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Precigen Showcases Rapid Commercialization Momentum and Growing Market Adoption of First-and-Only FDA-Approved Therapy for RRP at the 44th Annual J.P. Morgan Healthcare Conference

On January 12, 2026 Precigen, Inc. (Nasdaq: PGEN), a commercial-stage biopharmaceutical company specializing in the advancement of innovative precision medicines to improve the lives of patients, reported an update on the rapid commercialization momentum and growing market adoption of PAPZIMEOSTM (zopapogene imadenovec-drba), the first-and-only US Food and Drug Administration (FDA)-approved therapy for recurrent respiratory papillomatosis (RRP). Precigen’s company presentation at the 44th Annual J.P. Morgan Healthcare Conference will be on January 15, 2026 at 7:30 AM PT.

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"Commercialization of PAPZIMEOS is proceeding as planned following FDA approval, with rapid and broad adoption supported by compelling safety, efficacy, and long-term durability data, expanding payer access, and strong engagement across major medical centers and community practices nationwide," said Helen Sabzevari, PhD, President and CEO of Precigen. "We are seeing tremendous enthusiasm from physicians across the country who are eager to bring this therapy to their patients. Importantly, we are moving rapidly to make PAPZIMEOS available globally, highlighted by the European Medicines Agency validation of the Marketing Authorization Application. As the first-and-only FDA-approved therapy for adults with RRP, PAPZIMEOS is redefining the treatment paradigm, and the momentum we are seeing underscores its impact and value as we advance toward anticipated cash-flow break-even and global expansion."

"Our commercial launch continues to build strong momentum, with patient hub enrollment doubling since November, expanding commercial health plan coverage alongside Medicare and Medicaid, and near-complete field engagement across target centers," said Phil Tennant, Chief Commercial Officer of Precigen. "Manufacturing and supply chain capabilities are fully in place to meet current demand and anticipated growth, and patients are now receiving PAPZIMEOS nationwide. Importantly, PAPZIMEOS remains the only FDA-approved therapy for adults with RRP, providing an exclusive window to execute our commercial strategy. Based on these dynamics, we expect adoption to continue accelerating as PAPZIMEOS becomes established as the preferred first-line treatment and new standard of care."

PAPZIMEOS: Establishing a New Standard of Care for the Treatment of Adults with RRP

PAPZIMEOS full approval with broad label: In August 2025, the FDA granted full approval of PAPZIMEOS with a broad label and no requirement for a confirmatory trial for the treatment of adults with RRP.
PAPZIMEOS prescribing, treatment, and distribution: PAPZIMEOS is being prescribed nationwide, with patients actively receiving treatment. PAPZIMEOS is currently shipping to prescribers across the US, supported by established cold-chain logistics and coordinated solutions to address site-level needs.
Rapid commercialization: Rapid commercial launch execution is underway with over 96% of target centers engaged since full deployment of the sales team in September. To date, more than 200 patients have been registered in the PAPZIMEOS patient hub, doubling since November. In addition to these registered patients, a significant number of patients have been identified outside of the PAPZIMEOS hub through the Company’s field engagement efforts.
Positive payer coverage: Private health plan coverage is progressing rapidly with approximately 170 million lives covered to date, including the majority of leading insurers. PAPZIMEOS is also covered under Medicare and Medicaid.
Compelling long-term clinical and real-world evidence published: At AAO-HNSF 2025 and SITC (Free SITC Whitepaper) 2025, the Company reported long-term durable complete responses with PAPZIMEOS, and at ISPOR Europe 2025, the Company published data demonstrating the substantial healthcare resource utilization and patient-reported quality-of-life burden of RRP, underscoring the disease’s significant clinical, economic, and human impact.
EMA validation complete: Following a submission in November 2025, the European Medicines Agency has validated the Marketing Authorization Application for PAPZIMEOS for the treatment of adults with RRP.
About RRP
RRP is a rare, debilitating, and potentially life-threatening disease of the upper and lower respiratory tract caused by chronic HPV 6 or HPV 11 infection. RRP can lead to severe voice disturbance, compromised airways, and recurrent post-obstructive pneumonia. Although rare, RRP has the potential for transformation to malignant cancer and can be fatal. Management of RRP has primarily consisted of repeated surgeries, which do not address the underlying cause of the disease and can be associated with significant morbidity as well as significant patient and health system burden. As the number of lifetime surgeries increases, the risk for irreversible iatrogenic laryngeal injury increases with each surgery, and patients may undergo hundreds of these surgeries over their lifetimes. RRP can impact patients’ work and social lives, financial stability, and mental health. Patients with RRP can experience substantial impacts to daily living with decreased quality of life and high health care utilization. Based on an internal analysis of claims data and electronic health records, there are approximately 27,000 adult RRP patients in the US.

