We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
It was a busy week for the biotech sector. Key regulatory and pipeline updates from Sarepta Therapeutics (SRPT - Free Report) were in the spotlight during the week.
Recap of the Week’s Most Important Stories:
Sarepta’s DMD Therapy Approval: Sarepta Therapeutics, Inc. (SRPT - Free Report) received the FDA’s accelerated approval for Elevidys (delandistrogene moxeparvovec or SRP-9001), an adeno-associated virus-based gene therapy to treat ambulatory pediatric patients aged between four and five years with Duchenne muscular dystrophy (DMD).
The accelerated approval was based on the expression of Elevidys micro-dystrophin observed in patients treated with Elevidys. Continued approval for this indication will be contingent upon verification and description of clinical benefit in confirmatory trial(s). EMBARK, the global, randomized, double-blind, placebo-controlled phase III study for Elevidys, will serve as the post-marketing confirmatory trial and is fully enrolled, with top-line results expected in late 2023.
The road to approval faced several roadblocks. Last month, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (“CTGTAC”) voted 8:6, recommending approval for Elevidys. This was likely due to the FDA’s concerns about Elevidys. Per the CTGTAC, the clinical studies conducted to date do not provide unambiguous evidence that gene therapy will benefit DMD patients. Thereafter, Sarepta stated that the FDA has indicated that the regulatory body will potentially grant accelerated approval to the drug initially for use in DMD patients aged four-five years only. The agency has stated that it will grant a non-age restricted expansion to SRP-9001 provided the phase III EMBARK study achieves its objectives.
BMY’s Drug Approval: Bristol Myers Squibb (BMY - Free Report) announced that the European Commission has approved Camzyos (mavacamten, 2.5 mg, 5 mg, 10 mg, 15 mg capsules) for the treatment of symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy (HCM) in adult patients. The approval makes Camzyos the first and only cardiac myosin inhibitor approved in the European Union.
The approval was based on positive efficacy and safety results from two phase III studies, EXPLORER-HCM and VALOR-HCM. Both studies met all their primary and secondary endpoints, and Camzyos demonstrated efficacy across all primary and secondary endpoints, including improvements in exercise capacity and symptom burden for these patients.
In April 2023, the Committee for Medicinal Products for Human Use of the European Medicines Agency recommended approval of Camzyos for the same. The drug is already approved in the United States for the treatment of adults with symptomatic NYHA class II-III HCM to improve functional capacity and symptoms. It has also received regulatory approvals in various other countries.
FGEN Plummets on Drug Failure: Shares of FibroGen, Inc. (FGEN - Free Report) plunged after its late-stage study ZEPHYRUS-1 evaluating the safety and efficacy of pipeline candidate pamrevlumab in patients with idiopathic pulmonary fibrosis (IPF) was unsuccessful. The randomized, double-blind, placebo-controlled, multi-center phase III trial enrolled 356 patients with IPF. Patients were randomized (1:1) to receive either pamrevlumab or placebo for 48 weeks. The study did not meet the primary endpoint of change from baseline in forced vital capacity (FVC) at week 48, as the mean decline in FVC from baseline to week 48 was 260 ml in the pamrevlumab arm compared with 330 ml in the placebo arm. The secondary endpoint of time to disease progression was also not met.
As a result of disappointing results from the ZEPHYRUS-1 study, the company will now discontinue the second phase III study, ZEPHYRUS-2. Earlier in the month, FGEN announced that the phase III LELANTOS-1 placebo-controlled trial of pamrevlumab for the treatment of non-ambulatory patients with DMD on background corticosteroids did not meet the primary endpoint of the Performance of the Upper Limb 2.0 (PUL 2.0) score at week 52 compared with baseline.
ICPT Gets CRL for NASH Treatment: Intercept Pharmaceuticals announced that the FDA issued a complete response letter (CRL) in response to the company’s new drug application (NDA) seeking approval for obeticholic acid (OCA) for the treatment of pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH). The FDA indicated in the CRL that the NDA cannot be approved in its present form following a review. The NDA resubmission will require successful completion of the long-term outcomes phase of the REGENERATE study per the content of the CRL.
