Biotech Stock Roundup: Alexion, Regeneron Miss Estimates; Setback for CTI BioPharma

Major biotech companies like Regeneron REGN and Alexion ALXN reported fourth quarter and full year 2015 results last week with both companies missing expectations for the fourth quarter. Meanwhile, companies like CTI BioPharma CTIC and BioCryst BCRX saw their shares plunging on disappointing pipeline updates.

Recap of the Week’s Most Important Stories

1. CTI BioPharma’s shares plunged 60% on news that the FDA has placed the company’s Investigational New Drug (IND) application for pacritinib on partial clinical hold due to safety issues. The agency subsequently informed that the candidate is now on full clinical hold. Excess mortality and other adverse events were observed in pacritinib-treated patients compared to the control arm in the PERSIST-1 study.

Pacritinib is being evaluated for the treatment of myelofibrosis, a chronic blood cancer. With the full clinical hold being announced, all patients currently on the drug have to discontinue treatment immediately with no patients allowed to be enrolled or start pacritinib as initial or crossover treatment. The company also withdrew its NDA which was submitted earlier this year.

2. BioCryst’s shares plunged 71% with the company announcing disappointing results from the OPuS-2 study on avoralstat as a liquid-filled soft gel formulation for the prophylactic treatment of hereditary angioedema (HAE) attacks.

3. A couple of major biotechs – Alexion and Regeneron – reported fourth quarter and full year 2015 results last week. Both companies failed to top expectations with fourth quarter revenues and earnings missing estimates. Alexion’s 2016 revenues and earnings are expected to be impacted by about $120 million and 31 cents, respectively, by currency headwinds (Read More: Alexion Misses on Earnings and Revenues in Q4, Gives View).

Meanwhile, Regeneron said that it continues to expect a gradual uptick in sales of its PCSK9 inhibitor, Praluent (Read More: Regeneron Q4 Earnings Lag, Gives 2016 View for Eylea Sales).

4. Sarepta’s SRPT shares slipped on news that the FDA action date for its experimental Duchenne muscular dystrophy (DMD) treatment, eteplirsen, has been extended by three months to May 26. Meanwhile, the rescheduled date for an advisory panel meeting to discuss the company’s regulatory application is yet to be provided.

So far in 2016, Sarepta’s shares are down 70%. Concerns regarding the company’s chances of gaining FDA approval for eteplirsen increased when the FDA issued briefing documents questioning the experimental treatment’s efficacy in mid-Jan 2016 ahead of an advisory panel meeting that was subsequently canceled due to inclement weather.

With another contender in the DMD market, BioMarin, getting a CRL for its treatment, Kyndrisa, investors remain jittery about Sarepta’s chances of gaining FDA approval for eteplirsen (Read More: Sarepta Hits 52-Week Low; Eteplirsen Review Period Extended).

5. Vertex VRTX, which has had a pretty good track record in expanding the label of its cystic fibrosis (CF) drug, Kalydeco, suffered a setback with the FDA issuing a CRL for the company’s sNDA. The company was looking to gain FDA approval for Kalydeco for use in a patient population that would cover more than 1,500 people in the U.S. (Read More:Vertex Down on CRL for Kalydeco Label Expansion).

Performance

While Gilead’s GILD shares recorded an increase of 5.49% last week, Vertex lost 11.91% during this period with shares reflecting the CRL for the Kalydeco sNDA. Meanwhile, Vertex lost 41.82% over the last six months.

The NASDAQ Biotechnology Index declined 4.8% over the last five trading days (See the last biotech stock roundup here: Biogen, Amgen Top Estimates; Zika Virus Grabs Attention).

What's Next in the Biotech World?

Several mid-sized and small biotech companies are yet to come out with fourth quarter results.

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ALEXION PHARMA (ALXN): Free Stock Analysis Report
 
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