Regeneron, Sanofi Eye October for FDA Review of Skin Cancer Drug Cemiplimab

April 30, 2018

Regeneron and its longtime development partner Sanofi are keeping their proverbial fingers crossed that their profitable collaboration will yield yet another approved treatment this year.

This morning the companies announced the U.S. Food and Drug Administration (FDA) has set a PDUFA date of Oct. 28 to determine the fate of its anti-PD-1 skin cancer treatment, cemiplimab. The two companies firmly believe there is still room in the crowded checkpoint inhibitor field dominated by Merck, AstraZeneca and Bristol-Myers Squibb. Merck’s Keytruda or Bristol-Myers Opdivo are responsible for generating billions of dollars in revenue for both companies. And that is something that Regeneron and Sanofi are hoping will occur with cemiplimab as well. In January Regeneron and Sanofi demonstrated their commitment to the development of the drug with an additional $1 billion for research and development. That additional billion dollars means the investment in cemiplimab will be increased to a minimum of $1.64 billion. If the FDA approved cemiplimab in October, it’s quite likely that the two companies will immediately begin planning for approval of the checkpoint inhibitor in other indications.

In the announcement today Regeneron and Sanofi said the FDA granted a priority review for the Biologics License Application for cemiplimab based on impressive topline results from its pivotal Phase II study that continued to show a high response rate in patients. Those results, which were released in December, spurred the companies to begin seeking approval for the checkpoint inhibitor. Additionally, the FDA will consider Phase I data from two advanced CSCC expansion cohorts. Updated results from both clinical trials will be presented at the 2018 ASCO Annual Meeting, the two companies said this morning.

Sanofi and Regeneron are developing the PD-1 inhibitor cemiplimab to treat adult patients with metastatic cutaneous squamous cell carcinoma (CSCC) and adults with locally advanced and unresectable CSCC, the second deadliest skin cancer after melanoma. CSCC is the second most common type of skin cancer in the United States. It can be fatal to non-melanoma skin cancer patients, Although CSCC has a good prognosis when caught early, and it can prove especially difficult to treat when it progresses to advanced stages. Patients at this stage can be disfigured due to multiple surgeries to remove CSCC tumors on the head, neck and other parts of the body.

The Phase II topline results showed that of the 82 patients treated with cemiplimab 46.3 percent demonstrated an overall response rate. Last year the FDA granted the drug Breakthrough Therapy status following the Phase I results.

In addition to submitting cemiplimab for review in the United States, the two companies are also seeking approval in Europe. The European Medicines Agency accepted the Marketing Authorization Application earlier this month.

The Sanofi and Regeneron developmental partnership is proving to be a valuable one. In 2017 the two companies snagged FDA approval for two jointly-developed drugs. In May the companies gained approval for rheumatoid arthritis drug Kevzara (sarilumab). Data from a Phase III trial released in March showed that sarilumab outperformed AbbVie’s Humira. In March 2017 the FDA approved Dupixent (dupilumab) for the treatment of adults with moderate-to-severe atopic dermatitis.

https://bit.ly/2I1YdbW

FDA Clears Pluristem Application for Emergency Radiation Treatment

The U.S. Food and Drug Administration (FDA) cleared Haifa, Israel-based Pluristem Therapeutics’ Investigational New Drug (IND) application for PLX-R18 to treat acute radiation syndrome (ARS).

The U.S. National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID) sponsored and wrapped a successful Phase II-equivalent trial of PLX-R18 to treat ARS using the FDA’s animal rule pathway. The FDA Animal Efficacy Rule applies to developing and testing of drugs to reduce or prevent serious and life-threatening conditions caused by exposure to lethal or permanently disabling toxic agents where human testing it’s feasible or ethical.

It is also being evaluated by the U.S. Department of Defense (DOD) in supported of the military for treatment before or within the first 24 hours of radiation exposure. The company is currently in talks with the FDA and several U.S. governmental agencies for proposed pivotal study of PLX-R18 in ARS.

The traditional emergency treatment for radiation poisoning is potassium iodide (KI). This compound helps block radioactive iodine from being absorbed by the thyroid gland, which is the part of the body most sensitive to radioactive iodine.

Just last week, Canadian residents in Amherstburg, Ontario, directly across Lake Erie from the Fermi nuclear power plant in Monroe, Michigan, were offered potassium iodide pills as part of a plan to educate people in the “primary zone” of the Enrico Fermi nuclear power plant.

PLX-R18 is a cell therapy designed to treat bone marrow that can’t produce enough blood cells because of ARS, as well as certain cancers, cancer treatments, or immune-mediated bone marrow failure. Early animal studies were conducted in collaboration with the Hadassah Medical Center, and additional preclinical trials were performed with the NIH, Hadassah, the Charite in Berlin, and other research institutions.

“We are proud to have PLX-R18 join the exclusive club of IND approved medical countermeasures for the treatment of ARS,” said Zami Aberman, chairman and co-chief executive officer of Pluristem, in a statement. “This FDA clearance is one of the most significant milestones in the development of PLX-R18 to date and should provide Pluristem with significant support in advancing its off-the-shelf cell therapy into a pivotal trial. The fact that we are now able to treat human casualties in the case of a nuclear event provides us with the ability to protect from severe health consequences, savings lives of population in need.”

