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Biotech Stock Roundup: CRL for REGN, GILD's Drug Approval, LQDA Down on News
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As the earnings season is over in the biotech sector, the focus shifts to pipeline and regulatory updates. It was a busy week for the sector, with quite a few important drug approvals and other updates.
Recap of the Week’s Most Important Stories:
Regeneron Gets CRL: Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that it has received a complete response letter (CRL) from the FDA for its biologics license application (BLA) for pipeline candidate linvoseltamab. The BLA is seeking FDA approval of linvoseltamab for the treatment of relapsed/refractory (R/R) multiple myeloma (MM) that has progressed after at least three prior therapies.
Regeneron stated that the sole issue identified in the CRL is related to findings from a pre-approval inspection at a third-party fill/finish manufacturer for another company’s product candidate. The third-party fill/finish manufacturer has since informed Regeneron that it believes the issue has been resolved. The manufacturer is awaiting reinspection of its facility by the FDA that is expected to take place in the coming months.
The CRL delays a potential approval of the candidate. Linvoseltamab is also under review in the European Union.
Drug Approval for GILD: Gilead Sciences, Inc. (GILD - Free Report) obtained accelerated approval from the FDA for seladelpar under the brand name Livdelzi. The drug has been approved for the treatment of primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA), in adults who have had an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.
The accelerated approval was primarily based on data from the late-stage RESPONSE study. Results showed that 62% of the participants taking Livdelzi achieved the primary endpoint of composite biochemical response at month 12 compared to 20% of those taking placebo.
Treatment with Livdelzi led to the normalization of alkaline phosphatase (ALP) values, a cholestatic marker that is a predictor of risk for liver transplant and death, in 25% of trial participants at month 12. The accelerated approval was granted by the FDA to Livdelzi based on a reduction of ALP.
Improvement in survival or prevention of liver decompensation events has not been demonstrated. Continued approval of Livdelzi for the approved indication may be contingent on verification and description of clinical benefit in the confirmatory trial(s).
Livdelzi cannot be used for patients who have or develop decompensated cirrhosis. Seladelpar was added to GILD’s portfolio/pipeline through the acquisition of CymaBay Therapeutics Inc. for $4.3 billion in March 2024.
Updates From INCY: Incyte (INCY - Free Report) announced that the late-stage inMIND study on tafasitamab met its primary and secondary endpoints. This phase III study is evaluating the efficacy and safety of tafasitamab, a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody, or placebo in combination with Revlimid and Rituxan compared to Revlimid and Rituxan alone in patients with R/R follicular lymphoma (FL).
The trial met its primary endpoint of progression free survival (PFS) by investigator assessment in FL. It also met key secondary endpoints of PFS in the overall population by investigator assessment as well as the positron-emission tomography-complete response rate in the FDG-avid FL population. In addition, the secondary endpoint of PFS results by the blinded independent review is consistent with investigator-based PFS results.
Tafasitamab is marketed as Monjuvi in the United States and as Minjuvi in the ex-U.S. markets. The drug was granted accelerated approval by the FDA, in combination with Revlimid, for the treatment of adult patients with R/R diffuse large B-cell lymphoma.
Earlier, Incyte and partner Syndax Pharmaceuticals obtained FDA approval for axatilimab-csfr, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. The candidate was approved under the brand name Niktimvo.
Liquidia PAH Treatment Approval: Liquidia Corporation (LQDA - Free Report) announced that the FDA has granted tentative approval to Yutrepia (treprostinil) inhalation powder to treat adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). A tentative approval indicates that while a drug meets all regulatory requirement standards for quality, safety and efficacy set by the agency, it must await the expiration of regulatory exclusivity of a competing product before final approval can be granted.
However, shares of LQDA were down on the news as the FDA delayed granting full approval to Yutrepia. Instead, LQDA obtained only tentative approval for Yutrepia. The regulatory body pointed out that the final approval for the Liquidia product might be given after a three-year regulatory exclusivity granted to United Therapeutics’ Tyvaso Dry Powder Inhaler expires on May 23, 2025.
Tyvaso DPI is approved in PAH and PH-ILD indications. Liquidia claims that it will challenge this three-year regulatory exclusivity granted to United Therapeutics’ Tyvaso DPI to provide patients access to the drug with the least delay possible.
Performance
The Nasdaq Biotechnology Index has gained 3.02% in the past five trading sessions and MRNA’s shares have risen 4.87%. In the past six months, shares of REGN have rallied 25.91%. (See the last biotech stock roundup here: Biotech Stock Roundup: Biotech Stock Roundup: BMY, GSK, BIIB’s Q2 Results, VTVT Down on Setback)
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What's Next in Biotech?
Stay tuned for more pipeline updates.
