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PTC Therapeutics Provides Corporate Update and Reports First Quarter 2026 Financial Results

On May 7, 2026 PTC Therapeutics, Inc., (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ended March 31, 2026.

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"We are off to a strong start to 2026 with outstanding revenue performance this quarter that supports raising our full-year product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "The Sephience launch continues to be strong, with sustained momentum in the US and growing momentum internationally as more countries contribute to the global launch."

Key Corporate Updates

Q1 2026 total revenue of $273 million, with $226 million of product revenue
Sephience global launch continues strong momentum
Q1 2026 revenue of $125 million, including $112 million revenue in the US and $13 million revenue ex-US, up 36% from Q4 2025
1,244 total patients on commercial therapy worldwide as of March 31, 2026
Continued strong cadence of patient start forms in the US, with ~140 per month over past several months, surpassing 1,500 mark in the quarter
First commercial sales in Japan in Q1 2026, with revenue expected from up to 30 countries by year-end 2026
Reported positive topline results in April 2026 from 24-month interim analysis of PIVOT-HD long-term extension study of votoplam, an oral small molecule splicing agent
Dose-dependent benefit on cUHDRS in Stage 2 participants relative to natural history cohort, with 52% slowing of disease progression at 10 mg dose
Continued favorable safety and tolerability with no treatment-related NfL spikes
PTC and Novartis will complete data review and align on potential regulatory interactions
Findings support ongoing global Phase 3 INVEST‑HD study of votoplam led by Novartis
First patient dosed in Phase 3 INVEST-HD study, triggering $50 million milestone payment from Novartis to PTC in Q2 2026
Type C meeting with FDA held in April 2026 to discuss vatiquinone study to support NDA resubmission for Friedreich’s ataxia program
Based on meeting discussion and written feedback, PTC expects to initiate an open-label study using matched natural history control in Q3 2026
Primary endpoint will be change in mFARS from baseline to 24 months
First Quarter 2026 Financial Highlights

Total net product revenue and royalty revenue was $272.4 million for the first quarter of 2026, compared to $189.9 million for the first quarter of 2025.
Total net product revenue across the commercial portfolio was $225.6 million for the first quarter of 2026, compared to $153.4 million for the first quarter of 2025, representing a 47% increase.
Sephience net product revenues were $124.6 million for the first quarter of 2026, representing 36% growth compared to fourth quarter of 2025.
Translarna (ataluren) net product revenues were $59.0 million for the first quarter of 2026, compared to $86.2 million for the first quarter of 2025. Translarna first quarter of 2026 revenue includes a large government purchase order from Brazil.
Emflaza (deflazacort) net product revenues were $21.5 million for the first quarter of 2026, compared to $47.8 million for the first quarter of 2025, due to continued generic erosion.
Roche reported Evrysdi (risdiplam) sales of approximately 464 CHF million, resulting in royalty revenue of $46.8 million to PTC for the first quarter of 2026, compared to $36.4 million to PTC for the first quarter of 2025.
Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $100.9 million for the first quarter of 2026, compared to $109.0 million for the first quarter of 2025.
Non-GAAP R&D expenses were $89.7 million for the first quarter of 2026, excluding $11.1 million in non-cash, stock-based compensation expense, compared to $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $86.2 million for the first quarter of 2026, compared to $81.0 million for the first quarter of 2025.
Non-GAAP SG&A expenses were $73.9 million for the first quarter of 2026, excluding $12.3 million in non-cash, stock-based compensation expense, compared to $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense.
Net loss was $2.8 million for the first quarter of 2026, compared to net income of $866.6 million for the first quarter of 2025.
Cash, cash equivalents, and marketable securities were $1,892.5 million on March 31, 2026, compared to $1,945.4 million on December 31, 2025.
Shares issued and outstanding as of March 31, 2026, were 82,882,024.
PTC Updates Full-Year 2026 Financial Guidance

Total product revenue guidance raised to $750 to $850 million, with expected total revenue of $1.08 to $1.18 billion
GAAP R&D and SG&A expense guidance remains $775 to $815 million
Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million

(Press release, PTC Therapeutics, MAY 7, 2026, View Source [SID1234665343])

Prime Medicine Reports First Quarter 2026 Financial Results and Provides Business Updates

On May 7, 2026 Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a new class of differentiated one-time curative genetic therapies, reported financial results for the quarter ended March 31, 2026 and provided a business update.