Consequently, Intercept announced a restructuring program. Intercept will discontinue all NASH-related investments and immediately begin the process of closing out the REGENERATE study. Intercept expects to substantially complete the trial shut-down process by the end of 2023.
Apart from closing out REGENERATE, Intercept is also terminating all other NASH-related spending within its R&D, commercial, medical affairs and administrative functions. The company will also cut one-third of its workforce to reduce operating expenses. Intercept expects to initiate workforce reductions in the third quarter of 2023, with a vast majority to be completed by the end of 2023. Nevertheless, Intercept plans to maintain the scale of its current field sales organization to support the growth potential of Ocaliva.
MoonLake Jumps on Study Results: Shares of the clinical-stage biotechnology company, MoonLake Immunotherapeutics (MLTX - Free Report) , jumped after it announced positive top-line results from its global phase II MIRA trial evaluating the efficacy and safety of the Nanobody sonelokimab in patients with moderate-to-severe hidradenitis suppurativa (HS). The trial met its primary endpoint with a significantly greater proportion of patients treated with both sonelokimab 120mg and 240mg achieving Hidradenitis Suppurativa Clinical Response (HiSCR) 75 compared to those on placebo at week 12.
The primary analysis was based on the most stringent type of analysis for such trials, intent-to-treat non-responder imputation (ITT-NRI). Other secondary endpoints also reached statistical significance with clinically meaningful improvements at week 12, including HiSCR90, improvements in International Hidradenitis Suppurativa Severity Score System (IHS) 4, and various patient-reported outcomes. MoonLake now looks forward to its next steps in advancing the candidate.
Performance
The Nasdaq Biotechnology Index has lost 3.01% in the past five trading sessions. Among the biotech giants, Regeneron has lost 10.11% during the period. Over the past six months, shares of MRNA have declined 30.31%. (See the last biotech stock roundup here: Biotech Stock Roundup: DICE Gains on LLY Buyout, MRSN Down on Update & More).
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.
See More Zacks Research for These Tickers
Normally $25 each - click below to receive one report FREE:
Image: Bigstock
Biotech Stock Roundup: SRPT's DMD Therapy Approval, ICPT's Setback & More Updates
It was a busy week for the biotech sector. Key regulatory and pipeline updates from Sarepta Therapeutics (SRPT - Free Report) were in the spotlight during the week.
Recap of the Week’s Most Important Stories:
Sarepta’s DMD Therapy Approval: Sarepta Therapeutics, Inc. (SRPT - Free Report) received the FDA’s accelerated approval for Elevidys (delandistrogene moxeparvovec or SRP-9001), an adeno-associated virus-based gene therapy to treat ambulatory pediatric patients aged between four and five years with Duchenne muscular dystrophy (DMD).
The accelerated approval was based on the expression of Elevidys micro-dystrophin observed in patients treated with Elevidys. Continued approval for this indication will be contingent upon verification and description of clinical benefit in confirmatory trial(s). EMBARK, the global, randomized, double-blind, placebo-controlled phase III study for Elevidys, will serve as the post-marketing confirmatory trial and is fully enrolled, with top-line results expected in late 2023.
The road to approval faced several roadblocks. Last month, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee (“CTGTAC”) voted 8:6, recommending approval for Elevidys. This was likely due to the FDA’s concerns about Elevidys. Per the CTGTAC, the clinical studies conducted to date do not provide unambiguous evidence that gene therapy will benefit DMD patients. Thereafter, Sarepta stated that the FDA has indicated that the regulatory body will potentially grant accelerated approval to the drug initially for use in DMD patients aged four-five years only. The agency has stated that it will grant a non-age restricted expansion to SRP-9001 provided the phase III EMBARK study achieves its objectives.
BMY’s Drug Approval: Bristol Myers Squibb (BMY - Free Report) announced that the European Commission has approved Camzyos (mavacamten, 2.5 mg, 5 mg, 10 mg, 15 mg capsules) for the treatment of symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy (HCM) in adult patients. The approval makes Camzyos the first and only cardiac myosin inhibitor approved in the European Union.
The approval was based on positive efficacy and safety results from two phase III studies, EXPLORER-HCM and VALOR-HCM. Both studies met all their primary and secondary endpoints, and Camzyos demonstrated efficacy across all primary and secondary endpoints, including improvements in exercise capacity and symptom burden for these patients.