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Biotech Stock Roundup: CRL for REGN, GILD's Drug Approval, LQDA Down on News
As the earnings season is over in the biotech sector, the focus shifts to pipeline and regulatory updates. It was a busy week for the sector, with quite a few important drug approvals and other updates.
Recap of the Week’s Most Important Stories:
Regeneron Gets CRL: Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that it has received a complete response letter (CRL) from the FDA for its biologics license application (BLA) for pipeline candidate linvoseltamab. The BLA is seeking FDA approval of linvoseltamab for the treatment of relapsed/refractory (R/R) multiple myeloma (MM) that has progressed after at least three prior therapies.
Regeneron stated that the sole issue identified in the CRL is related to findings from a pre-approval inspection at a third-party fill/finish manufacturer for another company’s product candidate. The third-party fill/finish manufacturer has since informed Regeneron that it believes the issue has been resolved. The manufacturer is awaiting reinspection of its facility by the FDA that is expected to take place in the coming months.
The CRL delays a potential approval of the candidate. Linvoseltamab is also under review in the European Union.
Regeneron currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
Drug Approval for GILD: Gilead Sciences, Inc. (GILD - Free Report) obtained accelerated approval from the FDA for seladelpar under the brand name Livdelzi. The drug has been approved for the treatment of primary biliary cholangitis (PBC), in combination with ursodeoxycholic acid (UDCA), in adults who have had an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.
The accelerated approval was primarily based on data from the late-stage RESPONSE study. Results showed that 62% of the participants taking Livdelzi achieved the primary endpoint of composite biochemical response at month 12 compared to 20% of those taking placebo.
Treatment with Livdelzi led to the normalization of alkaline phosphatase (ALP) values, a cholestatic marker that is a predictor of risk for liver transplant and death, in 25% of trial participants at month 12. The accelerated approval was granted by the FDA to Livdelzi based on a reduction of ALP.
Improvement in survival or prevention of liver decompensation events has not been demonstrated. Continued approval of Livdelzi for the approved indication may be contingent on verification and description of clinical benefit in the confirmatory trial(s).
Livdelzi cannot be used for patients who have or develop decompensated cirrhosis. Seladelpar was added to GILD’s portfolio/pipeline through the acquisition of CymaBay Therapeutics Inc. for $4.3 billion in March 2024.
Updates From INCY: Incyte (INCY - Free Report) announced that the late-stage inMIND study on tafasitamab met its primary and secondary endpoints. This phase III study is evaluating the efficacy and safety of tafasitamab, a humanized Fc-modified cytolytic CD19 targeting monoclonal antibody, or placebo in combination with Revlimid and Rituxan compared to Revlimid and Rituxan alone in patients with R/R follicular lymphoma (FL).
The trial met its primary endpoint of progression free survival (PFS) by investigator assessment in FL. It also met key secondary endpoints of PFS in the overall population by investigator assessment as well as the positron-emission tomography-complete response rate in the FDG-avid FL population. In addition, the secondary endpoint of PFS results by the blinded independent review is consistent with investigator-based PFS results.
Tafasitamab is marketed as Monjuvi in the United States and as Minjuvi in the ex-U.S. markets. The drug was granted accelerated approval by the FDA, in combination with Revlimid, for the treatment of adult patients with R/R diffuse large B-cell lymphoma.
Earlier, Incyte and partner Syndax Pharmaceuticals obtained FDA approval for axatilimab-csfr, an anti-CSF-1R antibody, for the treatment of chronic graft-versus-host disease after the failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg. The candidate was approved under the brand name Niktimvo.
Liquidia PAH Treatment Approval: Liquidia Corporation (LQDA - Free Report) announced that the FDA has granted tentative approval to Yutrepia (treprostinil) inhalation powder to treat adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). A tentative approval indicates that while a drug meets all regulatory requirement standards for quality, safety and efficacy set by the agency, it must await the expiration of regulatory exclusivity of a competing product before final approval can be granted.
However, shares of LQDA were down on the news as the FDA delayed granting full approval to Yutrepia. Instead, LQDA obtained only tentative approval for Yutrepia. The regulatory body pointed out that the final approval for the Liquidia product might be given after a three-year regulatory exclusivity granted to United Therapeutics’ Tyvaso Dry Powder Inhaler expires on May 23, 2025.
Tyvaso DPI is approved in PAH and PH-ILD indications. Liquidia claims that it will challenge this three-year regulatory exclusivity granted to United Therapeutics’ Tyvaso DPI to provide patients access to the drug with the least delay possible.
Performance
The Nasdaq Biotechnology Index has gained 3.02% in the past five trading sessions and MRNA’s shares have risen 4.87%. In the past six months, shares of REGN have rallied 25.91%. (See the last biotech stock roundup here: Biotech Stock Roundup: Biotech Stock Roundup: BMY, GSK, BIIB’s Q2 Results, VTVT Down on Setback)
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.