Nov 04, 2024
Journey Medical’s Emrosi, a small-molecule treatment for rosacea, receives FDA approval. (Source)
Viking Therapeutics announces positive Phase 1 results for its novel small-molecule obesity treatment. (Source)
Formosa Pharmaceuticals reports positive Phase 3 trial results for APP13007, a small molecule drug designed to reduce inflammation and pain after cataract surgery in Chinese patients. (Source)
Lomond Therapeutics, a biotech company focused on blood cancer treatments, has merged with Venetian-1 Acquisition Corp. and raised $44 million in funding. (Source)
Relief Therapeutics and Renexxion to merge and go public, focusing on small molecule treatments for gastrointestinal mobility disorders like gastroparesis and delayed gastric emptying. (Source)
Nov 03, 2024
CinRx’s obesity treatments, CIN-109 and CIN-110, showed promising results in Phase 1 trials. (Source)
Nov 01, 2024
Disc Medicine announces the successful completion of its Phase 2 FDA meeting for bitopertin, a potential treatment for erythropoietic protoporphyria. (Source)
Ningdan New Drug Raises Significant Funds in Series C Financing Round. (Source)
Oct 31, 2024
Biofrontera‘s small molecule, Ameluz, shows promise in treating basal cell carcinoma with photodynamic therapy. Successful Phase 3 trial results have led to a significant stock surge. (Source)
Bristol Myers Squibb’s Phase 3 clinical trial of Cobenfy demonstrates promising 52-week results for the treatment of schizophrenia. (Source)
Supernus Pharmaceuticals‘ SPN-820 Delivers Encouraging Phase 2a Results for Major Depressive Disorder. (Source)
Eupraxia Pharmaceuticals raises $44.5M for small molecule drug development in eosinophilic esophagitis, knee osteoarthritis, and microsphere-based delivery. (Source)
Formosa Pharmaceuticals partners with DÁVI Farmacêutica to bring APP13007, a small molecule treatment for post-ocular surgery inflammation and pain, to the Portuguese market. (Source)
Hutchmed receives a $20 million milestone payment from AstraZeneca, triggered by Fruzaqla, a small molecule colorectal cancer drug, achieving $200 million in sales. (Source)
Mochida Pharmaceutical secures exclusive Japanese rights to Tyvaso DPI, a small molecule inhalation therapy for pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD), from United Therapeutics. (Source)
SPC Korea secures South Korean rights to Lipocine’s Tlando, a small-molecule testosterone replacement therapy. (Source)
Ventus Therapeutics’ VENT-03, a small molecule targeting systemic lupus erythematosus (SLE), has successfully completed Phase 1 trials and is now advancing to Phase 2. (Source)
Recce Pharmaceuticals receives positive safety review for R327G Phase 2 trial. Small molecule, skin infections, diabetic foot infections (Source)
Anavex Life Sciences‘ experimental Alzheimer’s treatment, blarcamesine, has met its primary endpoint in a Phase 2b/3 clinical trial. (Source)
Madrigal Pharmaceuticals’ Rezdiffra sees strong Q3 sales. Small molecule treatment for metabolic dysfunction-associated steatohepatitis (MASH). (Source)
Evommune Secures $115M for Phase 2 Clinical Trial of Novel Small Molecule Treatment for Chronic Hives and Eczema. (Source)
Avadel Pharmaceuticals secures victory over Jazz Pharmaceuticals. A US District Court judge upheld the FDA’s decision to approve Lumryz, Avadel’s competitor to Jazz’s Xywav, for treating cataplexy and excessive daytime sleepiness. (Source)
Oct 30, 2024
CalciMedica‘s small molecule drug, Auxora, shows promise in treating acute pancreatitis based on positive Phase 2b trial data. (Source)
Amplia Therapeutics reports promising Phase 2a data for narmafotinib in pancreatic cancer. (Source)
reMYND reports positive Phase 2a data for REM0046127 in Alzheimer’s disease. (Source)
Axonis Raises $115M Series A to Develop Small Molecule Therapeutics for Neurological Disorders, Including Pain and Epilepsy. (Source)
