ASH Preview: Celgene, Amgen, PD-1, CD-38, Agios, Epizyme and more

It is Celgene's ($CELG) world, we are just living in it--at least when it comes to the annual American Society of Hematology (ASH) conference. Hematology is Celgene's bread and butter, with mainstay Revlimid accounting for two-thirds of its revenue during the most recent quarter. The biotech giant has perfected the art of incrementally expanding revenue for its blockbuster by providing data for additional indications, longer treatment durations and more frequent treatment.

This year will be no exception, with Celgene detailing Phase III data for Revlimid plus dexamethasone to treat newly diagnosed multiple myeloma patients who aren't stem cell transplant candidates. The data were originally published in September in the New England Journal of Medicine. Revlimid is under regulatory review for expanding into the NMDD indication, with a PDUFA date of Feb. 22 and a CHMP decision expected during the first half of 2015.

Celgene expects Revlimid revenue to be almost $5 billion this year. It sees "significant runway on the growth trajectory for Revlimid" mostly due to the newly diagnosed indications in the U.S. and Europe, the biotech's president of global hematology and oncology Jackie Fouse said at the Credit Suisse Healthcare Conference on Nov. 11. She expects the indication could add "up to 10 market share points" in the U.S. In Europe, she said Celgene is starting essentially from a zero market share position, with "a lot of potential to increase share in the frontline setting."

The big biotech will also present second-line data for oral multiple myeloma treatment Pomalyst, which saw its sales grow 102% to $181 million during the third quarter and is currently approved as a third-line option. Celgene has more than 160 ASH abstracts, with 39 oral presentations at ASH.

But what may prove more interesting at ASH is the slew of early data that will be revealed by Celgene's army of biotech partners including Agios ($AGIO), Epizyme ($EPZM), Acceleron and MorphoSys. In recent years, Celgene has been exceptionally aggressive and imaginative with its partnering--drawing in a wide range of startups with innovative technologies.

Agios is up 18% since the day before ASH abstracts were released on Nov. 6, providing Wall Street a glimpse of what it should expect at the actual conference. That's despite an initial share price dip in the first few days as investors processed the massive influx of data.

"Agios' update on its IDH2 inhibitor AG-221 showed very compelling response rates with complete remissions lasting up to 4.5 months in patients with IDH2-positive advanced hematologic malignancies," Mario Linimeier of Medical Strategy told FierceBiotech. "One death caused by severe pneumonia was already reported at EHA but seems not to be alarming given that pneumonia is very common in AML." He sees the uptick since the abstracts were released as justified.

AG-221 is partnered under a deal with Celgene in which the big biotech pays all the development costs. Under the deal, Agios is eligible for up $120 million in milestones plus royalties on net sales. It also has the right to commercialize AG-221 in the U.S.

Agios has 6 ASH abstracts, including one for Phase I data for first-in-class IDH2 mutant inhibitor AG-221. The company also released Phase I data for its AG-120 in solid tumors recently. Based on both, Leerink analyst Howard Liang raised his target from $90 to $124. At Nov. 24, Agios was at about $97.

By contrast, another 2013 early-stage IPO company and Celgene partner, Epizyme, has taken a hit on ASH abstracts. It's down 21% after its DOT1L inhibitor EPZ-5676 failed to provide any new responses since those disclosed in January in leukemia patients with translocations of the MLL gene.

"This is quite disappointing and may raise questions on the viability of the compound," concluded Linimeier. Celgene has rights to Epizyme's DOT1L program, including EPZ-5676, and paid the biotech a $25 million milestone on the January data.

Also among Celgene's early stage partners at ASH are bluebird bio ($BLUE), Acceleron, and MorphoSys, which are up 10%, 9% and 3%, respectively, since abstracts for the conference were disclosed.

Bluebird will have an oral and a poster presentation on its ongoing Phase I/II trial of its LentiGlobin; the gene therapy isn't partnered with Celgene, but its chimeric antigen receptor-modified T cell program for oncology is.

Acceleron and Celgene have four ASH abstracts on Phase II data for sotatercept and luspatercept in end-stage renal disease, myelodysplastic syndrome (MDS) and beta-thalassemia. This includes first-time data on transfusion independence for both MDS and beta-thalassemia.

MorphoSys has three ASH abstracts for its MOR208 and MOR202 programs; the latter is partnered with Celgene. It will have preclinical data for MOR202, part of a new class of monoclonal antibody targeting CD38, in combination with Celgene's oral immunomodulator Pomalyst in multiple myeloma. Several more sets of early data will come in at ASH for CD38 targeting antibodies from Genmab ($GEN)/Johnson & Johnson ($JNJ), Sanofi ($SNY)/Immunogen ($IMGN) and Bristol-Myers Squibb ($BMY)/AbbVie ($ABBV).

Celgene will hold an investor event at the ASH conference on Dec. 7, which will doubtless provide further details on its own data and the advances of its partners.

Beyond Celgene, ASH is expected to feature:

• Data from Amgen that could ease some safety concerns about Kyprolis
• Early anti-PD1 data from Merck and Bristol-Myers Squibb will help show if hematology is as promising as solid tumors
• In a change of pace, Pharmacyclics will have more incremental data than big leaps
• Smaller caps trying for transformational data--TG Therapeutics, Karyopharm, Geron

-- Stacy Lawrence (email | Twitter)