"Prime Medicine continues to execute with clear momentum and a focused strategy designed to deliver on the transformative potential of Prime Editing," said Allan Reine, M.D., Chief Executive Officer of Prime Medicine. "We remain on track to file regulatory applications for PM577 in Wilson Disease in the first half of this year, and for PM647 in AATD mid-year, bringing two of our most advanced programs to the brink of clinical entry, with initial data from both expected in 2027. In parallel, we continue to progress toward a potential BLA filing for PM359, which, if successful, would bring a one-time, potentially curative therapy to people living with CGD, who today face a lifetime of debilitating infections and limited treatment options. With a strengthened leadership team and a continued commitment to disciplined capital allocation, we believe Prime Medicine is well positioned to execute across our pipeline and unlock the broad promise of Prime Editing for patients."

Prime Medicine’s Pipeline:

Prime Medicine is currently advancing in vivo programs to cure two of the largest genetic liver diseases, Wilson Disease (WD) and Alpha-1 Antitrypsin Deficiency (AATD). Prime Medicine expects to file an Investigational New Drug application (IND) and/or Clinical Trial Application (CTA) for PM577 in WD in the first half of 2026 and for PM647 in AATD in mid-2026; initial clinical data from both programs are expected in 2027.

Prime Medicine is also advancing an in vivo Cystic Fibrosis (CF) program with support from the Cystic Fibrosis Foundation, and efforts to develop Prime Edited CAR-T products for hematology, immunology and oncology in partnership with Bristol Myers Squibb. Additionally, following positive proof-of-concept data from the first two patients treated in its Phase 1/2 study of PM359 for the treatment of Chronic Granulomatous Disease (CGD), Prime Medicine continues to engage in regulatory dialogue with the U.S. Food and Drug Administration (FDA) toward a potential Biologics License Application (BLA) filing for PM359.

Recent Corporate Updates:

In April 2026, Prime Medicine announced the appointment of Svetlana Makhni as Chief Financial Officer (CFO). Ms. Makhni brings over 20 years of experience across biotechnology and healthcare CFO and investment banking roles, and will oversee Prime Medicine’s financial operations and strategy, including investor relations, and financial planning and analysis.
Upcoming Presentation:
Today, Prime Medicine announced that it will present new preclinical data for its Wilson Disease program at the American Society of Gene & Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2026 Annual Meeting (May 11-15 in Boston, MA). Details of the oral presentation are as follows:
•Session: Translational Gene Therapy and Editing in Storage Disorders

(Press release, Prime Medicine, MAY 7, 2026, View Source [SID1234665342])

Phio Pharmaceuticals Reports First Quarter 2026 Financial Results and Business Update

On May 7, 2026 Phio Pharmaceuticals Corp. (NASDAQ: PHIO) is a clinical-stage siRNA biopharmaceutical company developing therapeutics using its proprietary INTASYL gene silencing technology to eliminate cancer, reported its financial results for the quarter ended March 31, 2026, and provided a business update.

"We are enthusiastic with the successful completion of our Phase 1b clinical trial which now positions us for upcoming FDA interface which we expect will clarify next steps in advancing the PH-762 development program," said Robert Bitterman, President and Chief Executive Officer.

Recent Corporate Updates

PH-762 Progress

PH-762 was evaluated in a U.S. multi-center Phase 1b dose-escalating clinical trial through the intratumoral injection of PH-762 for the treatment of patients with cutaneous squamous cell carcinoma, melanoma and Merkel cell carcinoma. The trial (NCT 06014086) was designed to evaluate the safety and tolerability of neoadjuvant use of intratumorally injected PH-762, assess the tumor response, and determine the dose or dose range for continued study of PH-762. The study was fully enrolled in November 2025 with a total of 22 patients, 20 with cutaneous squamous cell carcinoma, one with melanoma and one with Merkel cell carcinoma. The clinical phase of the trial is complete, and the final data is currently being analyzed. While final study data is pending formal analysis, an FDA submission intended to propose and seek guidance for next steps in clinical study design for PH-762 is targeted for the second quarter of 2026.

Capital Sourcing

During 2025, Phio strengthened its balance sheet through a series of equity financings and warrant exercises that generated approximately $23.7 million in net proceeds. These transactions extended the Company’s cash runway into the first half of 2027 and will support ongoing clinical development, operational requirements and strategic initiatives.

Scientific News

The Company presented its Phase 1b clinical trial data for PH-762 at the American Academy of Dermatology (AAD) in the Late-Breaking Research Session in March 2026. In April 2026, the Company presented its lead clinical candidate, PH-762, and Phase 1b clinical trial results at multiple conferences including Deal Flow, Force Family Office Fireside Chats, the Investival Conference in Miami and the Centri Capital Conference in NYC.

Financial Results

Cash Position

As of March 31, 2026, the Company had cash and cash equivalents of approximately $17 million as compared with approximately $21 million at December 31, 2025.

In April 2026, the Company entered into an At The Market Agreement (ATM) with H.C. Wainwright & Co., LLC pursuant to which the Company may offer and sell shares of our Common Stock, having an aggregate price of up to $6.36 million.

