Insmed (NASDAQ: INSM) is breathing easier since the FDA has approved the company’s Brinsupri (brensocatib 10 mg and 25 mg tablets) as the first drug authorized to treat non-cystic fibrosis bronchiectasis (NCFB), with at least three analysts projecting peak-year sales climbing to multi-billion-dollar blockbuster levels.
The approval, announced August 12, brings a new treatment option to the approximately 500,000 Americans who have NCFB, according to a July 15 review article published in JAMA, with millions more diagnosed with the disease globally.
Exactly how many million have NCFB worldwide isn’t known because of differing diagnosis methods. However, the 2022 Global Impact of Respiratory Disease Report, published by the Forum of International Respiratory Societies, estimated a global presence for NCFB of between 67 and 566 per 100,000 inhabitants in Europe and North America, and as many as 1,200 per 100,000 inhabitants in China in adults ages 40 and older. Insmed last year estimated 400,000 NCFB patients in the European Union and 150,000 in Japan.
“For the first time, we have a treatment that directly targets neutrophilic inflammation and addresses a root cause of bronchiectasis exacerbations,” stated Doreen Addrizzo-Harris, MD, an investigator in the Phase III ASPEN trial (NCT04594369), one of two clinical studies whose data supported Insmed’s new drug application (NDA) for Brinsupri.
“Based on the strength of the data and the impact we’ve seen in patients, I believe this could become the new standard in non-cystic fibrosis bronchiectasis care,” declared Addrizzo-Harris, who is the Fiona and Stanley Druckenmiller Professor of Pulmonary, Critical Care, and Sleep Medicine at NYU Grossman School of Medicine, and director of the NYU Langone Health Bronchiectasis and NTM Program.
Martina Flammer, MD, Insmed’s chief medical officer
Added Martina Flammer, MD, Insmed’s chief medical officer: “The FDA approval of the first-ever treatment for non-cystic fibrosis bronchiectasis is a historic milestone for patients and for Insmed.”
Investors and analysts shared Insmed’s enthusiasm for Brinsupri. Insmed shares surged 17% on news of the approval this past week, rising from $109.55 on August 8 to $127.80 on Friday. The biggest one-day jump took place the day of the FDA approval, when shares jumped 8% from $112.89 to an even $122.
Shares of Insmed have nearly doubled, zooming 85% year to date from $69.04 on December 31, 2024, including a 24% spike in the month that started July 15, when shares closed at $102.87.
“New standard of care”
At least four analysts shared the enthusiasm shown by investors about Brunsupri, projecting peak year sales ranging from $3.7 billion by 2031 by Ritu Baral, a TD Cowen Securities managing director, health care–biotechnology research analyst, as reported by Reuters, to more than $7 billion by 2033 by Jessica Fye, a J.P. Morgan Securities managing director, equity research analyst-biotechnology.
“We expect Brinsupri to become the new standard of care in NCFB,” Fye wrote in a research note.
Brinsupri enjoyed positive data from the ASPEN trial as well as the Phase II WILLOW trial (NCT03218917). In ASPEN, patients treated with 25 mg Brinsupri reported significantly less decline in lung function, as measured by forced expiratory volume in one second (FEV₁) after using a bronchodilator, at week 52. Patients treated with Brinsupri 10 mg or 25 mg had reductions of 21.1% and 19.4%, respectively, in their annual rate of exacerbations compared to placebo.
More than 90% of physicians surveyed by Insmed told the company they intend to prescribe Brinsupri to patients with two or more pulmonary exacerbations, the company said in its second quarter presentation to investors.
Insmed’s data was in line with initial positive topline results announced in May 2024—results which, as GEN reported, sparked a stock-buying surge that caused Insmed’s stock price to skyrocket 150%.
“While overall expected, we see this approval and clean label as a clear win for INSM, and are confident in the company’s trajectory.”