About PAPZIMEOS (zopapogene imadenovec-drba), for subcutaneous injection only
PAPZIMEOS is the first-and-only FDA-approved therapy for the treatment of adults with RRP and the first-and-only approved therapy to address the root cause of RRP. PAPZIMEOS 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. PAPZIMEOS is designed to generate an immune response directed against HPV 6 and HPV 11 proteins in patients with RRP. Discovered and designed in Precigen’s labs using Precigen’s proprietary AdenoVerse therapeutic platform, PAPZIMEOS represents a new therapeutic paradigm for RRP. Full prescribing information can be found at www.precigen.com/papzimeos-prescribing-information.pdf.

(Press release, Precigen, JAN 12, 2026, View Source [SID1234661987])

Novita Pharmaceuticals Announces FDA Orphan Drug Designation Granted to NP-G2-044 for the Treatment of Pancreatic Cancer

On January 12, 2026 Novita Pharmaceuticals, Inc. ("Novita"), a privately held, clinical-stage pharmaceutical company dedicated to developing novel cancer drugs through its proprietary fascin inhibitor technology, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its investigational small-molecule fascin inhibitor, NP-G2-044, for the treatment of pancreatic cancer. Pancreatic cancer is among the deadliest malignancies, with a five-year survival rate of approximately 12% and limited effective treatment options, particularly in advanced disease.

"FDA Orphan Drug Designation for our fascin inhibitor represents an important regulatory milestone for Novita and validates the Company’s scientific and clinical approach in the fight against pancreatic cancer, as it remains one of the most lethal solid tumors with limited therapeutic progress over decades," said Stewart Campbell, Chief Executive Officer of Novita Pharmaceuticals. "Fascin inhibition offers a novel approach with the potential to enhance anti-tumor immune activity and improve outcomes for patients with this devastating disease, and we look forward to continuing advancement of NP-G2-044 to address the high unmet need."

Orphan Drug Designation is granted to investigational therapies intended to treat rare diseases affecting fewer than 200,000 patients in the United States. The designation provides development incentives, including eligibility for tax credits on qualified clinical trial costs, exemption from certain FDA user fees, and the potential for seven years of market exclusivity upon regulatory approval.

The FDA’s Orphan Drug Designation was granted by the FDA’s Office of Orphan Products Development (OOPD) and includes treatment of pancreatic cancer in the setting of immune checkpoint inhibitor (ICI) therapy, which falls within the scope of the approved orphan indication. As noted by the FDA, orphan designation applies to the active moiety of the drug rather than a specific formulation, and eligibility for orphan drug exclusivity will ultimately depend on the approved indication and demonstration of clinical benefit relative to any approved therapies in the same indication.

About Novita’s Pioneering Research in Fascin Inhibition
Cancer metastasis is the primary cause of over 90% of cancer-related deaths, yet there is currently no drug on the market specifically targeting metastasis. Furthermore, while Immuno-Oncology (IO) therapies, particularly immune checkpoint inhibitors, have made significant strides in cancer treatment, a large proportion of patients do not respond to existing IO treatments. Novita aims to address both of these critical medical needs by developing fascin inhibitors, which target a key protein involved in tumor cell motility and highly expressed in tumor cells and antigen-presenting cells within tumor tissues. The Company’s lead asset, NP-G2-044, is a small-molecule fascin inhibitor that has demonstrated the ability to block metastasis in both preclinical and clinical studies. Additionally, when combined with immune checkpoint inhibitors, NP-G2-044 has shown potential to reinvigorate anti-tumor immune responses. Novita’s multicenter Phase 2 clinical trial, titled "NP-G2-044 as Monotherapy and Combination Therapy in Patients with Advanced or Metastatic Solid Tumor Malignancies," is currently ongoing.

