On January 9, 2024 Revolution Medicine presented its corporate presentation (Presentation, Revolution Medicines, JAN 9, 2024, View Source [SID1234639166]).

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On January 9, 2024 Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene elimination, insertion, and excision, reported completion of a strategic transaction with TG Therapeutics, Inc. (Nasdaq: TGTX) for an exclusive license to develop Azercabtagene Zapreleucel (azer-cel) for autoimmune diseases, and other indications outside of cancer (Press release, Precision Biosciences, JAN 9, 2024, View Source [SID1234639165]).

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"We are excited to extend the utility of our allogeneic CAR T assets into immunology by collaborating with TG Therapeutics as they advance novel treatments for B-cell diseases. Key factors in our decision to partner with the TG team include their recent development, regulatory and commercial successes in the multiple sclerosis space, which we believe are strong indicators of the commitment and expertise they will bring to the development of azer-cel in autoimmune diseases," said Michael Amoroso, President and Chief Executive Officer at Precision BioSciences. "As TG Therapeutics assumes development of azer-cel for immunology, Precision will remain focused on capitalizing on the utility of ARCUS for gene elimination and gene insertion, beginning with our wholly owned PBGENE-HBV program for chronic hepatitis B and PBGENE-PMM for primary mitochondrial myopathy."

"After an extensive review of the CAR T products available for development in immunology, we are excited to bring azer-cel into our portfolio as we look to expand our offerings for patients suffering from autoimmune diseases," said Michael S. Weiss, Chairman and Chief Executive Officer of TG Therapeutics. "We are pleased to partner with Precision BioSciences for azer-cel, and the equity investment we are making is indicative of our optimism in the near- and long-term opportunities for ARCUS for in vivo gene editing."

In exchange for global rights to azer-cel for autoimmune diseases and indications outside of cancer, Precision will receive upfront and potential near-term economics valued at $17.5 million. The upfront payment of $7.5 million will consist of cash and the purchase of 2,920,816 shares of Precision common stock by TG Therapeutics at a price of $0.77 per share, a 100% premium to the 30-day volume-weighted average price (VWAP) prior to purchase. Precision will also receive $2.5 million within 12 months, as an equity investment in Precision’s common stock at 100% premium to the then 30-day VWAP prior to purchase. Upon the achievement of certain near-term clinical milestones, Precision will receive an additional $7.5 million payment in cash and the purchase of Precision common stock by TG Therapeutics at a 100% premium to the then current 30-day VWAP. Precision is eligible to receive up to $288 million in additional milestone payments based on the achievement of certain clinical, regulatory, and commercial milestones, in addition to high-single-digit to low-double-digit royalties on net sales.

"With this deal and the Imugene oncology collaboration for azer-cel announced in August 2023, Precision has now completed two cell therapy collaborations to realize value from our allogeneic CAR T platform while enabling development of azer-cel for patients in diseases with high unmet need. These transactions are expected to extend our runway and will fund continued development of our wholly owned in vivo gene editing programs. As a result of these two accretive partnerships, Precision has received or is eligible to receive $47 million in upfront and potential near-term payments and has the potential to receive more than $900 million in development, regulatory and commercial milestone payments," added Mr. Amoroso.

Although it has not finalized its full financial results for the year ended December 31, 2023, Precision expects to report that it had approximately $116 million in cash and cash equivalents as of December 31, 2023. Upfront and potential near-term cash from azer-cel transactions, along with existing cash and cash equivalents, expected operational receipts, continued fiscal and operating discipline, availability of Precision’s at-the-market (ATM) facility, and available credit are expected to extend Precision’s cash runway into the first half of 2026 and through clinical phase 1 readouts for its wholly owned HBV and PMM programs.

Precision will continue to evaluate potential partners for other assets from its allogeneic CAR T platform that are no longer being developed internally, including PBCAR19B stealth cell and BCMA targeting CAR T assets for multiple myeloma.

On January 9, 2024 Orphelia Pharma, a pharmaceutical company dedicated to the development and marketing of pediatric and orphan medicines, announces the publication of two scientific articles about KIZFIZO, the first pediatric drinkable formulation of temozolomide for the treatment of refractory or relapsed neuroblastoma, which is under registration review under centralised procedure by the EMA (Press release, ORPHELIA Pharma, JAN 9, 2024, View Source;utm_medium=rss&utm_campaign=orphelia-pharma-announces-the-publication-of-two-scientific-articles-on-kizfizo-the-first-pediatric-drinkable-formulation-of-temozolomide-for-the-treatment-of-refractory-or-relapsed-neuroblastoma [SID1234639164]).

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The first article, entitled Dispensing Oral Temozolomide in Children: "Precision and Stability of a Novel and Ready to Use Liquid Formulation in Comparison with Capsule Derived Mixtures", published in Pharmaceutics, demonstrates that the clinical practice of administering temozolomide capsules (Temodal) by mixing the content with food or drinks results in significant underexposure compared to administering the ready-to-use KIZFIZO 40 mg/ml oral suspension.