Research and Development Expenses

Research and development expenses for the three months ended March 31, 2026 were $2.8 million, which was an increase of 215%, or $1.9 million, as compared with the three months ended March 31, 2025. This increase in research and development expenses was primarily driven by clinical trial, chemistry, manufacturing and controls (CMC) and toxicology expenses in connection with advancing our PH-762 program. Management believes that research and development expenses will continue to increase as we continue to advance our PH-762 program.

General and Administrative Expenses

General and administrative expenses for the three months ended March 31, 2026 were $1.4 million, which was an increase of 39%, or $400 thousand, as compared with the three months ended March 31, 2025. The increase in general and administrative expenses was primarily driven by employee related costs, investor outreach and professional fees.

Net Loss

Net loss was $ 4.0 million for the three months ended March 31, 2026 as compared with $1.8 million for the three months ended March 31, 2025. The increase in net loss was attributable to increases in research and development and general and administrative expenses cited above.

(Press release, Phio Pharmaceuticals, MAY 7, 2026, View Source [SID1234665341])

Personalis Reports First Quarter Results and Recent Highlights

On May 7, 2026 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for precision oncology, reported financial and operational results for the first quarter ended March 31, 2026, highlighted recent business accomplishments, and reaffirmed financial guidance for the full year 2026.

First Quarter and Recent Strategic and Operational Highlights

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Secured Milestone Medicare Coverage for Lung Cancer: Received Medicare coverage approval in the first quarter for the surveillance of cancer recurrence in lung cancer patients for Stage I to III non-small cell lung cancer (NSCLC). This marks the Company’s second major coverage decision in six months, alongside breast cancer.
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Announced Early Access Launch of Real-Time Variant Tracker: Launched a pioneering new feature for NeXT Personal that empowers clinicians to longitudinally track resistance and therapeutically targetable mutations during routine disease monitoring, and potentially optimize treatment.
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Published Neoadjuvant Treatment Monitoring Results in Breast Cancer: Featured data in the Journal of Clinical Oncology from the PREDICT-DNA prospective study for Triple-Negative (TNBC) and HER2+ breast cancer patients that showed NeXT Personal can outperform current standard approaches for predicting patient outcomes following neoadjuvant therapy (NAT).

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Presented Compelling Data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting:
○
Colorectal Cancer (CRC) Podium Presentation: Highlighted the ultrasensitive ctDNA detection by NeXT Personal for predicting and tracking response to neoadjuvant immunotherapy in CRC patients, demonstrating a remarkable 100% negative predictive value and 100% specificity for disease relapse following surgery.
○
Lung Cancer Poster Presentation: Demonstrated that ultrasensitive ctDNA monitoring with NeXT Personal successfully predicts the early response of immunotherapy in recurrent metastatic NSCLC patients.
First Quarter 2026 Financial Results Compared with First Quarter 2025

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Quarterly Revenue: $15.5 million compared with $20.6 million; reflecting the planned decline in non-core revenue as the company focuses on growing revenue from its strategic MRD offering.
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Clinical Revenue: Clinical test revenue of $1.4 million, compared with $0.3 million; delivered 7,815 clinical tests compared with 2,184, representing a 258% increase.

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Core Revenue Streams: Pharma testing services and all other customers contributed $11.2 million. Revenue from enterprise sales (Natera) and population sequencing (the VA MVP) totaled approximately $2.9 million.
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Strong Cash Position: Ended the quarter with approximately $233.2 million in cash, cash equivalents, and short-term investments. This includes approximately $21.0 million in net proceeds from the Company’s At-The-Market (ATM) sales program, executed at a weighted-average price of $10.00 per share.
CEO Commentary

"Our accomplishments in the first quarter demonstrated that our ‘Win-in-MRD’ strategy is working to establish NeXT Personal as the new standard for how cancer is detected and monitored," said Chris Hall, Chief Executive Officer of Personalis. " Delivering 26% sequential and 258% year-over-year clinical volume growth—especially during what is traditionally the industry’s toughest seasonal quarter reflects the strong market demand for our ultrasensitive NeXT Personal test. With new Medicare coverage for lung cancer joining our existing breast cancer win, Personalis now has a reimbursement success in two of the largest oncology indications. We are transforming our ultrasensitive MRD technology from a clinical leader into a potential commercial powerhouse and we remain firmly on track to grow our clinical revenue five-fold this year."