The drug is injected into muscle two times, a week apart. It can be used up to 96 hours after exposure. Arik Eisenkraft, the company’s director of homeland defense projects, told Reuters, “We are increasing the survival rate following high-level radiation exposure. We see improvement in all three blood lineages—red cells, white cells and platelets. We think that the everyday use of the compound will be for bone marrow failure of any cause.”

It also has an advantage in that it doesn’t require tissue matching prior to administration. The company is also conducting Phase III trials in the U.S. and Europe of PLX-PAD, to treat patients with the end-stage of peripheral artery disease, to prevent amputation.

The company indicates it will begin to prepare an emergency stock of PLX-R18 for emergency use. Full approval will depend on the Phase III clinical trial results.

Pluristem’s technology uses placental cells and a proprietary 3D technology platform to develop cell therapies for a variety of conditions, including inflammation, ischemia, muscle injuries, hematological disorders and radiation exposure.

https://bit.ly/2HHCHps

iRadimed Q1 revenues up 37%; raises fiscal guidance

• iRadimed (IRMD) Q1 results: Revenues: $7.1M (+36.5%); Operating Income: $1.1M; Net Income: $0.8M; Non-GAAP Net Income: $1.2M; EPS: $0.07; Non-GAAP EPS: $0.10; CF Ops: $1.6M.
• 2018 Guidance: Revenues: $29.3M – 30.0M; EPS: $0.30 – 0.33 from $0.22 – 0.27; Non-GAAP EPS: $0.40 – 0.43 from $0.33 – 0.38.
• Q2 Guidance: Revenues: $7.2M- 7.3M; EPS: $0.07 – 0.08; Non-GAAP EPS: $0.10 – 0.11.

https://bit.ly/2rc8TKj

Spark Therapeutics sells priority review voucher for $110M

Spark Therapeutics (ONCE) announced it has entered into an agreement to sell its rare pediatric disease priority review voucher, or PRV. The PRV was received when LUXTURNA was approved by the FDA. Under the PRV program, a sponsor who receives FDA approval for a rare pediatric disease drug or biologic may qualify for a voucher to be redeemed at a future time for priority review of a subsequent marketing application for a different product. Per the terms of the agreement with Jazz Pharmaceuticals (JAZZ), Spark Therapeutics will receive $110M upon closing of the transaction, which is subject to customary closing conditions

https://bit.ly/2jg3EG5

United Therapeutics To Acquire SteadyMed

 United Therapeutics Corporation (UTHR) and SteadyMed Ltd. (STDY) announced today the signing of a definitive merger agreement under which United Therapeutics will acquire SteadyMed for $4.46 per share in cash at closing and an additional $2.63 per share in cash upon the achievement of a milestone related to the commercialization of Trevyent®. The transaction, including the $75 million in contingent consideration, is valued at $216 million.

SteadyMed is a specialty pharmaceutical company focused on the development and commercialization of drug product candidates to treat orphan and high-value diseases with unmet parenteral delivery needs. SteadyMed’s product portfolio includes Trevyent, a development-stage drug-device combination product that combines SteadyMed’s two day, single use, disposable PatchPump® technology with treprostinil, a vasodilatory prostacyclin analogue, for the subcutaneous treatment of pulmonary arterial hypertension (PAH). United Therapeutics is a leading biotechnology company focused on the development and commercialization of therapies for the treatment of PAH and other orphan diseases.

“We are optimistic about acquiring SteadyMed and adding Trevyent to our pipeline of products to treat PAH,” said Martine Rothblatt, Ph.D., Chairman and Chief Executive Officer of United Therapeutics. “We are especially impressed with SteadyMed’s management team and global supply chain. Trevyent fits in well with our mission, and we look forward to bringing the product to the maximum number of patients as soon as possible.”

“United Therapeutics has always been at the forefront of developing therapies to treat PAH, and we are delighted at the prospect of our companies coming together, as one, to continue that mission,” said Jonathan M.N. Rigby, President and Chief Executive Officer of SteadyMed. “We believe that this proposed acquisition will help us realize our commitment to bring Trevyent to market to improve the lives of patients with PAH.”

The Board of Directors of SteadyMed has unanimously approved the merger agreement and unanimously recommends that SteadyMed shareholders adopt the merger agreement. SteadyMed shareholders owning approximately 43.3 percent of the ordinary shares of SteadyMed have entered into an agreement to vote their shares in favor of the transaction.

The transaction is subject to customary closing conditions, including approval by SteadyMed’s shareholders and the expiration or termination of the required waiting period under the Hart-Scott-Rodino Antitrust Improvements Act, and is expected to be completed in the third quarter of this year.

https://yhoo.it/2jj4zFS

Allergan CEO: Big deals ‘unlikely’ from review

Says smaller deals more likely on the table. Says a fundamental shift in business strategy “unlikely.”

https://bit.ly/2Kqlwur

STAAR upped to buy by Blair

STAAR Surgical upgraded to Outperform from Market Perform at William Blair. William Blair analyst Brian Weinstein upgraded STAAR Surgical to Outperform saying he’s grown more comfortable with the company’s ability to forecast demand trends and drive a baseline case for mid- to high-teens revenue growth through 2020. STAAR has a number of potential positive catalysts over the next 12-18 months, including possible opening of the U.S. market again, which could boost growth above these levels and provide upside from current stock levels, Weinstein tells investors in a research note

https://bit.ly/2JFjs0e