Hillhurst Biopharmaceuticals, Inc.’s Post

Spinogenix Announces FDA Clearance of IND Application for SPG302, a Novel Therapy for the Treatment of ALS Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for its Phase 1/2 clinical trial of SPG302 for the treatment of people with Amyotrophic Lateral Sclerosis (ALS). The trial will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of SPG302 dosed as a once-a-day pill in ALS patients. “Having completed the Phase 1 safety study in healthy subjects in Australia, we are thrilled to have gained FDA acceptance of our U.S. IND for SPG302 in ALS,” said Stella Sarraf, Ph.D., Spinogenix Chief Executive Officer and Founder. “SPG302’s unique approach to regenerate synapses offers a fundamentally different treatment modality, focusing on synapse loss which is central to ALS. Current treatments have not sufficiently met the needs of ALS patients, as slowing disease progression alone is not enough. We are committed to advancing SPG302 with the hope of providing a new, transformative therapeutic that can significantly improve the lives of those battling this devastating disease.” SPG302 has been granted U.S. FDA Orphan Drug Designation (ODD) for the treatment of ALS and has received preclinical support from the U.S. National Institutes of Health (NIH) and the Department of Defense (DoD). SPG302 is currently undergoing a Phase 1/2 study in Australia, which has completed dosing of healthy volunteer cohorts, showing dose proportionality, excellent tolerability and plasma levels aligned with efficacy in animal models. Dosing of ALS patients in Australia began in April 2024. Additional information on the trial may be found on ClinicalTrials.gov (NCT05882695). Dr. Merit Cudkowicz, Chair of the Massachusetts General Hospital Department of Neurology, Director, Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, Julieanne Dorn Professor of Neurology at Harvard Medical School, and Spinogenix Advisory Board member added, “ALS is a complex and varied disease, affecting cognitive and motor functions as well as speech and respiration. Spinogenix’s new approach works at the synaptic level to regenerate synapses. This first study in people with ALS is an important step towards determining whether SPG302 helps recover lost functions in motor and cognitive symptom domains.” https://lnkd.in/e3v3CZZA

AstraZeneca and Ionis Pharmaceuticals, Inc.’s Wainzua (eplontersen) has been recommended for approval by the Committee for Medicinal Products for Human Use (CHMP) in the European Union (EU) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN. If approved by the European Commission, Wainzua will be the only approved medicine in the EU for the treatment of ATTRv-PN that can be self-administered monthly via an auto-injector. The CHMP based its opinion on the positive NEURO-TTRansform Phase III trial which showed that through 66 weeks, patients treated with Wainzua demonstrated consistent and sustained benefit on the co-primary endpoints of serum transthyretin (TTR) concentration and neuropathy impairment measured by modified Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint of quality of life (QoL) on the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) versus external placebo. Wainzua continued to demonstrate a favourable safety and tolerability profile throughout the NEURO-TTRansform trial. ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade. Dr. Laura Obici, Head of Rare Diseases Unit, Consultant at the Amyloidosis Research and Treatment Centre Istituto Di Ricovero e Cura a Carattere Scientifico Fondazione I.R.C.C.S. Policlinico San Matteo Pavia, Italy, said: “This debilitating disease is ultimately fatal if left untreated and can have a significant impact on many aspects of patients’ and caregivers' day-to-day lives. Having additional amyloidosis treatment options designed to reduce the production of TTR protein at its source would potentially give patients more time and ability to do what matters most to them and offer the hope of living longer with a higher quality of life." Ruud Dobber, Executive Vice-President, BioPharmaceuticals Business Unit, AstraZeneca, said: “Due to the progressive nature of polyneuropathy of hereditary transthyretin-mediated amyloidosis, it is critical to have timely diagnosis and new therapies to help people have greater control over this potentially fatal disease. Today’s recommendation brings Wainzua one step closer for patients in Europe, and if approved, will offer a new treatment option that can provide consistent TTR suppression and results in improved quality of life.” #rarediseases #polyneuropathy #eplontersen #Wainzua #Ionis #AstraZeneca #WhatScienceCanDo

2025 pharma industry updates 1)- 43rd Annual J.P. Morgan Healthcare Conference starting next week in San Francisco with high expectations for M&As and deals announcements. 2)- Verdiva Bio debuts with $410M Series A to develop GLP-1 obesity drugs. 3)- Biogen wants to acquire remaining Sage Therapeutics for $441.7M; Sage stock jumps 34% post-announcement. 4)- Abbvie records $3.5B impairment from Cerevel Therapeutics buy, after key brain drug failure triggered major stock drop. 5)- Metsera’s monthly GLP-1 injection shows up to 20% weight loss in Phase 2 trial and files for IPO within a year of launch. 6)- HIV capsid-targeting lenacapavir from Gilead Sciences “Discovery of the Year". 7)- Gilead Sciences signed a $1.7B deal with LEO Pharma on STAT6 research to expand in the inflammation disease area. 8)- Aviceda Therapeutics raises $207.5M for AVD-104, a dual-mechanism treatment for geographic atrophy, powering Phase 2b/3 and pivotal trials. 9)- Avian flu H5N1 claims first US victim, a 65-year-old exposed to birds. 10)- Tenvie Therapeutics launches with $200M leveraging Denali Therapeutics’s neuro assets to target proteins linked to neurodegeneration. 11)- Galapagos splits into 2 entities, and cuts 300 jobs. 12)- Novo Holdings leads $200M Series A in Windward Bio for severe respiratory diseases. 13)- Maze Therapeutics files for 2025 biotech IPO after $115M Series D. 14)- Ouro Medicines launches with $120M to develop T cell engager therapies for inflammatory disorders. 15)- IGM Biosciences, Inc. halts imvotamab, IGM-2644, cuts 73% staff; seeking strategic alternatives to preserve cash and value. 16)- Aspect Biosystems secures $115M to advance bioprinted tissue therapeutics. 17)- Orbis Medicines raises €90M to develop oral macrocycle peptide drugs. 18)- Sentynl Therapeutics, Inc.’s Menkes disease drug CUTX-101 gains FDA Priority Review 19)- Johnson & Johnson Rybrevant + Lazcluze combo outperforms AstraZeneca Tagrisso in advanced lung cancer survival. 20)- Hoth Therapeutics, Inc shares up 178% as HT-001 helps skin toxicities in EGFRi-treated cancer patients, showing 100% primary efficacy with no adverse effects. 21)- Candid Therapeutics signs $925M T-cell engager deal with WuXi Biologics, planning first-in-human trials by 2026. 22)- Sana Biotechnology, Inc. shares up 120% marking 1st demonstration of rejection-free cell therapy in type 1 diabetes patients, 23)- WuXi Biologics sells Irish vaccine plant to MSD for $500M. 24)- AZAFAROS B.V. receives orphan designations from FDA and EMA for lead asset nizubaglustat. 25)- GSK is in advanced talks to acquire IDRx for up to $1B. 26)- Novo Nordisk and Valo Health expand collaboration with $4.6B in potential milestones. 27)- MaaT Pharma’s microbiota therapy improves survival in blood-cancer patients with graft-versus-host disease; plans EU filing in mid-2025. 28)- Johnson & Johnson confirms Swiss workforce cuts and reportedly closing Zuchwil site.