Full Year 2026 Outlook

Personalis reaffirmed the following guidance for the full year of 2026:

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Total company revenue in the range of $78.0 to $80.0 million.
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Clinical test volume scaling rapidly to a range of 43,000 to 45,000 tests, reflecting 171% growth year-over-year at the midpoint.
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Clinical revenue of $10.0 to $11.0 million, representing roughly a five-fold growth year-over-year, driven by Medicare reimbursement from breast and lung cancer surveillance.
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Revenue from pharma testing services and all other customers in the range of $55.0 to $56.0 million.
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Revenue from population sequencing and enterprise sales of approximately $13.0 million.
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Gross margin in the range of 15% to 20%, reflecting the strategic decision to accelerate clinical volume adoption ahead of full reimbursement coverage to establish market share.
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Net loss of approximately $105.0 million.
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Cash usage of approximately $100.0 million, driven by commercial investments to support projected clinical test volume growth and expansion.

(Press release, Personalis, MAY 7, 2026, View Source [SID1234665340])

Nykode Therapeutics Doses First Patient in Abili-T Phase II Randomized Trial
evaluating Abi-suva in HPV16-Positive, PD-L1-Positive Recurrent or Metastatic
Head and Neck Cancer Patients

On May 7, 2026 Nykode Therapeutics ASA (OSE: NYKD), a clinical stage biopharmaceutical company dedicated to the discovery and development of novel immunotherapies, reported the dosing of the first patient in Abili-T, a randomised Phase II clinical trial evaluating abi-suva (formerly VB10.16) in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD1 therapy, KEYTRUDA (pembrolizumab) versus pembrolizumab alone as first-line treatment for patients with HPV16-positive, PD-L1-positive recurrent or metastatic head and neck squamous cell carcinoma (1L r/m HNSCC).

The initiation of Abili-T marks a pivotal milestone in Nykode’s clinical development programme and reinforces the Company’s strategy to advance abi-suva as a targeted immunotherapy for the significant unmet medical need that exists in HPV16-driven cancers.

"Initiating the first patient in the Abili-T trial is an important step forward for Nykode and most importantly for patients with first-line recurrent/metastatic head and neck squamous cell carcinoma who continue to have significant unmet medical needs," said Michael Engsig, Chief Executive Officer at Nykode Therapeutics. "Abi-suva is designed to generate strong and durable HPV16-specific immune responses and we look forward to generating data in a randomized setting. We are grateful to the clinical investigators and patients participating in this study."

"There is a significant unmet need for patients with HPV16-driven head and neck cancer, particularly in the first-line setting," said Åse Bratland, Coordinating Investigator of the Abili-T trial, The Norwegian Radium Hospital, Oslo University Hospital. "Abi-suva is designed to generate a targeted and durable immune response and combining it with pembrolizumab may offer a meaningful new approach for these patients. We are hopeful that this study will provide important insights and, ultimately, lead to improved outcomes."

Abili-T is a randomised, open-label, multicentre Phase II trial evaluating abi-suva in combination with pembrolizumab versus pembrolizumab alone as first-line treatment in patients with HPV16-positive, PD-L1-positive recurrent or metastatic head and neck squamous cell carcinoma (1L r/m HNSCC).

The trial will enroll approximately 100 patients across 40 sites in Europe and Canada. Patients will be randomised to receive either abi-suva in combination with pembrolizumab or pembrolizumab alone. The primary endpoints are progression-free survival (PFS) and objective response rate (ORR). Secondary endpoints include duration of response (DOR), disease control rate (DCR), and overall survival (OS).

The Abili-T trial follows the successful completion of three prior clinical studies with abi-suva, VB-C-01, VB-C-02 and VB-C-03, which established the safety and tolerability of abi-suva as monotherapy and in combination with checkpoint inhibitors and demonstrated a strong and durable clinical effect in patients with advanced HPV16-driven cancers.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA. KEYTRUDA has been supplied by MSD for the Abili-T trial according to the clinical trial collaboration and supply agreement between Nykode and MSD.

Disease Background and Market Opportunity

The combined incidence of HPV16-positive HNSCC in the EU and US is approximately 63,000 new cases annually and the incidence of HPV16-driven HNSCC is rising.

Despite recent advances in immuno-oncology, patients with recurrent or metastatic HNSCC continue to face a significant unmet medical need. Current standard of care achieves an objective response rate of 19% and a median overall survival of 12.3 months. The majority of treatments currently in development for HNSCC are focused on HPV-negative disease, leaving the HPV16-positive patient population particularly underserved.

Beyond HNSCC, abi-suva has the potential to address a broader HPV16-driven cancer population including cervical, anal, vulvar and penile cancers with an annual incidence of approximately 134,000 patients annually in Europe and the US. This broader opportunity is supported by the clinical data generated in the VB-C-02 trial, which demonstrated strong and durable clinical effects in advanced cervical cancer patients, and in the VB-C-01 demonstrating a promising efficacy and favorable safety profile and confirmed the induction of robust HPV16-specific T-cell responses in pre-cancerous lesions (CIN).

(Press release, Nykode Therapeutics, MAY 7, 2026, View Source [SID1234665339])