Welcome another edition of “3 Things In Biotech You Should Learn Today,” a daily digest of recent events from the world of pharma and biotechnology. Today, we’re tackling some exciting findings for conditions with notably poor prognosis, including breast cancer, amyloidosis, and post-partum bleeding.

Let’s get to the new!

The CDK4/6 race heats up with Lilly’s entry to the fray

Treatment options for hormone receptor-positive breast cancer have been expanding quickly with the approval of inhibitors targeting cyclin-dependent kinases 4 and 6 (CDK 4/6), first Pfizer’s (NYSE:PFE) palbociclib (which I have covered previously) and then the Novartis (NYSE:NVS) entry, ribociclib.

Both appear to be similarly effective in women with recurrent or metastatic breast cancer, with some differences seen between the adverse event profiles. Now, Eli Lilly’s (NYSE:LLY) abemaciclib is beginning to emerge from its late-stage clinical trial cocoon.

Recently, LLY announced that the MONARCH3 study, a phase 3 trial enrolling women with untreated advanced breast cancer, met its primary endpoint of progression-free survival compared with endocrine therapy alone. This study allowed patients to receive either letrozole or anastrozole, and addition of abemaciclib improved this outcome. LLY plans to present the full data later this year.

Looking forward: The CDK4/6 story is definitely an interesting one to follow. To date, these agents have not really distinguished themselves significantly, with a few exceptions relating to adverse events. Abemaciclib is currently receiving some hype because its dose schedule is continuous, whereas the other two CDK4/6 inhibitors must be given on a 3 weeks on/1 week off basis. It remains to be seen whether the relatively high rates of diarrhea will be seen in MONARCH3, since the dose was dropped from earlier studies from 200 mg to 150 mg. Still, abemaciclib offers an interesting bit of the new in this field, and I look forward to seeing the results of the MONARCH2 study at this year’s ASCO meeting.

Alnylam’s gene expression modifier show promise in amyloidosis

Familial amyloidosis is a rare but life-threatening inherited condition that results in the accumulation of certain proteins that eventually cause nerve damage and, eventually, fatal damage to the heart and kidneys. The only hope that patients have here is a liver transplant (because the protein in question is made mostly in the liver), but this carries a significant risk in itself.

As I detailed in a previous edition, gene-modifying CRISPR-based techs are starting to emerge as a potential treatment option for amyloidosis. Recently, Alnylam Pharmaceuticals (NASDAQ:ALNY) has been pursuing an antisense oligonucleotide-based approach to “knocking down” the expression of transthyretin, a drug called patisiran. This agent is being developed in partnership with Sanofi (NYSE:SNY).

ALNY recently presented results from their phase 2 study for patisiran in 24 patients with amyloidosis. In the overall population, 74% of patients experienced an improvement or no change in the neuropathy impairment score, and substantial reduction in the serum levels of transthyretin. Patients with signs of cardiac damage also showed improvement in cardiac-related biomarkers.

Looking forward: This study provides further support for the strategy of treating patients with this horrific hereditary condition using gene expression modification. ALNY now looks forward to presenting findings from the phase 3 APOLLO study later this year. But as it stands, the efficacy data look promising here. And all in all this should be an exciting field to follow in the years to come!

A Pfizer-developed agent that staves off fatal hemorrhage

Around the world, childbirth still presents a risk of fatal injury to mothers, mainly through uncontrolled bleeding. This creates a need for effective treatment strategies that can be delivered easily and quickly. Pfizer’s drug Cyklokapron has been approved for over 20 years to prevent hemorrhage in hemophilia patients when they have a tooth extraction.

A recent study published in The Lancet showed that administration of Cyklokapron significantly reduced the risk of death compared with placebo when given early in a bleeding event post-partum (155 [1.5%] deaths versus 191 [1.9%] in the active treatment and placebo arms, respectively).

However, Cyklokapron did not reduce the need for hysterectomy, which made the composite endpoint of death or hysterectomy not significantly different between the two arms.

Looking forward: These findings provide a clear picture of the benefit of this agent when delivered very early after the onset of bleeding. It may help to assuage a major cause of complication and death for women around the world, though it probably will not contribute substantially to PFE’s bottom line, since generic versions of Cyklokapron have been available for a long time. Still, progress made with existing drugs is very helpful, and it could perhaps lead to approval of this agent for women who are giving birth.


Clearly there are big things rumbling under the surface for breast cancer and amyloidosis. And the advent of an effective therapy to treat post-partum hemorrhage is exciting, as it reduces risk of death by 19%. These will all be very exciting to follow in the near future!

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Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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