SERDA Therapeutics Announces Completion of First Phase 1 Clinical Study of SN514. AMSTERDAM, THE NETHERLANDS, 29 July 2024. SERDA Therapeutics, a biopharmaceutical company focused on developing improved treatments for burns and chronic wounds, today announced completion of its first Phase 1 human clinical study of SN514 hydrogel, an innovative enzymatic wound debriding agent, in healthy adult volunteers. In preclinical studies, SN514 hydrogel showed outstanding efficacy and speed of action both with in vitro substrates and in vivo pig wound models. The first clinical safety question to be addressed was whether healthy skin at the edge of a wound (“periwound skin”) would require any type of protection against the enzyme when applied to the wound surface. The evaluator blinded, randomized study examined the immediate and 21-day cumulative irritation potential of a range of enzyme concentrations formulated into the proprietary hydrogel for off-the-shelf use, as well as the hydrogel vehicle itself. The use of a low irritant control and a positive irritant control allowed calculation of total scores for dermal responses and other effects according to the methodology of Berger and Bowman (Berger and Bowman 1982).  The findings indicate that protection of “periwound” skin is unlikely to be necessary in clinical use. The company plans to present detailed results  in December 2024 at the Innovations in Wound Healing Conference in Islamorada, Florida. SN514 hydrogel is easy to apply. It does not require expert medical professionals and can be self-administered or applied by caregivers. As a result, SN514 hydrogel is expected to reduce the cost-of-care associated with severe burn wounds and chronic ulcers. “We are extremely pleased with the results of this first study, as it really sets the product apart from other enzyme debriders”, said Gábor Heltovics, CEO of SERDA Therapeutics. “We remain confident that SN514 hydrogel will be best-in-class for safe and effective debridement of burn wounds and chronic ulcers.” “These encouraging findings confirm the design of our next planned Phase 1 study, a dose-ranging tolerability trial in deep partial thickness burn wounds”, said Dr. Bert Slade, CMO of SERDA Therapeutics. About SERDA therapeutics SERDA Therapeutics is a leader in developing innovative treatments for severe wound care. With an aging population and increasing diabetes prevalence, effective wound treatment is critical. Traditional debridement methods—surgical, mechanical, and enzymatic—are often painful and inefficient. SERDA’s solution is a hydrogel which offers an enzymatic wound debrider that has the potential to show fast speed of debridement, ease of use, high level of stability, and low-moderate irritation. The terms “SERDA” and “SERDA therapeutics” are used to refer to SERDA bv. For more information about SERDA therapeutics, please visit www.serda-therapeutics.com.

The Journey of Amyloid-Based Therapies Developing therapies to target this amyloid pathway has been challenging. From 2017 to 2019, four BACE1 inhibitors from Merck, AstraZeneca-Eli Lilly, Janssen-Shionogi, and Eisai-Biogen were discontinued in Phase III trials due to poor risk-benefit profiles, despite reducing amyloid-beta levels. Here we learned that targeting amyloid-beta monomers alone wasn’t enough, and broad BACE1 inhibition led to side effects due to BACE1’s other roles. Biogen then launched Aducanumab, the first FDA-approved drug targeting amyloid plaques, receiving accelerated approval in June 2021. However, results from two Phase III trials were mixed: the EMERGE trial showed significant cognitive benefit for early-stage Alzheimer’s patients, while ENGAGE did not. Although the FDA accepted amyloid plaques as a surrogate marker, it requested more data to confirm clinical benefits. Biogen ultimately decided to discontinue Aducanumab in May 2023 for reprioritizing resources. Next came Lecanemab, developed by BioArctic from Professor Lars Lannfelt’s research on the Arctic mutation in Alzheimer’s. Eisai joined in 2005, followed by Biogen in 2014. Lecanemab targets multiple amyloid forms, with a focus on clearing plaques. It received accelerated FDA approval in January 2023, and traditional approval in July 2023. In 2013, Eli Lilly began developing Donanemab, an antibody targeting N3pG—a highly aggregative, resilient form of amyloid. After more than 10 years efforts, it received traditional FDA approval three months ago. While Lecanemab and Donanemab effectively clear amyloid plaques, many plaques are near capillaries. Rapid clearance risks destabilizing these blood vessels, leading to bleeding and swelling (ARIA-E). What we have learned? The therapeutic development journey from amyloid monomers to plaques has shown that targeting monomers alone is insufficient, while protofibrils and plaques show promise. The last piece is amyloid oligomers, the most toxic form of amyloid. Oligomers vary in structure (linear and global), size (2-24 monomers), and diverse epitopes. Acumen Pharmaceuticals is now in Phase II trials focusing on these oligomers, and we may soon learn if targeting them alone could be a breakthrough. One day, if some of us lose my memory of this world, remember—it wasn’t by choice!

