UroGen wider-than-expected Q2 loss overshadows revenue beat

 UroGen Pharma Ltd (NASDAQ:URGN) reported second quarter revenue that exceeded analyst expectations, but shares fell 3.3% as the company posted a larger-than-expected quarterly loss amid rising expenses related to its recent product launch.

The biotech company, which specializes in urothelial and specialty cancer treatments, reported a second quarter loss of $1.05 per share, missing analyst estimates of a $0.83 per share loss. Revenue came in at $24.2 million, surpassing the consensus estimate of $23.13 million and representing an 11% increase compared to the same quarter last year. The revenue growth was driven by 7% higher underlying demand for its JELMYTO treatment and favorable pricing.

"The recent FDA approval of ZUSDURI for the treatment of adults with recurrent LG-IR-NMIBC represents a truly transformative milestone for patients and for UroGen, marking our evolution into a multi-product uro-oncology company and our leadership in the field," said Liz Barrett, President and Chief Executive Officer of UroGen.

The wider loss reflected significantly higher operating expenses, which jumped to $62.1 million from $45.5 million in the year-ago quarter. Research and development expenses increased by $3.5 million to $18.9 million, while selling, general and administrative expenses surged by $13.1 million to $43.2 million, primarily due to commercial preparation activities for the recently approved ZUSDURI.

UroGen maintained its full-year 2025 revenue guidance for JELMYTO at $94 to $98 million, representing year-over-year growth of approximately 8% to 12%. The company also reiterated its operating expense forecast of $215 to $225 million for the year.

As of June 30, 2025, UroGen had $161.6 million in cash, cash equivalents and marketable securities, down from $241.7 million at the end of 2024, reflecting the company’s continued investment in product launches and pipeline development.

https://www.investing.com/news/earnings/urogen-pharma-falls-as-widerthanexpected-q2-loss-overshadows-revenue-beat-93CH-4177086

Exact Sciences to acquire US rights for Freenome’s blood-based CRC test

 Freenome has entered an exclusive licence agreement with Exact Sciences, granting the latter the rights to commercialise Freenome's blood-based colorectal cancer (CRC) screening test in the US.

Exact Sciences plans to accelerate the market adoption of the CRC blood test by leveraging its commercial infrastructure.

Under the agreement, Exact Sciences will pay Freenome an upfront cash payment of $75m that is payable by this November.

Freenome is set to receive up to an additional $700m, contingent upon reaching specific milestones related to its CRC screening tests.

These milestones include a $100m payment upon receiving first-line approval from the Food and Drug Administration (FDA) for their inaugural test and another $100m for the approval of a subsequent, next-generation test that meets or exceeds certain performance criteria, such as a minimum of 19% advanced precancerous lesions (APL) sensitivity and 83% overall CRC sensitivity.

However, should the performance fall below these benchmarks, the payment would be adjusted accordingly.

Furthermore, Freenome could earn $500m if its test is classified as a first-line A or B test in the US Preventive Services Taskforce (USPSTF) guidelines or if it achieves certain payer-contracted coverage requirements.

Again, a decreased payment is applicable if the test is included in the USPSTF guidelines as a second-line A or B test.

Freenome could be entitled to obtain royalties that vary between 0% and 10%, depending on the profitability of the test, while also adhering to standard provisions for royalty stacking. However, if specific conditions are not fulfilled, Exact Sciences reserves the right to end the agreement.

https://finance.yahoo.com/news/exact-sciences-acquire-us-rights-091716831.html

Steris beats, ups outlook

STERIS plc (NYSE: STE) reported strong fiscal 2026 first quarter results, with total revenue increasing 9% to $1.4 billion and constant currency organic revenue growing 8%. The company's net income from continuing operations reached $177.4 million, or $1.79 per diluted share, while adjusted EPS grew to $2.34.

Performance was driven by growth across all segments: Healthcare revenue grew 8% to $974.7 million, Applied Sterilization Technologies increased 13% to $281.2 million, and Life Sciences rose 5% to $135.2 million. Free cash flow significantly improved to $326.5 million.

STERIS updated its fiscal 2026 outlook, raising revenue growth expectations to 8-9% from 6-7%, while maintaining adjusted EPS guidance of $9.90-$10.15. The company now expects free cash flow of approximately $820 million, up from previous guidance of $770 million.

https://www.stocktitan.net/news/STE/steris-announces-financial-results-for-fiscal-2026-first-bei0dhdh3d2f.html

Replimune Fiscal First Quarter 2026 Results and Corporate Update

Replimune Group (NASDAQ:REPL) reported fiscal Q1 2026 financial results and provided updates on its oncolytic immunotherapy pipeline. The company received a Complete Response Letter (CRL) from the FDA for RP1 BLA in advanced melanoma on July 22, 2025. Key financial metrics include cash position of $403.3M, increased R&D expenses of $57.8M (vs $43.0M YoY), and a net loss of $86.7M (vs $53.8M YoY).

