The BeyondSpring roller coaster has plunged downward again. While BeyondSpring wowed investors with its phase 3 lung cancer data in August, the FDA found less to like in another indication, rejecting the drug on the grounds that the single registrational trial was too flimsy to show the drug’s benefit.
BeyondSpring’s FDA filing covered the use of plinabulin in chemotherapy-induced neutropenia (CIN), a key dose-limiting toxicity of the cancer treatment. CIN was initially seen as the main indication for plinabulin, but BeyondSpring raised hopes for the molecule in August by linking its use to improved overall survival in non-small cell lung cancer (NSCLC).
Subsequent updates have taken the shine off the summer romance between BeyondSpring and its investors, who sent its share price surging by 170% to above $26 when the NSCLC data dropped but drove it back down to around $13 by the time of the FDA decision on the CIN filing. The complete response letter brought BeyondSpring’s share price down to around $4.
That steep fall reflects the FDA’s request for BeyondSpring to run a second well-controlled trial in CIN to confirm the effect seen in the first study, which suggests there is no quick way back for the company in the indication.
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The FDA’s demand raises the question of why BeyondSpring thought it could win approval on the basis of one phase 3 trial. Having spoken to management at the company, the Jefferies analysts think BeyondSpring and the FDA got their wires crossed at an earlier meeting.
“Our understanding is that after BYSI's pre-NDA mtg and orientation mtg w/ FDA in May, there still must have been some misalignment on expectations; it seems BYSI thought FDA would consider totality of the data (6 studies), but FDA only relied on the ph.III, which was determined to be insufficient. Mgmt said FDA did not make pt demographics a primary issue,” the analysts wrote.
BeyondSpring is yet to commit to a second study, instead saying that it will work with the FDA on a possible future clinical pathway that may include another trial. More details will emerge after BeyondSpring meets with the FDA, which analysts at Jefferies expect to happen early next year.
The FDA and BeyondSpring are also meeting to discuss a potential filing for approval in NSCLC. The lung cancer indication is a major opportunity for BeyondSpring—giving it a chance to recover quickly from the CIN rejection—but the Jefferies analysts “see more uncertainty in NSCLC” after the CRL. Noting weaknesses in subgroups and long-term overall survival data, the analysts raised concerns the FDA “may have similar issues w/ NSCLC package too.”