Exelixis and Aerie Have Good News

Exelixis & Aerie Have Good News

Exelixis’ Good News about Cabometyx

Takeda Pharmaceuticals applied to the Japanese Ministry of Health, Labor and Welfare (MHLW) for approval to manufacture and to sell Exelixis’ (EXEL) product Cabometyx® (cabozantinib) for unresectable and metastatic renal cell carcinoma (RCC) in Japan.

The submission brings $10 million in milestone payments to Exelixis from Takeda in the second quarter of 2019.

Takeda’s application in Japan is based on the results of three clinical trials:

The METEOR Trial – 

Exelixis-sponsored phase 3 pivotal trial of cabozantinib versus everolimus in patients with advanced RCC who experienced disease progression following treatment with at least one prior VEGF receptor tyrosine kinase inhibitor (VEGFR-TKI).

The CABOSUN Trial –

The Alliance for Clinical Trials in Oncology-sponsored phase 2 trial comparing cabozantinib with sunitinib in patients with previously untreated advanced RCC with intermediate- or poor-risk disease.

The Cabozantinib-2001 – 

A Takeda-sponsored phase 2 trial in 35 Japanese patients with advanced RCC that progressed after prior VEGFR-TKI therapy.

Takeda’s phase 2 trial was the subject of a late-breaking abstract at the 107th Annual Meeting of the Japanese Urological Society on April 18, 2019.

A Quote from the President & CEO of Exelixis

Michael M. Morrissey, Ph.D., President and Chief Executive Officer of Exelixis said, “Takeda has proven to be a very effective partner in cabozantinib’s development program in Japan since the signing of our collaboration and licensing agreement in early 2017. This Japanese regulatory filing is an important milestone on the path toward offering Cabometyx as a new therapeutic option for patients with unresectable, metastatic renal cell carcinoma in Japan. We congratulate our Takeda colleagues on the filing and look forward to further progress.”

The Terms of Exelixis/Takeda’s Collaboration

Exelixis received a $50 million upfront payment at the time of signing.

Following the milestone associated with this regulatory filing, Exelixis will be eligible to receive, from Takeda, further development, regulatory and first-sale milestone payments of up to $80 million; related both to previously treated and previously untreated RCC and previously treated hepatocellular carcinoma (HCC). As well as additional development, regulatory and first-sale milestones for potential future cabozantinib indications.  

Exelixis is also eligible for sales revenue milestones and royalties on net sales of cabozantinib in Japan.

Takeda is to fully fund cabozantinib development activities. Which are exclusive for the benefit of Japan.

Takeda is also responsible for 20% of the costs associated with global cabozantinib clinical trials, providing the company opts into those trials.

As of today, Takeda has opted into, and is co-funding, CheckMate 9ER, the ongoing phase 3 pivotal trial of cabozantinib plus nivolumab versus sunitinib, in previously untreated advanced RCC. 

About Renal Cell Carcinoma (RCC)

Kidney cancer is among the top ten most commonly diagnosed forms of cancer. Clear cell RCC is the most common type of kidney cancer in adults. If detected in its early stages the five-year survival rate is high. For patients with advanced or late-stage metastatic RCC; however, the five-year survival rate is only 12%, with no identified cure for the disease. 

Approximately 32,000 patients in the U.S. and 70,000 globally require treatment. An estimated 15,000 patients in the U.S. each year are in need of a first-line treatment for advanced kidney cancer.

In the U.S. Cabometyx tablets are approved for patients with advanced RCC and for the treatment of patients with HCC, who have been previously treated with sorafenib. Cabometyx tablets have also received regulatory approvals in the European Union and in various countries worldwide.

In 2016 Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. In 2017 Exelixis granted exclusive rights to Takeda for the commercialization and further clinical development of cabozantinib for all future indications in Japan.

For U.S. Important Safety Information please read the firm’s press release.  

Prohost Observations

Each and every move forward toward gathering additional approvals for cabozantinib adds money to Exelixis’ coffers. The increase in revenues helps the firm design and conduct clinical trials for the drug to be used alone or in combination treatments. Exelixis and Bristol-Myers (BMY) are conducting trials with a combination of Exelixis’ product cabozantinib and Bristol-Myers’ immunotherapy checkpoint protein inhibitor drug, Opdivo (nivolumab) as well as other products for first line treatment of RCC. Soon we will know the results,which we believe will confirm cabozantinib as the most effective treatment for kidney cancer.

Do not forget to listen to Exelixis’ first quarter 2019 financial results on May 1, 2019.

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Aerie Pharmaceuticals’ Good News

Aerie Pharmaceuticals (AERI) announced that the FDA has reviewed the Investigational New Drug Application (IND) for its novel multi-kinase (Rho kinase/Protein kinase C inhibitor sustained-release) implant, the AR-13503 implant, and allowed  Aerie to initiate human studies. The studies are neovascular age-related macular degeneration (nAMD) and diabetic macular edema (DME).

Aerie expects to initiate a first-in-human clinical study in the second quarter of 2019.

The AR-13503 implant

The bio-erodible polyesteramide polymer implant provides a controlled release of AR-13503 over a sustained period. It is designed to be administered approximately once every six months by intravitreal injection.

Preclinical Studies’ Insinuations for AR-13503

The studies suggest that AR-13503 has the potential to inhibit angiogenesis, preserve the blood-retinal barrier and reduce retinal fibrosis in retinal diseases such as: nAMD and DME, while potentially reducing the treatment burden associated with more frequent intravitreal injections.

A Quote from the Chairman & CEO of Aerie

Vicente Anido, Jr., Ph.D., Chairman and Chief Executive Officer of Aerie, said,  “While the standard-of-care anti-VEGF therapies are effective for many patients with nAMD and DME, there are many patients who do not respond adequately to these therapies or who lose their initial gains in vision over time. By targeting multiple disease processes that underlie progression in these common, sight-threatening retinal diseases, our research suggests that ROCK/PKC inhibitors such as AR-13503 may prove useful as alternative or additive therapies to the currently available treatments. The AR-13503 implant marks our second foray into the retina space, with this product candidate leveraging both our scientific leadership in the creation of small-molecule kinase inhibitors for disease intervention along with our proprietary ophthalmic drug delivery platform.” 

About Retinal Disease (RCC)

The retina, in the back of the eye, is composed of highly specialized cells that convert light into electrical impulses that reach the brain through the optic nerve. Various diseases, including age-related macular degeneration (AMD) and diabetic macular edema, can damage the retina and cause visual impairment or permanent blindness. In the United States AMD is the leading cause of significant visual acuity loss in people over the age of 50. Diabetic retinopathy is the most common cause of irreversible blindness among working-age people. Bottom line, the retinal disease market is the largest therapeutic category in ophthalmology with revenues in the United States reaching approximately $6.0 billion, in 2018. Because current medical therapies are not adequately effective for many patients, and do not stop disease progression, new treatment options are needed.

Prohost Observations

Aerie is bringing new effective treatments for eye diseases, with unmet needs or with imperfect treatments, into the clinic. The firm has already put a novel treatment for glaucoma on the market which ophthalmologists are prescribing for specific cases. The road is long for the novel multi-kinase sustained release implant, the AR-13503 implant, to be completely validated in all trials. Today’s news; however, is good enough for Aerie and its stockholders to enjoy. 

Do not miss the upcoming Prohost Letter about CRISPR’s gene editing technology and CAR T improvements.

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