BERKELEY HEIGHTS, N.J., Jan. 06, 2026 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully integrated, commercial biotechnology company, announced today that Tonix management will present and host investor meetings at the following January 2026 investor conferences. Sachs Associates 9th Annual Neuroscience Innovation Forum Company Presentation Presenter: Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals Date: Sunday, January ...

Summary of Pharming Group Conference Call Company Overview - Pharming Group is a Dutch-based biotech company primarily operating in the US, known for its recombinant protein platform and the drug RUCONEST, which has been on the market for 10 years [3][4] - The company has transitioned from a one-asset firm to a high-growth biotech with two market assets, RUCONEST and Joenja, both experiencing double-digit growth [3][5] Financial Performance - Pharming reported strong third-quarter results, raising revenue guidance due to the growth of RUCONEST (29% year-on-year in Q4) and Joenja (35% growth) [5][6] - The company aims to maintain double-digit growth while investing in high-growth assets from generated cash [10][32] Product Insights RUCONEST - RUCONEST serves a specific segment of the Hemophilia A (HA) market, targeting patients who are not controlled by prophylactic treatments and have severe crises [7][9] - The drug's unique value proposition includes its ability to replace the missing protein and provide a fast onset of action through IV administration [7][8] - Pharming does not foresee any new treatments that could significantly impact RUCONEST's market share, expecting continued growth [9][10] - The company has exited non-US markets due to financial unsustainability, with less than 1.5% of RUCONEST sales coming from those regions [11][12] Joenja - Joenja is positioned as a high-growth drug with potential to become Pharming's first billion-dollar product, currently indicated for APDS (Activated PI3K Delta Syndrome) [14][15] - The company is expanding Joenja's indications to pediatric patients, with FDA approval expected by the end of January, which could increase the addressable market by 30% [16][18] - There is also an opportunity to reclassify Variant of Uncertain Significance (VUS) patients as APDS patients, potentially increasing the patient base by 50% [17][18] Market Expansion - Joenja has launched successfully in the UK, with plans for further expansion into Europe and Japan, pending regulatory approvals [23][24] - The company is focused on ensuring appropriate pricing that reflects the drug's value in new markets [24] Acquisition and Pipeline - Pharming's acquisition of Abliva for KL1333, aimed at treating primary mitochondrial disease, is seen as a strategic addition to its rare disease portfolio [25][26] - KL1333 has a well-defined patient population and is in registrational phase 2 trials, with significant market potential [26][28] Operational Strategy - The company is focused on optimal capital allocation to drive growth and has recently cut 20% of G&A headcount to reallocate resources towards growth catalysts [32][33] - Pharming emphasizes the importance of disciplined management to enhance value creation in the long term [33] Conclusion - Pharming Group is positioned for continued growth through its existing products and strategic acquisitions, with a strong focus on expanding its market presence and optimizing operational efficiencies [32][33]

Core Insights - ANI Pharmaceuticals reported Q3 CY2025 results exceeding market revenue expectations, with a year-on-year sales increase of 53.6% to $227.8 million [1][3] - The company's full-year revenue guidance of $863.5 million at the midpoint is 1.8% above analysts' estimates, and its non-GAAP profit of $2.04 per share surpassed consensus estimates by 15.2% [1][5] Financial Performance - Revenue for Q3 was $227.8 million, beating analyst estimates of $214.1 million, reflecting a 53.6% year-on-year growth and a 6.4% beat [5] - Adjusted EPS was reported at $2.04, exceeding analyst estimates of $1.77 by 15.2% [5] - Adjusted EBITDA reached $59.6 million, surpassing analyst estimates of $55.24 million, with a margin of 26.2% [5] - The company raised its full-year revenue guidance to $863.5 million from $830.5 million, marking a 4% increase [5] - Full-year Adjusted EPS guidance was lifted to $7.51 at the midpoint, a 4.7% increase [5] - EBITDA guidance for the full year is set at $224.5 million at the midpoint, above analyst estimates of $221.9 million [5] - Operating margin improved to 15.9%, up from -13.8% in the same quarter last year [5] - Market capitalization stands at $1.87 billion [5] Business Segments and Growth Drivers - The significant growth was attributed to strong performance in the Rare Disease and Generics segments, with Cortrophin Gel nearly doubling net revenue compared to the prior year [3] - The company experienced record new patient starts and expanded prescriber adoption, particularly in pulmonology and ophthalmology [3] - Management emphasized strong underlying demand and addressable patient populations as key drivers of success [3] Future Outlook - Updated guidance reflects expectations of continued growth, particularly from the Rare Disease portfolio, with a focus on Cortrophin Gel [4] - The company aims to expand clinical evidence, invest in commercial initiatives, and enhance patient convenience [4] - Management expressed confidence in the multi-year growth trajectory of Cortrophin and acknowledged ongoing efforts to support ILUVIEN's adoption [4] - Future business development opportunities are anticipated to expand the Rare Disease segment [4]