Fye offered four reasons for her optimism:
List price: Both doses of Brinsupri will command the same $88,000/year list price, 10% above J.P. Morgan’s estimate of $80,000/year, and at the high end of guidance by Insmed last year that it would charge between $40,000/year and $96,000/year. Insmed says it will offer financial assistance to eligible patients, plus ongoing education and support through its inLighten patient support program.
Label: Both the 10 mg and 25 mg doses were approved with no limitation around prior exacerbations. The label includes the FEV1 benefit reported for the 25 mg dose.
Metrics: In addition to quarterly revenue, Insmed plans to provide as early launch metrics new patient starts, and the cumulative number of physicians who have prescribed Brinsupri, until the drug generates sufficient revenue for the company to start providing guidance to investors.
Overseas: J.P. Morgan anticipates international launches of Brinsupri in 2026 as applications have been accepted for review by the European Medicines Agency and the U.K.’s Medicines and Healthcare products Regulatory Agency, with a filing expected later this year in Japan. And we expect INSM to file in Japan this year.
Fye reiterated J.P. Morgan’s “Overweight” rating on Insmed shares.
Kelly Shi, PhD, a senior vice president and senior research analyst in biotechnology with Jefferies, and colleagues are projecting Brinsupri will rack up more than $6 billion in peak-year sales. She cited Brinsupri’s broad approval label and observed that she expected the FDA approval, given a run-up of Insmed stock in the year since the company released positive Phase III data on the drug.
“We believe INSM can grind higher from here on execution with brenso[catib]’s first-in-disease launch,” Shi wrote in a research note.
Enhancing M&A appeal
Shi added that the presence of a marketed blockbuster drug in Insmed’s portfolio would enhance its appeal to larger would-be buyers as a potential acquisition. Insmed is one of the 10 companies appearing in GEN’s most recent A-List of “Top 10 Takeover Targets of 2025.”
“We could also see higher M&A [merger and acquisition] premium in the stock post approval today as this could be an easy tuck-in for interested pharma with stronger commercial muscle,” Shi wrote.
Shi raised Jefferies’ price target on Insmed stock 15%, from $129 to $148 a share.
Also raising its price target on Insmed shares this past week was Joseph P. Schwartz, senior managing director, rare diseases, and a senior research analyst with Leerink Partners. Schwartz raised Leerink’s price target 6%, from $125 to $133 a share, and reiterated the firm’s “Outperform” rating on Insmed stock, with he and colleagues projecting peak-year sales for Brinsupri of more than $5 billion.
That forecast is in line with Insmed’s own estimate—made last year and repeated in its Q2 presentation—that Brinsupri could generate peak year sales of “>$5 billion” in NCFB alone.
“This marks an exciting milestone in the history of the company,” Schwartz wrote of Brinsupri’s approval in a research note. “While the approval of Brinsupri (brensocatib) was anticipated, it doesn’t diminish the significance of the news as INSM gears up to launch the drug.”
Particularly encouraging, Schwartz added, was the inclusion in Brinsupri’s label of inclusion of potential disease-modifying FEV1 benefit observed at the higher 25 mg dose in the label, “given this is an important metric and could drive adoption among pulmonologists.”
Schwartz and Fye of J.P. Morgan Securities projected that the percentage of “gross-to-net” (GTN) patients in the United States—patients whose potential revenue is lost to discounts, rebates, and other adjustments—will be 30% of those treated with Brinsupri, the midpoint of Insmed’s forecast of between 25% and 35%. The company said its talks with payers are ongoing.
Expansion plans
Key to the blockbuster peak-year sales forecasts is the expectation that Insmed will succeed in expanding Brinsupri’s prescribing label with additional indications. Insmed has guided investors to two upcoming releases of clinical data for the drug under its generic name of brensocatib:
By year’s end, the company expects to release data from the Phase II BiRCh trial (NCT06013241) assessing brensocatib in adults with chronic rhinosinusitis without nasal polyps.