TXN Systems raises SEK25M ($2.3M) in Series A funding to develop novel small-molecule antibiotics targeting multi-drug resistant urinary tract infections (UTIs). (Source)
Septerna, Inc. a biotech company focused on GPCR oral small molecule drug discovery, completed its IPO, raising $331.2 million. (Source)
Shorla Oncology’s Jylamvo (methotrexate) gains FDA approval for expanded use in pediatric patients with acute lymphoblastic leukemia (ALL) and polyarticular juvenile idiopathic arthritis (pJIA). (Source)
Oct 29, 2024
Biogen inks $1.45B deal with Neomorph to access its molecular glue degrader platform for developing treatments for Alzheimer’s, autoimmune diseases, and other conditions. (Source)
Soal Therapeutics‘ small molecule drug candidate, SL-1002, demonstrates positive results in a Phase 2 trial for limb spasticity. (Source)
Cognition Therapeutics Releases Phase 2 Data for CT1812 in Alzheimer’s Disease. (Source)
Novartis Scemblix® approved by the FDA for newly diagnosed CML, offering superior efficacy and safety. (Source)
Shionogi Therapeutics‘ Phase 3 trial demonstrates the potential of Xocova, a small molecule drug, to prevent COVID-19 infection after exposure. (Source)
Zai Lab’s small-molecule drug KarXT demonstrates positive results in Phase 3 clinical trials for schizophrenia. (Source)
EyePoint Pharmaceuticals upsizes IPO to raise $140M for its small-molecule, sustained-release drug-device combo targeting diabetic macular edema. (Source)
Dr. Falk Pharma expands its therapeutic focus to include acute pancreatitis through the acquisition of Kynos Therapeutics. (Source)
Oct 28, 2024
Vensica Medical raises $11M to develop a needle-free medical device for overactive bladder treatment. (Source)
EyePoint Pharmaceuticals Shares Positive 16-Week Results from the Ongoing Phase 2 VERONA Trial Evaluating DURAVYU™ for Diabetic Macular Edema. (Source)
Odyssey Therapeutics and Terray Therapeutics Announce Strategic Partnership to Identify and Advance Innovative Therapies Targeting Transcription Factors. Small molecule, drug discovery, drug development (Source)
Oct 27, 2024
Novartis’s oral Fabhalta® (iptacopan) showed sustained efficacy in a Phase III C3G trial for kidney disease. (Source)
CORXEL and LENZ Release Positive Topline Results from Phase 3 Clinical Trial of LNZ100 for Presbyopia in China. (Source)
Oct 26, 2024
Aardvark Therapeutics acquires MEI Pharma’s ME-344 asset for $500k upfront and $62 million in potential milestone payments. (Source)
Apellis Pharmaceuticals and Sobi announced positive Phase 3 VALIANT study results at ASN Kidney Week. The study highlighted the effectiveness of systemic pegcetacoplan treatment for patients with C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), two rare and severe kidney diseases. (Source)
Oct 25, 2024
Iterum Therapeutics announces FDA approval of ORLYNVAH™ (oral sulopenem) for the treatment of uncomplicated urinary tract infections. (Source)
Bristol Myers Squibb and Ideaya Bioscience Announce Promising Interim Phase 1 Clinical Trial Results for IDE397, a Novel Small Molecule Inhibitor, in Patients with Urothelial and Lung Cancer. (Source)
Revolution Medicines presents encouraging initial Phase 1/1b clinical trial data for RMC-9805, a small molecule drug candidate targeting pancreatic ductal adenocarcinoma. (Source)
Agomab Therapeutics NV Raises $89 Million in Series D Funding to Fuel Development of Broad Fibrosis Therapies. (Source)
Oct 24, 2024
Dermavant Biosciences announces promising Phase 3 results for Vtama, a novel small-molecule topical treatment for atopic dermatitis. (Source)
NTC licenses its innovative colonoscopy prep drug, NTC015, to Chinoin for Mexico and Latin America. (Source)
Arcus Biosciences’ HIF-2a inhibitor, casdatifan, showed promising results in treating metastatic kidney cancer, including tumor shrinkage. (Source)
Monopar Therapeutics has partnered with Alexion, AstraZeneca Rare Disease to develop ALXN-1840, a Phase 3 drug candidate for Wilson disease.