Genentech, a member of the Roche Group, announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) for Columvi® (glofitamab-gxbm) in combination with gemcitabine and oxaliplatin (GemOx) for the treatment of people with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who have received at least one prior line of therapy and are not candidates for autologous stem cell transplant. The FDA is expected to make a decision on approval by July 20, 2025.   The standard second-line therapy for R/R DLBCL patients has historically been high-dose chemotherapy followed by stem-cell transplant, however, not all patients are a candidate due to age or coexisting medical conditions. While newer therapies are becoming available, barriers remain for many and alternative treatment options are needed for these patients to improve survival outcomes.   “For people with aggressive lymphomas like DLBCL, timely intervention with effective therapies can be crucial to reduce the risk of disease progression and improve long-term outcomes,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “We are encouraged by the overall survival benefit seen with this Columvi combination and hope it can become an important treatment option for those who are in need of alternative therapies.”   The sBLA is based on results from the Phase III STARGLO study, which were presented at the European Hematology Association Congress earlier this year and recently published in The Lancet. Data showed Columvi in combination with GemOx demonstrated a statistically significant and clinically meaningful overall survival (OS) improvement versus Rituxan® (rituximab) and GemOx (R-GemOx), making it the first CD20xCD3 bispecific antibody to show a survival benefit in DLBCL in a randomized Phase III trial. Safety of the combination appeared consistent with the known safety profiles of the individual medicines.   Data from the STARGLO study have been submitted to other health authorities around the world for approval consideration, including the European Medicines Agency.    Columvi is part of Genentech’s industry-leading CD20xCD3 bispecific antibody program, which has seen more than 3,000 patients treated in clinical trials and more than 2,600 treated in clinical practice to date. Columvi was the first fixed-duration bispecific antibody to receive accelerated approval by the U.S. FDA and conditional marketing authorization in the EU as a monotherapy to treat people with R/R DLBCL after two or more lines of systemic therapy and is currently approved in more than 50 countries around the world. #DLBCL #Columvi #Genentech #Roche

Featured highlights from today's Efortless Update: [Clinical Trials] 07/18 「Teva Pharmaceuticals's phase 3 SPACE Study Finds AJOVY Effective in Preventing Episodic Migraine in Children and Adolescents Over 12 Weeks」 same day : ( ↑ NYSE: TEVA | +2.16% ) 07/18 「Vivoryon Therapeutics N.V. Reports Beneficial Effect on Kidney Function in Diabetes Patients with Varoglutamstat: phase 2b Study Results」 07/18 「BIOSENIC S.A. Completes Post-Hoc Analysis of phase 2 Clinical Trial of Arsenic Trioxide, Reports 75% Overall Response Rate in cGvHD Treatment」 07/18 「Paratek Pharmaceuticals Announces phase 3 Results: Omadacycline Meets All FDA Endpoints in CABP Treatment, Similar Adverse Effects as Moxifloxacin」 07/17 「Genentech's Vabysmo Maintains Vision Improvements in 80% of DME Patients for Four Years in RHONE-X Study」 [Deals] 07/18 「Lupin Limited to Market Huons Co., Ltd.'s Cyclosporine Ophthalmic Nanoemulsion in Mexico for Treating Dry Eye Syndrome and Corneal Ulcers」 07/18 「Exscientia Acquires Full Control of CDK7 Inhibitor Programme from GT Apeiron for $20M and Royalty Commitments」 same day : ( ↓ NASDAQ: EXAI | -0.56% ) 07/18 「Haystack Oncology and Lisata Therapeutics, Inc. Collaborate to Utilize MRD Technology in Certepetide Study for Metastatic Pancreatic Cancer」 same day : ( ↓ NASDAQ: LSTA | -4.1% ) 07/17 「CytoMed Therapeutics Limited acquires Cellsafe International assets for $490K aiming to expand therapeutic use of cord blood units」 07/17 「OPKO Health, Inc. secures $250M non-dilutive note purchase deal with