Multiple clinical trials are progressing, including the Phase 3 IGNYTE-3 trial for RP1 in melanoma, ARTACUS trial showing 34.6% response rate in transplant patients, and the Phase 2/3 REVEAL trial for RP2 in uveal melanoma. The company expects its current cash to fund operations into Q4 2026.

https://www.stocktitan.net/news/REPL/replimune-reports-fiscal-first-quarter-2026-financial-results-and-vbwn8c5kljfb.html

'Despite Safety Drama, Sarepta Beats Q2 Estimates—But No Thanks to Elevidys Sales'

 

Sarepta did not hold an investor call for its second-quarter earnings report or provide an updated full-year revenue outlook.

Even as Sarepta’s shares are battered by patient deaths linked to its gene therapy portfolio and the surrounding FDA drama, the Massachusetts biotech still managed to outdo analyst forecasts in the second quarter.

For the three months ending June 30, Sarepta’s total revenues hit $611 million, a 68% increase from the same period last year and coming respectably ahead of consensus, which had the biotech’s revenue at $532 million. Sarepta opted not to hold an investor call for its Q2 report and did not provide an updated 2025 outlook.

William Blair noted that Sarepta’s performance in the quarter was “driven by [a] milestone payment from Roche” in conjunction with Elevidys’ approval in Japan, worth $63.5 million, instead of the gene therapy’s market performance. Indeed, while Elevidys, the company’s Duchenne muscular dystrophy gene therapy, surged 132% year-on-year to bring in nearly $282 in the quarter, it still suffered a 25% quarter-on-quarter decline.

“While the company posted a revenue beat, which translated to profitability for the quarter, we note that the results were less reliant on its product revenues,” William Blair wrote on Wednesday. Looking ahead, the analyst firm expects “lumpiness” in Elevidys’ sales in the third quarter, given the rough few months it’s had.

In March, Sarepta reported that a patient had died of acute liver failure following treatment with Elevidys. The complication is a known side effect of adeno-associated virus vectors used to deliver gene therapies. A second mortality followed months later, then, last month, news broke that Sarepta had documented a third death, but this time in a patient treated with a different investigational gene therapy for limb-girdle muscular dystrophy.

The fallout has been severe. Soon after the third death was made public, the FDA asked Sarepta to halt all U.S. shipments of Elevidys. After initially pushing back, the company eventually relented. Roche, which owns ex-U.S. rights to the gene therapy, followed suit and soon suspended deliveries to certain countries that based their domestic approvals on the FDA’s actions.

The matter has since been resolved. Last week, the FDA recommended that the voluntary hold on Elevidys shipments to ambulatory patients be lifted, but pressure on the company remains.

Sarepta CEO Doug Ingram addressed this back-and-forth with the FDA in the company’s news release on Wednesday, noting that the biotech is “very pleased” with the regulator’s decision to endorse the resumption of shipments to ambulatory patients. “We have already resumed deliveries,” Ingram confirmed.

Still, analysts at William Blair “believe the news headlines regarding the patient deaths will affect commercial interest in the near term, and we expect to see some hesitancy from patients and physicians once shipments resume,” the firm wrote. “Therefore, we think investors will be hesitant to reenter the stock until the company is able to provide updated Elevidys revenue guidance for the coming quarters.”

Jefferies, meanwhile, predicted that third quarter sales for Elevidys could be challenged given the upheavel. Sales for that period are likely to decline by half to $130 million before stabilizing.

https://www.biospace.com/business/despite-safety-drama-sarepta-beats-q2-estimates-but-no-thanks-to-elevidys-sales

'Novartis Continues Hunt for New Assets With Rumored Takeover of RNA Specialist Avidity'

 

There is no certainty that the buyout will come to pass, according to The Financial Times, which first reported the rumors.

Novartis has approached rare disease-focused Avidity Biosciences with an acquisition offer, The Financial Times reported on Wednesday, citing anonymous sources familiar with the matter.

There is no certainty that a deal will come to fruition, and other suitors could approach Avidity, the sources cautioned. Novartis has expressed its interest in Avidity in recent weeks, which noted that a buyout would likely be at a premium to Avidity’s market cap. As of market close Wednesday, Avidity was worth $5.82 billion.

The biotech’s shares surged 26% on Wednesday after rumors of the Novartis takeover broke.

Analysts at BMO Capital Markets, writing to investors Wednesday, called these buyout rumors “unconfirmed” but “reasonable.” Avidity is an attractive acquisition target for Novartis given the biotech’s “leadership in muscular RNAi therapies across” key neuromuscular indications “that could enable two first-in-disease . . . multi-billion dollar opportunities,” they added.