Duhn Therapeutics SAS, backed by Landmark BioVentures and OM Pharma SA, has licensed OM-174 (now DHN198) to develop and commercialize lipid-based cancer immunotherapies. (Source)
Shenyang Sunshine (3SBio) acquires China and Hong Kong rights for RMX Biopharma’s (Haihe Biopharma) oral Paclitaxel solution, a small molecule cancer treatment. (Source)
Tyra Biosciences reports promising Phase 1/2 results for TYRA-300 in treating urothelial carcinoma, despite some reported side effects. (Source)
AbbVie and Gedeon Richter Plc. have partnered to discover and develop new treatments for neuropsychiatric conditions, building on their successful history of collaboration on CNS projects like cariprazine and ABBV-932. (Source)
Oct 23, 2024
PharmaTher has received a Complete Response Letter from the FDA pertaining to its New Drug Application for Ketarx, a racemic ketamine formulation. (Source)
Lumos Pharma to go private in merger with Double Point Ventures. The company focuses on developing small molecule treatments for pediatric growth hormone deficiency. (Source)
Merck acquires Modifi Biosciences for $30M upfront and up to $1.3B in milestone payments. Small molecule, glioblastoma, cancer (Source)
PMV Pharmaceuticals collaborates with MD Anderson Cancer Center and Memorial Sloan Kettering Cancer Center to initiate new Phase 1b clinical trials evaluating rezatapopt, a novel small molecule drug, in patients with myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML). (Source)
Eccogene Receives $60M Milestone Payment from AstraZeneca for Initiating Phase 2b Trial of ECC5004/AZD5004 for the Treatment of Obesity and Type 2 Diabetes. (Source)
Alkeus Pharmaceuticals reports “significant trend” in reducing GA lesions with gildeuretinol, a small molecule for geographic atrophy. (Source)
Zhaoke Ophthalmology’s myopia drug NVK002 shows positive results in a two-year Phase III trial. (Source)
Revolution Medicines Shares New Findings from RMC-6236 Monotherapy Trial in Advanced Pancreatic Ductal Adenocarcinoma. (Source)
Alyssum Therapeutics secures $26M Series A to advance AT-1965, a small molecule targeting “immunologically cold tumors”. (Source)
Oct 22, 2024
Hansoh Pharma Reports Positive Phase III Trial Results for 阿美乐® Ameile (Aumolertinib) in Combination with Chemotherapy for EGFR-Mutated Advanced Lung Cancer. (Source)
RaQualia Pharma‘s partner, Shandong Luoxin Pharmaceutical Group, secures Chinese NMPA approval for its small molecule drug to treat H. pylori infections. (Source)
RedHill and a leading U.S. academic medical center collaborate to explore Opaganib as a potential treatment for phosgene inhalation injury. (Source)
Rectify Pharma announces RTY-694 as a breakthrough functional modulator for primary sclerosing cholangitis, moving into development. (Source)
Oct 21, 2024
Angelini Pharma obtains a license for Cureverse‘s early-stage drug candidate, CV-01. It is a novel compound currently in Phase 1 clinical trials with potential applications across various brain health disorders. Small molecule, epilepsy, Alzheimer’s disease (Source)
Novo Nordisk‘s oral semaglutide lowered major cardiovascular event risk by 14% in type 2 diabetes patients (SOUL trial). (Source)
Bright Minds Biosciences and Firefly Neuroscience will jointly analyze EEG data from the BREAKTHROUGH Phase 2 trial, investigating BMB-101 for absence epilepsy and developmental epileptic encephalopathy. (Source)
Seaport Therapeutics raises $225 million in Series B. Small molecule, major depressive disorder, generalized anxiety disorder (Source)
Oct 20, 2024
Foresee Pharmaceuticals announces positive Phase 1 results for Linvemastat. Small molecule, autoimmune, asthma, chronic obstructive pulmonary disease (COPD) (Source)
Oct 19, 2024
Nurix Therapeutics announced positive interim data from its ongoing phase 1/2 clinical trial evaluating NX-5948, a BTK degrader, in patients with relapsed or refractory Waldenström’s macroglobulinemia. (Source)
Gilead and Merck’s lenacapavir/islatravir combination showed positive Phase 2 results. Small molecule, human immunodeficiency virus (HIV) infection (Source)
Oct 18, 2024
Vertex Pharmaceuticals will present Phase 3 data on suzetrigine, a potential first-in-class, highly selective pain signal inhibitor, at the upcoming American Society of Anesthesiologists Annual Meeting. (Source)