Core Insights - Tonix Pharmaceuticals Holding Corp. is a fully-integrated biotechnology company with a focus on developing and marketing products for various medical conditions, including fibromyalgia and acute migraine [3]. Company Overview - Tonix has received FDA approval for Tonmya, a first-in-class, non-opioid analgesic for fibromyalgia, marking the first new prescription medicine approval for this condition in over 15 years [3]. - The company markets two treatments for acute migraine in adults and has a diverse development portfolio targeting central nervous system disorders, immunology, rare diseases, and infectious diseases [3]. - Key products in development include TNX-102 SL for acute stress reaction and major depressive disorder, TNX-1500 for organ transplant rejection and autoimmune diseases, and TNX-2900 for Prader-Willi syndrome [3]. - Tonix is also developing vaccines and monoclonal antibodies for infectious diseases, including TNX-801 for mpox and smallpox, and TNX-4800 for Lyme Disease prevention [3]. - The company has a contract with the U.S. Department of Defense for TNX-4200, a broad-spectrum antiviral agent, valued at up to $34 million over five years [3]. Upcoming Events - Seth Lederman, M.D., the CEO of Tonix, will present at the Stifel 2025 Healthcare Conference on November 13, 2025 [1][2]. - Investors can arrange meetings with the company's management during the conference through their Stifel representative [2]. Additional Information - A webcast of the presentation will be available on the company's website, with a replay accessible for 90 days post-event [2]. - Tonix operates a state-of-the-art infectious disease research facility in Frederick, Maryland [3].

Company Overview - Tonix Pharmaceuticals Holding Corp. is a fully-integrated commercial biopharmaceutical company with marketed products and a pipeline of development candidates [3] - The company has received FDA approval for Tonmya™, a first-in-class, non-opioid analgesic for the treatment of fibromyalgia, marking the first approval for a new prescription medicine for this condition in over 15 years [3] - Tonix also markets two treatments for acute migraine in adults and focuses on central nervous system disorders, immunology, immuno-oncology, rare diseases, and infectious diseases [3] Pipeline and Development - The company’s development portfolio includes TNX-102 SL for acute stress reaction and major depressive disorder, funded by the U.S. Department of Defense [3] - Tonix's immunology portfolio features TNX-1500, a monoclonal antibody for preventing organ transplant rejection and treating autoimmune diseases [3] - The rare disease portfolio includes TNX-2900 for Prader-Willi syndrome, while the infectious disease portfolio includes TNX-801 for mpox and smallpox, and TNX-4800 for Lyme Disease prevention [3] - TNX-4200 is a broad-spectrum antiviral agent with a contract from the U.S. DoD for up to $34 million over five years [3] Upcoming Events - Seth Lederman, M.D., CEO of Tonix Pharmaceuticals, will present at BIO-Europe 2025 on November 4, 2025, highlighting the company's strategy and pipeline [1][2]

Core Insights - Tonix Pharmaceuticals is conducting a pilot study named FOCUS to evaluate its investigational intranasal potentiated oxytocin products in patients with Arginine-Vasopressin Deficiency (AVP-D), a rare endocrine disorder linked to oxytocin deficiency and mental health issues [1][2] Group 1: Study Overview - The FOCUS study is a randomized, double-blind, placebo-controlled crossover trial aimed at generating preliminary data for future clinical studies on oxytocin replacement therapy in AVP-D [1][2] - The study will assess the effects of two different doses of investigational intranasal oxytocin products (6 IU, TNX-2900 and 24 IU, TNX-1900) on anxiety, depression, and socioemotional functioning in patients with AVP-D [2] Group 2: Product Information - TNX-1900 and TNX-2900 are based on Tonix's patented intranasal magnesium-potentiated oxytocin formulations, designed to enhance oxytocin receptor binding and its effects on trigeminal neurons [3] - TNX-1900 is being developed for chronic migraine prevention, while TNX-2900 targets Prader-Willi syndrome in children and adolescents [3] Group 3: Company Background - Tonix Pharmaceuticals is a fully integrated biotechnology company with a focus on central nervous system disorders, immunology, and rare diseases, among other areas [10] - The company has received FDA approval for Tonmya™, a non-opioid analgesic for fibromyalgia, marking the first new prescription medicine approval for this condition in over 15 years [10]