(Press release, Novita Pharmaceuticals, JAN 12, 2026, View Source [SID1234661986])

CARsgen and Dispatch Bio Announce Clinical Collaboration to Evaluate Flare Platform and Zevor-cel in Solid Tumors

On January 12, 2026 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, and Dispatch Bio, a biotech company engineering a universal treatment across solid tumors leveraging its first-in-class Flare platform, reported a clinical collaboration to conduct a Phase 1 trial in China, planned to begin in 2026.

The trial will evaluate DISP-11, an investigational therapy leveraging Dispatch’s first-in-class Flare platform – including DV-10, the company’s novel tumor-specific virus – and CARsgen’s zevorcabtagene autoleucel (zevor-cel, R&D code: CT053), an autologous B-cell maturation antigen (BCMA)-targeting CAR T-cell therapy, in patients suffering from solid tumors. Zevor-cel is approved by the National Medical Products Administration (NMPA) in China for the treatment of multiple myeloma.

"CARsgen is working to transform outcomes for patients with solid tumors by advancing the potential of CAR T technologies," said Zonghai Li, M.D., Ph.D., Founder, Chairman of the Board, Chief Executive Officer and Chief Scientific Officer of CARsgen. "Dispatch’s Flare platform offers a differentiated and highly complementary approach to expanding where and how CAR T can be applied, particularly for solid tumors lacking specific targets. We are excited to explore this combination as part of our broader efforts to unlock meaningful benefit for patients."

In the planned study, patients with solid tumors of epithelial origin, which account for 90% of all solid tumors, will first receive DV-10, followed by zevor-cel.

"We are working to expand the impact of the Flare platform, and this collaboration represents a step forward in a region with infrastructure for efficient and impactful oncology development and significant unmet medical need," said Sabah Oney, Ph.D., Chief Executive Officer of Dispatch. "CARsgen’s scientific rigor and track record of rapid clinical advancement make them an ideal partner. As our second collaboration with a company that has successfully commercialized a CAR T therapy, this partnership reinforces the growing confidence in Flare’s potential across a variety of immunotherapies and will help shape our global development strategy."

About Zevor-cel

Zevor-cel is a fully human, autologous BCMA CAR T-cell product for the treatment of multiple myeloma (MM). Zevor-cel was approved by the NMPA on February 23, 2024, for the treatment of adult patients with relapsed or refractory MM who have progressed after at least three prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent). Zevor-cel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019.

(Press release, Carsgen Therapeutics, JAN 12, 2026, View Source [SID1234661985])

PIERRE FABRE PHARMACEUTICALS STATEMENT REGARDING RECEIPT OF COMPLETE RESPONSE LETTER FOR TABELECLEUCEL BIOLOGICS LICENSE APPLICATION FROM THE U.S. FOOD AND DRUG ADMINISTRATION

On January 12, 2026 Pierre Fabre Pharmaceuticals, Inc. reported a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) stating that the Agency is unable to approve the tabelecleucel Biologics License Application (BLA) in its present form.

We are surprised and deeply disappointed by the FDA’s decision, particularly given the urgent and life-threatening unmet medical need faced by patients with Epstein-Barr virus–positive post-transplant lymphoproliferative disease (EBV+ PTLD) after failure of standard-of-care therapy. These patients have no FDA-approved treatment options and a life expectancy often measured in weeks to months.

The BLA was resubmitted following clear alignment with the FDA on the acceptability of the resubmission criteria and fulfillment of the conditions outlined in the January 15, 2025, CRL, which identified a single GMP-related deficiency and raised no concerns regarding safety, efficacy, or trial design. Upon acceptance of the resubmission in July 2025, the FDA granted tabelecleucel accelerated approval status.

In the new CRL, despite acknowledging that the GMP issue had been resolved and raising no safety concerns, the FDA stated that it no longer considers the previously accepted single-arm ALLELE study to be adequate to support accelerated approval and requested a new study. This represents a significant and unexpected change in position, and one that is contrary to extensive dialogue with the Agency over more than five years.