European regulators endorse Novartis’s Kisqali® as a treatment to lower the risk of breast cancer relapse in patients with hormone-receptor-positive, HER2-negative disease. Small molecule, breast cancer (Source)
Bright Minds Biosciences Secures $35 Million in Private Equity. Small molecule, refractory epilepsy, depression (Source)
Lykos Therapeutics is optimistic after reaching an agreement with the FDA on a path forward, including an additional Phase 3 trial. Small molecule, post-traumatic stress disorder, MDMA, psychedelic (Source)
Oct 17, 2024
AbbVie’s VYALEV™ (foscarbidopa and foslevodopa) has received FDA approval for treating motor fluctuations in adults with advanced Parkinson’s disease. It’s the first subcutaneous 24-hour levodopa-based therapy of its kind. (Source)
Avadel Pharmaceuticals’ LUMRYZ™ extended-release oral suspension (CIII) has received FDA approval for treating cataplexy or excessive daytime sleepiness in patients with narcolepsy aged 7 years and older. (Source)
Novartis pays $70 million upfront and up to $1.1 billion in milestone payments for exclusive rights to Baiyu Pharmaceutical’s anti-tumor drug. (Source)
Viatris acquires rights to Inpefa from Lexicon Pharmaceuticals for $25M upfront and $200M milestones. Small molecule, heart failure, type 2 diabetes (Source)
Orasis Pharmaceuticals has granted Optus Pharmaceuticals exclusive rights to commercialize Qlosi™ (pilocarpine hydrochloride ophthalmic solution) 0.4% in Korea. presbyopia (Source)
Noema Pharma‘s NOE-105 (gemlapodect) meets primary and secondary endpoints in Phase 2a Tourette syndrome study. (Source)
Terray Therapeutics Secures $120M in Oversubscribed Series B Funding to Accelerate the Development of Its Generative AI-Designed Small Molecule Therapeutics Toward Clinical Trials. Small molecule, drug development, AI (Source)
Marinus Pharmaceuticals Highlights Refractory Status Epilepticus Clinical Data from the RAISE Trial at the Neurocritical Care Society Annual Meeting. (Source)
AgeneBio’s Phase 2B trial demonstrates that AGB101 can reduce the rate of brain shrinkage in individuals with mild cognitive impairment caused by Alzheimer’s disease who do not carry the APOE-4 gene variant. Small molecule, Alzheimer’s Disease (Source)
SpyGlass Pharma‘s drug delivery platform shows promise in glaucoma treatment after cataract surgery. Small molecule, glaucoma, ocular hypertension, drug delivery (Source)
Supernus Reports Encouraging Results from Open-Label Phase 2a Study of SPN-820 for Major Depressive Disorder. Small molecule, depression (Source)
Verastem Oncology Highlights Avutometinib and Defactinib Combination Success in Recurrent Ovarian Cancer at IGCS 2024. (Source)
Oct 16, 2024
GSK and ViiV Healthcare announce 99% effectiveness of Apretude for HIV prevention. (Source)
Athos‘ AI-generated G9A inhibitor, ATH-063, shows promising results in Phase 1 trials, selectively boosting anti-inflammatory T cells. Inflammatory bowel disease, AI (Source)
Neurogastrx announces promising preclinical data (Positive Proof-of-Concept Data) for NG101, a potential new treatment to alleviate nausea and vomiting caused by GLP-1 agonist medications. (Source)
HUTCHMED has announced that TAGRISSO® in combination with ORPATHYS® showed a high, clinically significant response rate in lung cancer patients with high levels of MET overexpression and/or amplification in the SAVANNAH Phase II trial. (Source)
TenNor Therapeutics, a Suzhou-based clinical-stage biotech, secured a significant funding boost with the closing of its Series E round, raising over $41 million. Small molecule, bacterial infections, metabolic disease (Source)
Oct 15, 2024
Novartis uses priority review for Kisqali supplement NDA. breast cancer, priority review voucher (Source)
Ocean Biomedical has entered into an exclusive licensing agreement with Molecure to develop and commercialize selective YKL-40 inhibitors. Small molecule, cancer, inflammatory disease (Source)
Thryv Therapeutics‘ Wave I Part 2 clinical trial for long QT syndrome Long QT Syndrome Type 2 and Type 3 Patients has yielded positive top-line data. Small molecule, congenital Long QT Syndrome (Source)
Jazz Pharmaceuticals reports positive results for Zepzelca® and atezolizumab in treating Extensive-Stage Small Cell Lung Cancer. (Source)
