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Syndax's (SNDX) Axatilimab Gets FDA's Orphan Drug Tag for GVHD

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  • SNDX
  • NBRV
  • RGEN

Syndax Pharmaceuticals, Inc. SNDX announced that the FDA has granted an orphan drug designation to its anti-CSF-1R monoclonal antibody, axatilimab, for the treatment of patients with chronic graft versus host disease (cGVHD), a life-threatening complication that occurs after stem cell transplant.

Shares of Syndax were up 6.7% following the announcement of the news on Wednesday. In fact, the stock has risen 0.5% so far this year against the industry’s decline of 3.7%.

price chart for SNDX
price chart for SNDX


Notably, the orphan drug designation is granted to drugs that are capable of treating rare diseases that affect less than 200,000 people in the United States. This tag also makes the company entitled to certain other benefits, including tax credits related to clinical trial expenses, an exemption from the FDA user fee and seven-year marketing exclusivity upon potential approval.

The phase II AGAVE-201 study is currently evaluating the safety and efficacy of three doses and schedules of axatilimab for treating patients with cGVHD. Top-line data from the same in expected in 2023.

The primary endpoint of the study is to check the objective response rate based on the 2014 NIH consensus criteria for GVHD, while secondary endpoints are the duration of response and improvement in modified Lee Symptom Scale score.

Please note that, in December 2020, Syndax announced updated data from its phase I study evaluating axatilimab in patients with cGVHD at the American Society of Hematology. Data from the same showed that treatment with axatilimab led to deep, durable responses and multiorgan clinical benefit in patients who are refractory to multiple therapeutic agents.

Per the company, axatilimab could become a meaningful therapeutic approach for cGVHD, as well as for other fibrotic diseases.

We note that, if successfully developed and upon potential approval, axatilimab is likely to face competition from Incyte’s INCY JAK1/JAK2 inhibitor, Jakafi (ruxolitinib), which is approved for the treatment of steroid-refractory acute GVHD in adult and pediatric patients aged 12 years or older.

Meanwhile, in February 2021, the FDA accepted for Priority Review the supplemental new drug application for Jakafi to treat steroid-refractory chronic GVHD in adult and pediatric patients aged 12 years and above. The regulatory body has set an action date of Jun 22, 2021.

Zacks Rank & Stocks to Consider

Syndax currently carries a Zacks Rank #4 (Sell).

Better-ranked stocks in the biotech sector include Nabriva Therapeutics AG NBRV and Repligen Corporation RGEN, both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Nabriva Therapeutics’ loss per share estimates have narrowed 45.8% for 2021 and 50.9% for 2022, over the past 60 days.

Repligen’s earnings estimates have been revised 15.1% upward for 2021 and 9.8% upward for 2022 over the past 60 days. The stock has increased 1.4% year to date.

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