It was a busy week for the biotech sector. While the fourth-quarter earnings season is nearing the end, other pipeline and regulatory updates were in focus.

Recap of the Week’s Most Important Stories:

BLUE Stock Plunges on Update

bluebird bio’s BLUE shares plunged after the company announced that it has entered into a definitive agreement to be acquired by funds managed by global investment firms Carlyle and SK Capital Partners, LP.

Per the terms of the agreement, bluebird stockholders will receive $3.00 per share in cash and a contingent value right per share, entitling the holder to a payment of $6.84 per share in cash. This amount will become payable if bluebird’s current product portfolio achieves $600 million in net sales in any trailing 12-month period prior to or ending on Dec. 31, 2027.

bluebird’s board determined that this transaction is in the best interest of stockholders following a comprehensive review of strategic alternatives.

Updates From GILD

Gilead Sciences, Inc. GILD announced that the European Medicines Agency (“EMA”) has validated the company’s marketing authorization application and EU-Medicines for all applications for its twice-yearly injectable HIV-1 capsid inhibitor, lenacapavir. Gilead is seeking approval for lenacapavir for the prevention of HIV as pre-exposure prophylaxis (PrEP).

Both these applications are being assessed under an accelerated assessment review timeline as the EMA’s Committee for Medicinal Products for Human Use (CHMP) determined that twice-yearly lenacapavir for HIV prevention is a product of major interest for public health and therapeutic innovation. 

The European Commission also granted conditional marketing authorization for seladelpar for the treatment of primary biliary cholangitis in combination with ursodeoxycholic acid (UDCA) in adults who have an inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA. 

The EC’s approval followed the positive opinion of the CHMP of the EMA and is primarily based on results from the pivotal placebo-controlled phase III RESPONSE study.

Gilead currently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Regulatory Update From TRDA

Entrada TRDA announced that the FDA has lifted the clinical hold on ENTR-601-44. TRDA also obtained FDA authorization to initiate ELEVATE-44-102, a phase Ib multiple ascending dose (MAD)  clinical study of ENTR-601-44, for the potential treatment of Duchenne muscular dystrophy (DMD) in adult patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping. 

The global ELEVATE-44 program includes the ELEVATE-44-102 study in the United States and ELEVATE-44-201 outside the country.

The clinical hold was placed in December 2022. Thereafter, TRDA submitted additional data as requested by the company.

BMRN Stock Up on 2025 Outlook

BioMarin Pharmaceutical BMRN reported fourth-quarter adjusted earnings per share (EPS) of 92 cents, which beat the Zacks Consensus Estimate of 73 cents. Quarterly earnings rose 88% year over year, driven by higher product sales and lower operating expenses.

Total revenues were $747.3 million, which rose 16% year over year on a reported basis and 21% on a constant-currency basis. Strong sales of Voxzogo drove this upside. The top line beat the Zacks Consensus Estimate of $711.9 million.

For 2025, BioMarin expects to record total revenues in the range of $3.1-$3.2 billion, implying an increase of around 10% year over year at the midpoint. Management reiterated its target to achieve $4 billion in total revenues by 2027.
BMRN expects Voxzogo to be a significant contributor to the top line. It anticipates the drug’s sales to be in the range of $900-$950 million. Based on the encouraging uptake for Voxzogo, BioMarin expects the drug’s sales to witness a compound annual growth rate of more than 25% during 2023-2027.

It anticipates adjusted EPS to be in the range of $4.20-$4.40, suggesting 22% year-over-year growth at the midpoint. Shares of BioMarin gained after the announcement due to the encouraging guidance.

Performance

The Nasdaq Biotechnology Index has lost 0.15% in the past five trading sessions and MRNA’s shares have lost 6.91%. In the past six months, shares of MRNA have plunged 57.53%. (See the last biotech stock roundup here: Biotech Stock Roundup: MRNA’s Q4 Results, GILD’s Update & Other News)

Image Source: Zacks Investment Research

What's Next in Biotech?

Stay tuned for more pipeline updates.


 

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report

BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report

Gilead Sciences, Inc. (GILD) : Free Stock Analysis Report

bluebird bio, Inc. (BLUE) : Free Stock Analysis Report

Entrada Therapeutics, Inc. (TRDA) : Free Stock Analysis Report

This article originally published on Zacks Investment Research (zacks.com).

Zacks Investment Research