During the first quarter of 2026, Insmed plans to announce data from a futility analysis portion of the Phase II CEDAR trial (NCT06685835), which is evaluating brensocatib in adults with moderate to severe hidradenitis suppurativa (HS).
Brinsupri is one of three drugs Insmed expects to have on the market in the coming years. The second, Arikayce® (amikacin liposome inhalation suspension or ALIS), is already approved and marketed to treat mycobacterium avium complex (MAC) lung disease in combination with multidrug therapy, for adults who still test positive for MAC lung disease after at least six months on multidrug treatment alone. During the first half of 2026, Insmed plans to read out data from the Phase III ENCORE trial (NCT04677569), which is studying Arikayce under its generic name in adults with nontuberculous mycobacterial lung infection caused by Mycobacterium avium complex.
The third drug, treprostinil palmitil inhalation powder (TPIP), will be evaluated in a Phase III trial set to be launched later in the second half of this year in pulmonary hypertension associated with interstitial lung disease (PH-ILD)—as well as a Phase III study in patients with pulmonary arterial hypertension (PAH) that is planned for initiation in early 2026.
In June, Insmed announced positive topline results from a Phase IIb trial (NCT05147805) evaluating a once-daily dosage of TPIP in patients with PAH. The study showed TPIP met its primary endpoint by generating a placebo-adjusted 35% reduction from baseline in pulmonary vascular resistance (PVR).
Precigen shares catapult on historic FDA approval
Precigen (NASDAQ: PGEN) shares catapulted 59% Friday from $1.85 to $2.94 after the company said it had been granted full FDA approval for the first-ever treatment indicated for adults with the rare disease of recurrent respiratory papillomatosis (RRP). Shares spiked 89% to $3.49 at 11 am, setting a 52-week high for the stock before sliding on apparent profit-taking.
Helen Sabzevari, PhD, Precigen’s president and CEO
Papzimeos (zopapogene imadenovec-drba) is a non-replicating adenoviral vector-based immunotherapy designed to express a fusion antigen comprising selected regions of human papillomavirus (HPV) types 6 and 11 proteins—the root cause of RRP. Papzimeos is delivered via four subcutaneous injections over 12 weeks.
Infection with HPV 6 or HPV 11 causes RRP, a lifelong neoplastic disease of the upper and lower respiratory tracts that can be fatal. According to Precigen, some 27,000 U.S. adults have RRP based on a recently updated internal analysis derived from claims data, and more than 125,000 patients outside the United States have the disease. Until now, the standard of care for RRP patients has consisted of numerous surgeries.
“Today marks a historic turning point. With the landmark FDA approval of Papzimeos and broad label, all adult RRP patients are now eligible for access to the first and only approved therapy that targets the root cause of the disease,” Helen Sabzevari, PhD, Precigen’s president and CEO, said in a statement. “This milestone affirms the power of our AdenoVerse platform and the exceptional capabilities of our team to rapidly advance a wholly novel therapy from discovery to approval considerably faster than industry benchmarks.”
The FDA approval is also a milestone for Precigen—its first-ever therapy to win FDA authorization—and for Sabzevari, who began her study of biology as a high school student in Iran, and continued her education in the United States after the Iranian Revolution of 1979. Speaking on GEN’s “Close to the Edge” video interview series in 2021, Sabzevari recounted her life and career, in which she advanced from a postdoc at the Scripps Research Institute, to a researcher at the NIH’s National Cancer Institute, to positions at Merck & Co. and Compass Therapeutics before joining Precigen as it refocused on drug development.
The FDA’s approval of Papzimeos came nearly two weeks before the agency’s August 27 target date for deciding on Precigen’s Biologics License Application (BLA) under the Prescription Drug User Fee Act (PDUFA). While Precigen completed submission of a rolling BLA in December 2024 under an accelerated approval pathway, and was granted Priority Review by the FDA, the agency gave Papzimeos a full approval, which does not require a confirmatory clinical trial.