HealthCare Royalty, backed by Pfizer's payments for NGENLA treatment」 same day : ( ↓ NASDAQ: OPK | -1.39% ) [Regulatory] 07/18 「Everest Medicines' drug NEFECON gets full approval application acceptance by China's National Medical Products Administration」 07/18 「FDA grants second Fast Track designation to Diamyd Medical for type 1 diabetes treatment in pediatric patients with HLA DR3-DQ2 genotype」 07/18 「ONO PHARMACEUTICAL CO., LTD. receives additional approval for Opdivo in combination therapy for advanced urothelial carcinoma in South Korea」 07/18 「Marinus Pharmaceuticals gets Chinese approval for ganaxolone oral suspension to treat epileptic seizures in CDD patients following phase 3 trial results」 same day : ( ↑ NASDAQ: MRNS | +0.72% ) 07/18 「FDA approves Phathom Pharmaceuticals' VOQUEZNA for Non-Erosive GERD in adults based on PHALCON-NERD-301 study results」 same day : ( ↓ NASDAQ: PHAT | -3.22% ) 07/17 「Shanghai Junshi Biosciences Co Ltd seeks approval for toripalimab and bevacizumab combination as a first-line treatment for advanced HCC based on HEPATORCH study results」 View the full newsletter contents for free via the link in the comments.

In the past year, the market values of the top 10 BioPharma companies increased by approximately 24%, reaching $3.5 trillion, while the broader S&P 500 rose by around 30%. Eli Lilly and Novo Nordisk: These companies experienced significant market cap increases (62% and 37% YoY, respectively) due to the expanding indications for their GLP-1s and increasing supply. AbbVie: AbbVie's 27% YoY market cap gain was attributed to the strong performance of its immunology drugs, Skyrizi and Rinvoq, and the acquisition of ImmunoGen's ovarian cancer treatment, Elahere. Elahere's approval and potential peak sales exceeding $2 billion position AbbVie for further growth. Amgen: Amgen's performance was driven by excitement surrounding its weight loss medicine MariTide, the acquisition of Horizon Therapeutics, and strong sales of its bone-building drug Evenity and cholesterol-lowering medication Repatha. The BioPharma industry's growth demonstrates the sector's resilience and potential. The success of companies like Eli Lilly, Novo Nordisk, AbbVie, and Amgen highlights the importance of innovative therapies, strategic acquisitions, and expanding indications for existing drugs. As valuations become more favorable, larger BioPharma companies may be more inclined to engage in partnerships and M&A deals, further driving growth and innovation in the sector. This trend could lead to increased investment in research and development, ultimately benefiting patients by providing access to novel and improved treatments. How’s your idea about this trend? Please kindly share your opinion for discussion. :D Vincent

📈𝘽𝙞𝙤𝙥𝙝𝙖𝙧𝙢𝙖 𝘽𝙪𝙡𝙡𝙚𝙩𝙞𝙣: 𝙎𝙩𝙖𝙮 𝘼𝙝𝙚𝙖𝙙 𝙬𝙞𝙩𝙝 𝙏𝙝𝙚𝙨𝙚 5 𝙍𝙚𝙖𝙙𝙨 📚 📌 PTC Therapeutics, Inc.'s Huntington’s disease candidate, PTC518, is no longer under an FDA partial clinical hold after positive interim results from a Phase II study. The PIVOT-HD trial showed that PTC518 significantly lowered mutant huntingtin (mHTT) protein levels in both blood and cerebrospinal fluid (CSF). Additionally, there were “favorable trends” on several HD clinical assessments, including Total Motor Score and Composite Unified Huntington's Disease Rating Scale. The candidate was also found to be safe and well-tolerated after 12 months of treatment. 📌 Altimmune, Inc. has shared a full Phase 2 analysis of its "double G" drug pemvidutide, showing impressive results in preserving lean mass and regulating lipids. In the 48-week MOMENTUM trial, 21.9% of the total weight loss was lean mass, improving from the 25.5% reported in the partial analysis from March. Additionally, 78.1% of the weight loss was from fat, up from 74.5%. These findings come from 50 patients who underwent a full MRI analysis, out of the 391 participants in the trial. 📌 Alnylam Pharmaceuticals' RNA silencing treatment has shown remarkable results in a highly anticipated clinical trial. The therapy reduced the risk of death, heart-related hospital visits and hospitalizations by 28% compared to placebo in patients with heart muscle disease. These promising results position vutrisiran to become the first RNA silencing therapy for ATTR amyloidosis with cardiomyopathy, a condition where misfolded proteins build up in the heart, potentially leading to heart failure. 