to up to ten boxes of insulin pens and/or 10ml vials per month at the reduced price.

Set to begin from June this year, the initiative is part of the French drugmaker’s Insulins Valyou Savings Program that previously offered one 10ml vial for $99 and a box of pens for $149.

Launched in April last year, the Insulins Valyou Savings Program is said to have led to around $10m in savings for patients.

Sanofi North America Primary Care head Michelle Carnahan said: “By giving those who require both long-acting and/or mealtime insulins or use more than one box of pens or one vial per month access to their insulins for one flat price, we aim to help limit the burden on the individuals who have high out-of-pocket costs at the pharmacy counter.”

The announcement was made hours before Sanofi was to testify before the US Energy and Commerce Committee on the increasing insulin prices in the country.

In January this year, the congressional committee asked Sanofi, Eli Lilly and Novo Nordisk to provide information on the increased insulin costs in recent years and the companies’ profit from these products.

The drugmakers were requested to disclose the average price of their insulin products for the last ten years and also the reasons behind any price hikes.

Sanofi is not the first company to cut insulin charges amid rising pressure to reduce prescription medicine prices.

Last month, Lilly said it would launch a lower-priced, generic version of its insulin injection Humalog. A single vial of the generic is priced at $137.35, 50% less than the current list price of Humalog.

10 April 2019

FDA approves Amgen’s postmenopausal osteoporosis drug Evenity

Amgen has received FDA approval for Evenity (romosozumab-aqqg) to treat osteoporosis in postmenopausal women at high risk for fracture.

Evenity is a humanised monoclonal antibody designed to inhibit sclerostin in order to improve bone formation and to some extent decrease bone loss.

One dose of the drug comprises two injections and a full course consists of 12 monthly doses.

Following these 12 doses, the bone forming effect of the drug diminishes and further therapy with an anti-resorptive agent is recommended to address the chronic condition.

Osteoporosis, which lacks cure, affects nearly ten million people in the US. Bone breaks are a common occurrence in these patients and the condition is known to cause around two million fractures per year.

Postmenopausal women are at a high risk of loss of mobility due to osteoporosis-related fractures.

Amgen Research and Development executive vice-president David Reese said: “One in two women will experience a fracture due to osteoporosis in her lifetime. These fractures can be devastating, with many leading to hospital stays and life-altering consequences.

“The FDA approval of Evenity represents an important therapeutic development for patients who need a medicine that can rapidly increase bone mineral density and help reduce the risk of future fractures within 12 months.”

The FDA approval comes after review of findings from Phase III FRAME and ARCH clinical trials.

In FRAME, Evenity led to a significant decrease in new vertebral fracture at 12 months, compared to placebo. The decrease was retained through the second year in patients who received the drug during the first year and transitioned to denosumab.

Furthermore, patients treated with Amgen’s drug experienced increase in bone mineral density (BMD) at the lumbar spine, total hip and femoral neck at 12 months, which continued through month 24.

The ARCH study involved treatment with Evenity and alendronate for 12 months each. This minimised the incidence of new vertebral fracture at 24 months and the risk of clinical fracture after a median 33-month follow-up.

In addition, 12 months of treatment with Evenity followed by 12 months alendronate therapy significantly improved BMD, compared to alendronate alone.

Evenity comes with a boxed warning of potential increase in the risk of heart attack, stroke and cardiovascular death. The drug will be commercially launched in the US next week.

09 April 2019

Researchers develop new vaccine to teach immune system to attack cancer

Researchers at Mount Sinai in the US have developed a new immunotherapy that can be injected directly into a tumour cell and teach a patient’s own immune system to kill cancer throughout the body.

Called in situ vaccination, the new approach involves administration of two immune stimulants.

One of the stimulants recruits a type of immune cells called dendritic cells, which will then be activated by the second stimulant to direct T cells to attack cancer cells while sparing non-cancer cells.

The researchers noted that the immune cells learn to identify tumour cell characteristics in order to find and destroy the cancer throughout the body. This approach is said to turn the tumours into cancer vaccine factories.

Icahn School of Medicine at Mount Sinai The Tisch Cancer Institute lymphoma immunotherapy program director Joshua Brody said: “The in situ vaccine approach has broad implications for multiple types of cancer. This method could also increase the success of other immunotherapies such as checkpoint blockade.”

When tested in mice, the new vaccine therapy was able to significantly boost the checkpoint blockade immunotherapy.

The researchers then went on to evaluate the treatment in a clinical trial involving 11 patients with advanced-stage lymphoma.

Results, which have been published in Nature Medicine journal, demonstrated full remission ranging from months to years in some patients.

Based on these promising data, the team initiated trials in breast, and head and neck cancer patients to assess the vaccine in combination with checkpoint blockade drugs.

The Damon Runyon Cancer Research Foundation, the Cancer Research Institute and Merck funded this research, while Celldex and Oncovir supplied reagents for the clinical trial and the lab testing.

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