At the European Society of Gene and Cell Therapy’s 2023 annual meeting, Precigen presented strong positive data from the Phase I portion of a first-in-human Phase I/II study (NCT04724980) of Papzimeos, then known as PRGN-2012, in patients with severe RRP. At the recommended Phase II dose of 5×1011 particle units, six of 12 patients (50%) receiving the therapy achieved a complete response by becoming “surgery-free,” no longer needing to be operated on for at least 12 months post-treatment. All complete responders continued to be surgery-free as of the data cutoff, ranging from 440 to 600 days depending on the patient.
Precigen has projected to investors a “multibillion $ global blockbuster potential” for Papzimeos, but has yet to quantify that to investors, or to project a list price for the RRP therapy—though that may emerge when Precigen holds a conference call with investors to discuss the FDA approval, “including key aspects of the label and commercialization.”
Papzimeos was developed through the company’s AdenoVerse® platform, which uses a library of adenovectors for efficient gene delivery of therapeutic effectors, immunomodulators, and vaccine antigens designed to modulate the immune system. Precigen said its AdenoVerse therapies have been shown to generate high-level and durable antigen-specific T-cell immune responses, as well as boost these responses via repeat administration.
Leaders and laggards
Editas Medicine (NASDAQ: EDIT) shares soared 34% from $2.29 to $3.08 on Wednesday, the first trading day after the company reported second-quarter results with a series of upbeat updates. Editas said it plans to select its first in vivo development candidate in September; file an IND application for its lead program by mid-2026 and achieve human proof-of-concept by the end of next year. Editas also announced the first IND/CTA application accepted for the CD19 HD Allo CAR T program being co-developed through a collaboration with Bristol Myers Squibb, triggering a milestone payment of an undisclosed amount to Editas, the first time Editas’ in-house developed technology will be used clinically in the allogenic CAR-T setting for the potential treatment of autoimmune disease. Among firms raising their 12-month price targets on Editas shares was Baird, which went from $4 to $6, maintaining its “Outperform” rating on the stock.
Generation Bio (NASDAQ: GBIO) shares ballooned 63% from $4.05 to $6.61 on Wednesday, after the company said it will slash its workforce approximately 90% as part of a phased strategic restructuring it is implementing between mid-August and the end of October. Generation Bio reported a workforce of 115 employees as of December 31, 2024, according to its Form 10-K annual report for last year, so a 90% cut would translate to about 104 jobs being eliminated. Generation Bio also said it has begun exploring strategic alternatives focused on maximizing shareholder value, with TD Cowen acting as the company’s financial advisor.
IO Biotech (NASDAQ: IOBT) shares yo-yoed this past week, starting with a 42% nosedive from $1.81 to $1.05 on August 11 after the company reported results from its pivotal Phase III trial (NCT05155254) assessing its immune-modulatory, off-the-shelf cancer vaccine Cylembio® (imsapepimut and etimupepimut, adjuvanted) in combination with Merck & Co. (NYSE: MRK)’s Keytruda® (pembrolizumab) vs. Keytruda alone as a first-line treatment in 407 patients with unresectable or metastatic (advanced) melanoma. The combination showed clinical improvement in the primary endpoint of progression-free survival (PFS) vs. Keytruda alone after 3.5 years (19.4 months vs. 11.0 months), but narrowly missed statistical significance. On Friday, however, IO shares leaped 69% from $1.28 to $2.16 after investors shared the company’s optimism about Cylembio: “We are focused on discussing the results with the FDA and determining the next steps for a potential submission of a [BLA] for the treatment of advanced melanoma,” stated Mai-Britt Zocca, PhD, IO’s president and CEO.
The post StockWatch: After FDA Approval, Analysts Predict Blockbuster Sales for Insmed Drug appeared first on GEN – Genetic Engineering and Biotechnology News.