📌 Tandem Diabetes Care (Nasdaq: TNDM) has shared new real-world insights on Tandem Mobi user satisfaction and wearability at the 84th Scientific Sessions of the American Diabetes Association (ADA) in Orlando, Florida. The survey revealed that 86% of participants were satisfied or very satisfied with Tandem Mobi, regardless of prior therapy, and agreed that it helps improve their quality of life. Tandem Mobi, the world’s smallest, durable automated insulin delivery (AID) system, became commercially available in the United States in February 2024. 📌 ANI Pharmaceuticals, Inc. has signed a significant deal to acquire ophthalmology specialist Alimera Sciences and its two approved commercial drugs, Iluvien and Yutiq. For $5.50 per share and a contingent value right (CVR) worth up to $0.50 per share, ANI will purchase Alimera. The CVR will be payable to Alimera's investors if the company meets specific revenue targets in 2026 and 2027. #biotechnews #biotech #biopharma #medicaldevice

<!-- Content start --> Treatment for: Neurofibromatosis SpringWorks Therapeutics Completes Submission of New Drug Application to the FDA for Mirdametinib for the Treatment of Children and Adults with NF1-PN STAMFORD, Conn., July 01, 2024 (GLOBE NEWSWIRE) — SpringWorks Therapeutics, Inc. (Nasdaq: SWTX), a commercial-stage biopharmaceutical company focused on severe rare diseases and cancer, announced today that the Company has completed the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for mirdametinib, an investigational MEK inhibitor, for the treatment of pediatric and adult patients with neurofibromatosis type 1- associated plexiform neurofibromas (NF1-PN). “We are pleased to be one step closer towards our goal of bringing mirdametinib to patients with NF1-PN in the U.S. and believe that our ReNeu data support the potential for mirdametinib to be a differentiated and best-in-class therapy for both children and adults living with this devastating disease,” said Saqib Islam, Chief Executive Officer of SpringWorks. “We look forward to working closely with the FDA throughout the review process and also plan to file for regulatory approval in the European Union later this year.” The NDA submission includes data from the pivotal Phase 2b ReNeu trial, which evaluated mirdametinib in patients ≥ 2 years of age with NF1-associated PN causing significant morbidity. Results were presented in an oral presentation at the 2024 American Society of Clinical Oncology Annual Meeting and demonstrated that mirdametinib treatment resulted in significant objective response rates confirmed by blinded independent central review, deep and durable responses, improvement in pain and health-related quality of life as well as a manageable safety profile across both the adult and pediatric cohorts.1 The FDA and the European Commission have granted Orphan Drug designation for mirdametinib for the treatment of NF1. The FDA has also granted Fast Track designation for the treatment of patients ≥ 2 years of age with NF1-PN that are progressing or causing significant morbidity and Rare Pediatric Disease designation for the treatment of NF1. In the second half of 2024, SpringWorks also plans to file a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for mirdametinib for the treatment of children and adults with NF1-PN. About the ReNeu Trial ReNeu (NCT03962543) is an ongoing, multi-center, open-label Phase 2b trial evaluating the efficacy, safety, and tolerability of mirdametinib in patients ≥ 2 years of age with an inoperable NF1-associated PN causing significant morbidity. The study enrolled 114 patients to receive mirdametinib at a dose of 2 mg/m2 twice daily (maximum dose of 4 mg twice daily) without regard to food. Mirdametinib was administered orally in a 3-week on, 1-week off dosing schedule as either a capsule or dispersible tablet. The primary endpoint is confirmed objective