An Approval for Celgene and Two Breakthrough Designations for AstraZeneca

CELGENE

Good news from Celgene (CELG) announced  that the FDA approved the firm’s targeted drug Idhifa (enasidenib) for adults with relapsed or refractory acute myeloid leukemia (AML) associated with isocitrate dehydrogenase-2 (IDH2) mutations.

Good news also announced that the FDA approved the RealTime IDH2 companion assay, which pinpoints the IDH2 mutations.

Richard Pazdur, MD, of the FDA Office of Hematology and Oncology Products, said in a statement. “The use of Idhifa was associated with a complete remission in some patients and a reduction in the need for both red-cell and platelet transfusion.”

As a matter of fact, results from a single arm trial with Celgene’s drug Idhifa comprising 199 patients with IDH2-positive relapsed/refractory AML demonstrated that 19% of patients treated with enasidenib achieved complete response (CR) persisting for a median duration of 8.2 months, and 4% of patients attained partial response with a median duration of 9.6 months. Around a third of 157 patients who required transfusions prior to treatment were no longer in need for blood transfusion following the treatment with enasidenib.

Enasidenib common side effects of included nausea, vomiting, diarrhea, lowered of appetite and increased bilirubin levels. The drug is also contraindicated for women who are pregnant or breastfeeding.

A boxed warning was included about a risk of differentiation syndrome, which could be fatal if not diagnosed and treated on time. The syndrome symptoms include fever; dyspnea; acute respiratory distress; radiographic evidence of pulmonary infiltrates; pleural or pericardial effusion; rapid weight gain; peripheral edema; and liver, kidney, or multi-organ dysfunction.

Prohost Observations

 Indeed, this is good news for Celgene and the people with relapsed or refractory acute myeloid leukemia (AML) associated with (IDH2) mutations. The approval of the companion test also confirms that the drug will be prescribed only for the people who have AML associated with the IDH2 mutation. The approval also confirms that Celgene has solid scientific fundamentals and a team of skilled scientists and researchers governed by a capable management.

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ASTRAZENECA

Two Drugs Designated Breakthroughs in Two Consecutive Days

On Monday, the U.S. Food and Drug Administration (FDA) awarded AstraZeneca cancer drug IMFINZI (duravulumab) a breakthrough therapy designation. Imfinzi is a checkpoint immunotherapy product targeting for inhibition PD-L1 aimed to treat patients with locally-advanced unresectable non-small cell lung cancer (NSCLC) whose disease has not progressed following platinum-based chemoradiation therapy.

The FDA granted the designation following the announcement of interim results from Phase Pacific Trial in May. The results confirmed that the product has met its primary endpoint, which was statistically significant and clinically meaningful progression-free survival (PFS). It also showed a favorable benefit/risk profile. AstraZeneca and its biologics research and development arm MedImmune are still awaiting results on overall survival.

A Second Breakthrough Designation

On Tuesday, i.e., one day following the breakthrough awarded designation for AZN drug Imfinzi, the FDA awarded AstraZeneca drug Acalabrutinib a breakthrough designation. Acalabrutinib is a Bruton tyrosine kinase (BTK) inhibitor in development for the treatment of multiple B-cell cancers in patients who have received at least one prior therapy.

The breakthrough designation is based on data from the Phase 2 ACE-LY-004 clinical trial in patients with relapsed or refractory mantle cell lymphoma (MCL). In addition to MCL, Acalabrutinib has demonstrated preliminary positive results on chronic lymphocytic leukemia (CLL). The drug is also in development for solid tumors.

Prohost Observations

The news is great not only for AstraZeneca, but also for the patients suffering from lung cancer and mantle cell lymphoma. Infinzi worked on a lung cancer whose prognosis remained poor with low long-term survival rate.  The same can be said about Mantle cell lymphoma, which is an aggressive form of B-cell non-Hodgkin’s lymphoma (NHL) with poor prognosis and accounts for approximately 3 percent to 6 percent of new NHL cases annually. It has a poor prognosis.

Bottom line. This news confirms Astrazenca’s solid scientific fundamentals and that is far from going down as described by some critics.

It is important noting that AZN’s drug Imfinzi is a checkpoint inhibitor immunotherapy antibody targeting PD-L1 and that it has demonstrated excellent results in clinical trials so far. One can speculate that it will have the same success in treating other cancers, which will be extraordinary news translated to saving thousands of lives and bringing billions of dollars to AZN’s coffers.

The quarterly financial results pattern’s adopted by traders – causing a selloff in the biotech sector has become nauseating. For us, the outcome of the selloff opens the door to accumulating the stocks that we love at lower prices.  

Expect Prohost Letter #412 Part 3 to be posted soon.

Prohost Forward-Looking: Material presented here is for informational purposes only. Nothing in this article should be taken as a solicitation to purchase or sell securities. Before buying or selling any stock you should do your own research and reach your own conclusion. Further, these are our ‘opinions’ and we may be wrong. We may have positions in securities mentioned in this article. You should take this into consideration before acting on any advice given in this article. If this makes you uncomfortable, then do not listen to our thoughts and opinions. The contents of this article do not take into consideration your individual investment objectives so consult with your own financial adviser before making an investment decision. Investing includes certain risks including loss of principal.

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