3 Things In Biotech You Should Learn Today: July 13, 2017

Summary

• Pfizer gets the nod despite safety concerns in AML.
• Groundbreaking CAR-T cell ODAC thumbs-up.
• Lilly's newest breast cancer drug gets priority review.

Welcome to another edition of "3 Things In Biotech You Should Learn Today," a daily digest dedicated to helping you keep up with the news in biotech and pharmaceutical research.

And today's a big day with the FDA! Let's get to it.

Pfizer gets the nod despite safety concerns in AML

On July 11, Pfizer (PFE) faced a big oncology drug advisory committee (ODAC) meeting to discuss the fate of its antibody-drug conjugate gemtuzumab ozogamicin for treatment of patients with AML.

As of a few days ago, it seemed that there were some serious outstanding questions relating to the toxicity of this agent, with an increased level of severe liver toxicity casting some doubt on the future of gemtuzumab.

The ODAC agreed on a 6 to 1 vote that a revised "fractionated" dosing scheme sufficiently reduced the tolerability concerns for this agent. Moreover, the event-free survival observed in clinical trials was deemed meaningful.

So the scientists have now weighed in, and gemtuzumab ozogamicin is a go. The final FDA decision is expected in September.

Looking forward: This is definitely good news for PFE and for patients with AML, as 2017 has proven to be a banner year for treatment of this difficult form of leukemia. The lone dissenter indicated that he would have liked for treatment to be restricted to patients with favorable or intermediate-risk cytogenetics, but the panel ultimately decided that this should be a decision left up to patients and their doctors.

Groundbreaking CAR-T cell ODAC thumbs-up

Recently we covered the briefing document that Novartis (NVS) received for CTL019 in acute lymphoblastic leukemia, which highlighted some areas of concern that NVS would be asked to discuss with the oncology drug advisory committee.

Specifically, safety and manufacturing concerns had an outside shot at threatening the approval of CAR-T cells.

But it was not to be. The panel of experts voted unanimously to recommend the approval of CTL019. This goes to illustrate just how skewed the risk/benefit profile of this approach was, considering the risk of serious or even fatal complications. Furthermore, NVS might just be innovative enough with its distribution channels to get adequate therapy penetration into the treatment landscape.

Undoubtedly, there is going to be a large demand for it.

Looking forward: I was writing about another important finding in acute leukemia when news came down the wire about the ODAC thumbs-up. That entry was removed, and this one was inserted in its place. Not often will I drop everything to pay attention, but this was a big one. Now, the FDA is expected to give its final ruling on October 3, so it's going to be an anxious nearly three months.

Lilly's newest breast cancer drug gets priority review

The field of CDK4/6 inhibitors has been exploding ever since the 2015 approval of PFE's palbociclib. Since then, other competitors have launched themselves into the fray, with NVS gaining approval for its entry ribociclib.

Eli Lilly (LLY) is also intensely involved in this research with a late-stage compound, abemaciclib. This agent has shown encouraging results recently when used in combination with hormone therapy for metastatic, estrogen/progesterone receptor-positive breast cancer, and LLY previously announced breakthrough designation in 2015 for this agent.

Now, the FDA has informed LLY that abemaciclib has received priority review status for its NDA, which covers abemaciclib monotherapy in previously treated disease as well as the combination with endocrine therapy in previously untreated metastatic breast cancer.

Looking forward: LLY has a big chance here to differentiate itself on the marketplace, as it is (currently) the only drug that can be given continuously. The PFE and NVS entries both require three weeks on/one week off due to the risk of myelosuppression. Abemaciclib, on the other hand, trades the myelosuppression for gastrointestinal side effects, as well as possible cardiac toxicity. So its entry into the latest stages of development is an important harbinger of options for patients.

Conclusions

Lots of positive developments for patients with serious forms of cancer. This edition can be filed under the lessons about how much priority the FDA puts on new treatment options in cancer.

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