Tests for the accuracy of the administered dose and stability of temozolomide were performed with the capsule contents mixed with food vehicles (apple juice, applesauce, cream, milk and mashed potatoes) and compared to an equivalent dose of KIZFIZO. The dose delivered was systematically lower with capsule-based preparations, regardless of the vehicle used.

In addition, certain food vehicles (dessert cream, milk, mashed potatoes) had a significant effect on the chemical degradation of temozolomide, with the significant increase of its hydrolysis product, amino-imidazole-carboxamide (AIC).

The second article, also published in Pharmaceutics, as : A Multicenter Randomized Bioequivalence Study of a Novel Ready-to-Use Temozolomide Oral Suspension vs. Temozolomide Capsules", demonstrates the bioequivalence between KIZFIZO oral suspension and the capsule form of temozolomide (Temodal).

In conclusion, the administration of KIZFIZO, which is bioequivalent with the capsule form of temozolomide, avoids the misuse associated with the administration of capsules in the paediatric population. This ready to use oral liquid formulation, which is taste masked and ensures the chemical stability of temozolomide, accurately delivers the prescribed dose.

About KIZFIZO 40 mg/ml

KIZFIZO (temozolomide oral suspension, 40 mg/ml) is a ready-to-use oral liquid pediatric formulation of temozolomide developed for use in the treatment of relapsed or refractory high-risk neuroblastoma, which carry a very poor prognosis. This age-adapted and taste-masked formulation delivers an accurate dose in a small volume, while avoiding drug handling and caregiver exposure to temozolomide. It is the result of a collaboration between the pharmacists and clinicians at Gustave Roussy hospital and the development team at Orphelia Pharma.

In March 2022, KIZFIZO was granted Early Access Authorization (Autorisation d’Accès Précoce) by the French authorities, for the treatment of refractory and relapsed high-risk neuroblastoma as monotherapy or in combination with irinotecan or topotecan.

KIZFIZO has received an Orphan Drug Designation from the EMA and the FDA and the formulation is covered by granted patents in Europe and the US. The company filed an MAA with the EMA in the summer of 2023.

On January 9, 2024 Oric Pharmaceuticals presented its corporate presentation (Presentation, ORIC Pharmaceuticals, JAN 9, 2024, View Source [SID1234639163]).

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On January 9, 2024 Nerviano Medical Sciences S.r.l. (NMS), a part of NMS Group S.p.A. (NMS Group) and Nerviano Medical Sciences, Inc. (NMS-US), a wholly owned subsidiary of NMS Group, focused on the discovery and development of oncology drugs and the largest oncological R&D company in Italy, reported that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for its next-generation FLT3 inhibitor NMS-03592088 for the treatment of Acute Myeloid Leukemia (AML) (Press release, Nerviano Medical Sciences, JAN 9, 2024, View Source [SID1234639161]).

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NMS-03592088 is a potent inhibitor of FLT3, KIT and CSF1R that is being assessed as new therapeutic option for AML patients who are relapsed or refractory after treatment with prior standard of care drugs including prior FLT3 inhibitors. Preclinical characterization demonstrated that NMS-03592088 has superior features with respect to approved FLT3 inhibitor drugs supporting its positioning as next generation FLT3 inhibitor. Notably, in addition to the higher biochemical and cellular potency and superior in vivo efficacy, NMS-03592088 showed potent activity in the presence of the gatekeeper resistance mutation F691L, reported as cause of relapse after treatment with first generation FLT3 inhibitors opening the potential for treatment of patients who failed prior FLT3 inhibitor treatment.

The clinical efficacy of NMS-03592088 is being evaluated as monotherapy in a Phase I/II study in Europe and the US. Preliminary evidence of activity was observed during Phase I dose escalation with efficacy demonstrated in patients who had failed prior standard of care including patients who failed prior FLT3 treatments (LINK: AACR (Free AACR Whitepaper)2023). The trial is currently enrolling patients in Phase II with different FLT3 positive AML settings explored (NCT03922100).

"The orphan drug designation for NMS-03592088 is a significant achievement for NMS. It underscores the potential of NMS-03592088 and our dedication to making a difference in the lives of patients with FLT3 AML. We are proud to be at the forefront of innovation and remain steadfast in our mission to address unmet medical needs" said Lisa Mahnke, MD, PhD, CMO of NMS | CEO and Managing Director of NMS-US.

"Receiving orphan drug designation for NMS-03592088 is a critical milestone in our development journey. This recognition not only highlights the scientific merit of our approach but also reinforces our commitment to bringing novel therapies to those who need them most. We look forward to advancing NMS-03592088 through further clinical development and regulatory milestones" said Elena Ardini, MSc, Asset Leader of NMS.

The FDA’s Office of Orphan Products Development grants ODD status to drugs and biologics intended for the safe and effective treatment, diagnosis or prevention of rare diseases or conditions affecting fewer than 200,000 people in the United States. ODD provides benefits to drug developers designed to support the development of drugs and biologics for small patient populations with unmet medical needs. These benefits include potential for market exclusivity for seven years upon FDA approval eligibility for tax credits for qualified clinical trials, and waiver of Prescription Drug User Fee Act Application fee.