Avidity’s clinical candidates “target high unmet need neuromuscular indications that lack disease-modifying treatments,” BMO said, noting that these assets also “enable significant commercial synergies among them.”

Chief among these promising assets is the investigational antibody-oligonucleotide conjugate (AOC) delpacibart zotadirsen, also called del-zota, in development for the treatment of patients with Duchenne muscular dystrophy who are amenable to exon 44 skipping. Del-zota made it to BioSpace’s list of the five most promising DMD assets to watch for this year.

Phase I/II data showed that del-zota boosted dystrophin expression to 25% of normal levels. Last month, the FDA granted del-zota Breakthrough Therapy designation. Avidity at the time said that it remains “on track” to submit a Biologics License Application for the asset by the end of the year. The biotech also expects to be able to report topline Phase II data in the fourth quarter.

Avidity is also developing del-desiran for myotonic dystrophy type 1 and del-brax for facioscapulohumeral muscular dystrophy. It also has a handful of preclinical programs running for a variety of other neuromuscular indications.

For Novartis, the Avidity deal would continue the pharma’s deal-making spree in recent months. In June, for instance, the company signed a four-year pact with Flagship Pioneering’s startup ProFound Therapeutics, putting up to $750 million on the line per cardiovascular target that they work on. Details of the deal have been scant, however: The companies have not disclosed how many targets they are eyeing, nor have they named specific priority indications.

In April, Novartis dropped $1.7 billion to acquire Regulus Therapeutics and its miRNA-targeting molecules. A few months earlier, in February, the pharma made a $3.1 billion bet to buy Anthos Therapeutics, the current owner of abelacimab, an anticoagulant antibody that Novartis previously owned.

https://www.biospace.com/business/novartis-continues-hunt-for-new-assets-with-rumored-takeover-of-rna-specialist-avidity

'Big Pharma-Backed mRNA Startup Bags $153M Series B Amid Policy Headwinds'

 

Strand Therapeutics’ lead asset is STX-001, an intra-tumor self-replicating mRNA therapy that carries a payload expressing the immunomodulatory protein IL-12.

Strand Therapeutics on Thursday closed a $153 million series B round that will help it advance its pipeline of novel mRNA therapies for cancer and other chronic conditions.

The fundraising round, backed by some of the biggest names in pharma, including Eli Lilly, Amgen and Regeneron, brings Strand’s total raise value to more than $250 million, according to its news release on Thursday. The Massachusetts biotech brought in $97 million in series A earnings in November 2022 and bagged a $400,000 award from the National Institutes of Health a year earlier.

Strand is using the money to develop programmable mRNA therapies, with its pipeline anchored by the early-stage STX-001, an investigational construct that encodes for the immunomodulatory protein IL-12. STX-001 is delivered directly into a tumor in an attempt reshape its microenvironment, in turn triggering a systemic anti-cancer immune response. Strand is testing STX-001 for advanced solid tumors, particularly melanoma and triple-negative breast cancer.

Uniquely, Strand’s approach makes use of self-replicating mRNA. On its website, the biotech notes that its therapies include “logic circuits” that can help control and determine the location, timing and degree of expression of its payload, in turn “enabling precise and controlled delivery.”

STX-001 is in an ongoing first-in-human Phase I study in patients with solid tumors that are refractory to immune checkpoint inhibitors. STX-001 monotherapy has shown signals of treatment response, Strand reported in May this year. These results likewise detected several partial responses and a confirmed case of complete response.

Aside from STX-001, Strand is also advancing STX-003, which on Thursday the biotech called a “world-first systemically administrable mRNA therapy with tumor targeting.” STX-003 is also self-replicating and similarly carries an IL-12-expressing payload.

Strand expects to start dosing STX-003 in patients next year, as per a company announcement in May.

Strand’s mRNA push, however, could run into some high-level headwinds. On Wednesday, Health Secretary Robert F. Kennedy Jr. axed 22 contracts under the Biomedical Advanced Research and Development Authority that dealt with mRNA vaccine technology. Kennedy said that he elected to scrap these programs, collectively worth nearly $500 million, “because the data show these vaccines fail to protect effectively against upper respiratory infections like COVID and flu.” He did not offer evidence to support this claim.

There is no indication yet that this push will extend to the use of mRNA technology in other modalities outside of vaccines, but experts are already concerned that the political pushback against mRNA vaccines could have far-reaching implications. Speaking to BioSpace in May, Paul Offit, director of the Vaccine Education Center at Children’s Hospital of Philadelphia, said that mRNA “became a victim of disinformation, and a victim of sort of the political zeitgeist, which was to push back on vaccines.” Offit additionally expressed concern that this sentiment could extend to mRNA research into other applications, including cancer therapeutics.

https://www.biospace.com/business/big-pharma-backed-mrna-startup-bags-153m-series-b-amid-policy-headwinds