In April 2023, the Committee for Medicinal Products for Human Use of the European Medicines Agency recommended approval of Camzyos for the same. The drug is already approved in the United States for the treatment of adults with symptomatic NYHA class II-III HCM to improve functional capacity and symptoms. It has also received regulatory approvals in various other countries.
FGEN Plummets on Drug Failure: Shares of FibroGen, Inc. (FGEN - Free Report) plunged after its late-stage study ZEPHYRUS-1 evaluating the safety and efficacy of pipeline candidate pamrevlumab in patients with idiopathic pulmonary fibrosis (IPF) was unsuccessful. The randomized, double-blind, placebo-controlled, multi-center phase III trial enrolled 356 patients with IPF. Patients were randomized (1:1) to receive either pamrevlumab or placebo for 48 weeks. The study did not meet the primary endpoint of change from baseline in forced vital capacity (FVC) at week 48, as the mean decline in FVC from baseline to week 48 was 260 ml in the pamrevlumab arm compared with 330 ml in the placebo arm. The secondary endpoint of time to disease progression was also not met.
As a result of disappointing results from the ZEPHYRUS-1 study, the company will now discontinue the second phase III study, ZEPHYRUS-2. Earlier in the month, FGEN announced that the phase III LELANTOS-1 placebo-controlled trial of pamrevlumab for the treatment of non-ambulatory patients with DMD on background corticosteroids did not meet the primary endpoint of the Performance of the Upper Limb 2.0 (PUL 2.0) score at week 52 compared with baseline.
ICPT Gets CRL for NASH Treatment: Intercept Pharmaceuticals announced that the FDA issued a complete response letter (CRL) in response to the company’s new drug application (NDA) seeking approval for obeticholic acid (OCA) for the treatment of pre-cirrhotic fibrosis due to nonalcoholic steatohepatitis (NASH). The FDA indicated in the CRL that the NDA cannot be approved in its present form following a review. The NDA resubmission will require successful completion of the long-term outcomes phase of the REGENERATE study per the content of the CRL.
Consequently, Intercept announced a restructuring program. Intercept will discontinue all NASH-related investments and immediately begin the process of closing out the REGENERATE study. Intercept expects to substantially complete the trial shut-down process by the end of 2023.
Apart from closing out REGENERATE, Intercept is also terminating all other NASH-related spending within its R&D, commercial, medical affairs and administrative functions. The company will also cut one-third of its workforce to reduce operating expenses. Intercept expects to initiate workforce reductions in the third quarter of 2023, with a vast majority to be completed by the end of 2023. Nevertheless, Intercept plans to maintain the scale of its current field sales organization to support the growth potential of Ocaliva.
Intercept currently has a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
MoonLake Jumps on Study Results: Shares of the clinical-stage biotechnology company, MoonLake Immunotherapeutics (MLTX - Free Report) , jumped after it announced positive top-line results from its global phase II MIRA trial evaluating the efficacy and safety of the Nanobody sonelokimab in patients with moderate-to-severe hidradenitis suppurativa (HS). The trial met its primary endpoint with a significantly greater proportion of patients treated with both sonelokimab 120mg and 240mg achieving Hidradenitis Suppurativa Clinical Response (HiSCR) 75 compared to those on placebo at week 12.
The primary analysis was based on the most stringent type of analysis for such trials, intent-to-treat non-responder imputation (ITT-NRI). Other secondary endpoints also reached statistical significance with clinically meaningful improvements at week 12, including HiSCR90, improvements in International Hidradenitis Suppurativa Severity Score System (IHS) 4, and various patient-reported outcomes. MoonLake now looks forward to its next steps in advancing the candidate.
Performance
The Nasdaq Biotechnology Index has lost 3.01% in the past five trading sessions. Among the biotech giants, Regeneron has lost 10.11% during the period. Over the past six months, shares of MRNA have declined 30.31%. (See the last biotech stock roundup here: Biotech Stock Roundup: DICE Gains on LLY Buyout, MRSN Down on Update & More).
Image Source: Zacks Investment Research
What's Next in Biotech?
Stay tuned for more pipeline updates.