We are concerned that this decision may have far-reaching consequences for the development of rare disease treatments, effectively creating barriers for generating clinical evidence within a unique patient population with ultra-rare conditions thereby significantly delaying—or preventing altogether— patient access to urgently needed therapies.

We firmly believe that tabelecleucel represents an important treatment advance for patients with EBV+ PTLD and that the totality of data supports its efficacy and safety. We intend to engage with the FDA to urgently pursue a path forward, in collaboration with Atara Biotherapeutics (Nasdaq: ATRA) and our clinical and patient partners, to enable timely accelerated approval of tabelecleucel. We continue to be committed to making tabelecleucel available to patients through our Expanded Access Program.

Approval and real-world use of tabelecleucel over several years in multiple countries outside the United States further support its clinical value. We remain fully committed to securing approval of this critical treatment option for U.S. patients and the physicians who care for them.

(Press release, Pierre Fabre, JAN 12, 2026, View Source [SID1234661984])

10x Genomics to Advance Research in Diagnostic Applications of Tumor Single Cell and Spatial Profiling

On January 12, 2026 10x Genomics, Inc. (Nasdaq: TXG), a leader in single cell and spatial biology, reported a collaboration with Dana-Farber Cancer Institute to analyze tumor samples from patients with major solid cancer types to uncover biological features associated with treatment response, resistance and disease progression. The company also announced plans to establish a CLIA-certified laboratory. Together, these efforts mark the beginning of a multi-year research initiative to incorporate single cell and spatial tumor analysis into potential diagnostic workflows to support cancer patient care.

The study with Dana-Farber intends to examine samples from hundreds of patients to evaluate therapeutic targets and relevant tumor microenvironment features for the most promising emerging therapies in oncology, including antibody-drug conjugates (ADCs), radioligand therapies (RLTs), bispecific antibodies, immune checkpoint inhibitors and other precision approaches. These treatments are reshaping the standard of care, yet patients can experience dramatically different outcomes. By understanding why patients respond differently to the same treatments, this research initiative may eventually help clinicians optimize therapy accordingly and move beyond current assays to capture the cellular ecosystems, immune activity and spatial context that influence how tumors respond to treatment.

Using 10x’s Chromium Flex single cell assay and Xenium spatial platform, the investigators will generate molecularly detailed maps of tumors that integrate both cellular composition and spatial architecture. By pairing these profiles with known clinical outcomes, the collaboration aims to reveal the biological features that distinguish patients who benefit from treatment from those who do not. Additionally, 10x and Dana-Farber will collaborate in defining actionable biomarkers for future clinical reporting, exploring how spatial and single cell insights, such as target expression patterns, immune contexture or indicators of therapeutic sensitivity, could be summarized in a format designed to support oncologists as they navigate increasingly complex treatment decisions.

"Collaborations like this are important for advancing precision oncology," said Himisha Beltran, MD, Director of Translational Research in Medical Oncology at Dana-Farber. "Integrating high-resolution tumor profiling with clinical outcomes allows us to study treatment response and resistance across solid tumors, generating insights that can inform future clinical applications."

In parallel with this scientific collaboration, 10x’s planned CLIA laboratory build-out will provide the regulated infrastructure needed for assay implementation, analytical validation and clinical sample processing, and also create the critical infrastructure necessary to deploy innovative diagnostic services for future clinical use.

"Through partnerships announced in 2025, we’ve already seen how single cell and spatial profiling can impact translational research, and we believe this is only the beginning," said Serge Saxonov, Co-founder and CEO of 10x Genomics. "Our goal is to accelerate the arrival of a world where deep, multidimensional biology directly informs patient care. That means continuing to empower our customers in their research, while also engaging in clinical collaborations like the one with Dana-Farber that help us advance how these insights translate into improved outcomes for patients. Establishing a CLIA lab is a natural next step for 10x and will strengthen the support we provide to investigators in academia and biopharma as they design, validate and bring forward the next generation of precision medicines."

This collaboration marks the beginning of a multi-year research effort through which 10x will collaborate with leading institutions to generate the scientific evidence needed to develop the clinical potential of single cell and spatial technologies. Additional studies and collaborations are planned, each contributing to the foundational work required to develop potential clinical tests in the future.

(Press release, 10x Genomics, JAN 12, 2026, View Source [SID1234661983])