Impact of Funding Cuts - NIH funding cuts do not directly impact the company's current business [1] - NIH funding cuts will require others to offset research and innovation progress [2] Healthcare Economics & Rare Diseases - Rare diseases have a trillion dollar economic impact in the United States [3] - A significant portion of the economic impact of rare diseases is due to lack of diagnosis, leading to treatment of symptoms and hospitalizations [3] - Early diagnosis of diseases can save the healthcare system significant dollars [4] - The company delivers better clinical outcomes and saves the healthcare system money [2]

Core Insights - The biotech investment strategy focuses on a mix of commercial stage (85%) and clinical stage (15%) companies, emphasizing disciplined position sizing and a long-term perspective on sales growth and clinical momentum [5][8][21]. Investment Strategy - The investment approach is characterized by strict position sizing rules, with a maximum of 10% in any commercial stage company and 5% in clinical stage companies [6]. - The strategy involves trading infrequently, typically two to four times a month, to avoid overtrading and to focus on quality investments [7][8]. - The community aspect of the ROTY Biotech Community allows for collaborative idea sharing and constructive criticism, enhancing investment decisions [9][10][15]. Market Trends - Recent successes in gene therapy and rare diseases are attracting more generalist investors to the biotech sector, creating a "halo effect" that could lead to increased investment [17][21]. - The FDA's favorable regulatory environment for rare diseases is seen as a significant tailwind for biotech investments [21][41]. Company Examples - Syndax Pharmaceuticals (SNDX) has shown a 60% gain since entry, driven by the approval of their lead drug and positive market reception [32][33]. - Geron Corporation (GERN) is highlighted as a potential investment opportunity due to its novel drug for blood cancer, which is currently undervalued relative to its peak sales potential [49][51]. Risk Management - Investors are advised to maintain a trade log to analyze performance and replicate successful strategies while avoiding past mistakes [22]. - The importance of understanding personal risk tolerance is emphasized, particularly in a volatile sector like biotech [30][31].

Financial Data and Key Metrics Changes - The company is preparing to file its New Drug Application (NDA) for bitopertin in October, with a potential PDUFA date between June and October of the following year, marking a significant milestone for the company [4][5][11] - The company reported a strong reduction in protoporphyrin-9 (PP9) levels with a P-value of less than 0.001, indicating clinically meaningful results from its phase 2 studies [9][10] Business Line Data and Key Metrics Changes - The lead program, bitopertin, is focused on treating erythropoietic protoporphyria, with plans for an accelerated approval pathway [4][5] - The second program is expected to present data on anemia of myelofibrosis and anemia of chronic kidney disease at upcoming conferences [5][6] Market Data and Key Metrics Changes - The company identified approximately 14,000 diagnosed patients in the U.S. for its lead indication, with expectations for rapid enrollment in clinical trials [19] - The market for bitopertin is expected to be significant, with pricing comparisons to existing therapies like Scenesse, which is priced at $300,000 to $350,000 per year [36] Company Strategy and Development Direction - The company is focused on increasing disease awareness and ensuring that healthcare providers are informed about available therapies for rare diseases [23][24] - The company plans to leverage claims data to identify and engage with physicians who treat diagnosed patients, enhancing its commercial strategy [24][25] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the regulatory pathway for bitopertin, citing positive interactions with the FDA regarding the use of PP9 as a surrogate endpoint [11][12] - The company is well-funded with $650 million on its balance sheet, providing a runway into 2028 to support ongoing trials and commercial preparations [62] Other Important Information - The company has orphan drug designation for bitopertin, providing seven years of market exclusivity in the U.S. [37] - The company is also exploring additional indications for its iron modulation programs, indicating a broad pipeline strategy [62] Q&A Session Summary Question: Can you recap the interactions with the FDA regarding the accelerated approval path for bitopertin? - Management detailed a series of meetings with the FDA that confirmed the rationale for an accelerated approval pathway based on phase 2 data and the use of PP9 as a surrogate endpoint [11][12] Question: What are the key differences between the Apollo and Aurora trials? - The Apollo trial will focus on measuring the time patients can spend in light as a co-primary endpoint, alongside the reduction of PP9 levels [19][20] Question: How does the company plan to address the patient population for bitopertin? - The company plans to segment the patient population based on engagement with healthcare providers, identifying approximately 6,000 engaged patients and 8,000 less engaged patients [27][28] Question: What is the expected pricing strategy for bitopertin? - The company anticipates pricing in line with existing therapies for rare diseases, potentially around $300,000 to $350,000 per year [36] Question: How does the company view the competitive landscape for its anemia treatments? - Management highlighted the significant unmet need in treating anemia in myelofibrosis, positioning their drug as the only one currently in development for this indication [43][44]

Regulatory Landscape - FDA agreed to reconsider its rejection of a drug for one rare disease [1] - FDA rejected a medicine for another rare disease based on "strange reasoning" [1]