Johnson & Johnson (J&J) has discontinued several clinical trials, including a Phase II study of seltorexant in patients with probable Alzheimer’s disease. (Source)
Monument Therapeutics receives £1M ($1.3M) funding from Forster Foundation to advance its schizophrenia treatment development. Small molecule, schizophrenia (Source)
Oct 14, 2024
Inventiva receives €348 million to fund the development of its NATiV3 drug in a Phase 3 clinical trial. (Source)
Lundbeck is set to acquire Longboard Pharmaceuticals in a strategic move that will bolster its neuroscience pipeline. Small molecule, Dravet syndrome, Lennox-Gastaut syndrome (Source)
Exelixis and Merck have partnered to investigate the potential of Zanzalintinib in combination with KEYTRUDA for head and neck cancer and with WELIREG for renal cell carcinoma. (Source)
Oct 11, 2024
Stealth BioTherapeutics Receives Positive FDA Advisory Committee Backing for Elamipretide in Barth Syndrome. (Source)
Denali-Sanofi Terminates Phase IIb Trial for Multiple Sclerosis Drug, Oditrasertib. (Source)
Gilead has decided to terminate its $15 million research collaboration with Yuhan on the MASH drug candidate after reviewing preclinical data. (Source)
Oct 10, 2024
Small molecule drug Itovebi from Roche (Genentech, Inc.) receives FDA approval for use in a triple-combination treatment regimen for breast cancer. (Source)
Pfizer’s Talzenna and Xtandi combination therapy achieves positive phase III results in expanding its treatment options for prostate cancer. (Source)
Booster Therapeutics Secures $15M to Develop Small Molecule Treatments for Parkinson’s and Alzheimer’s. (Source)
AI-Powered Drug Discovery Alliance Between Eurofins and Dompé Enters the Second Year. Small molecule, drug discovery, AI. (Source)
Oct 09, 2024
Vanqua Bio’s VQ-101, a small molecule drug for Parkinson’s and related disorders, demonstrates positive safety data in Phase 1 studies. (Source)
About Small Molecules
Small molecules are organic compounds with relatively low molecular weights that interact with biological targets, such as proteins, to exert therapeutic effects. They play a crucial role in drug discovery and development, particularly in the pharmaceutical industry.
Key Characteristics of Small Molecules:
- Low molecular weight: Typically less than 1,000 Da.
- Organic compounds: Composed of carbon and hydrogen, often with additional elements like oxygen, nitrogen, sulfur, and phosphorus.
- Target interaction: Bind to specific biological targets, such as enzymes, receptors, or nucleic acids, to modulate their function.
- Therapeutic effects: Can produce a variety of therapeutic effects, including:
- Disease modification: Treating or preventing diseases.
- Symptom relief: Alleviating symptoms of illnesses.
- Prophylactic use: Preventing diseases or infections.
Advantages of Small Molecules:
- Oral administration: Many small molecules can be administered orally, making them convenient for patients.
- High bioavailability: They often have good absorption and distribution in the body.
- Well-established drug development pathways: There are well-developed processes for discovering, developing, and manufacturing small molecule drugs.
- Diverse therapeutic applications: Small molecules can target a wide range of biological targets and treat various diseases.
Examples of Small Molecule Drugs:
- Aspirin: A nonsteroidal anti-inflammatory drug (NSAID) used to treat pain, fever, and inflammation.
- Paracetamol (acetaminophen): An analgesic and antipyretic used to relieve pain and fever.
- Statins: Drugs used to lower cholesterol levels.
- Insulin: A hormone used to treat diabetes.
Challenges in Small Molecule Drug Discovery:
- Off-target effects: Small molecules can sometimes interact with unintended targets, leading to side effects.
- Drug resistance: Pathogens and cancer cells can develop resistance to small molecule drugs.
- Complexity of biological targets: Many biological targets are complex and difficult to understand, making drug discovery challenging.
Despite these challenges, small molecules remain a cornerstone of drug discovery and development. Advances in medicinal chemistry, computational biology, and high-throughput screening have facilitated the discovery of novel small molecule drugs for a wide range of diseases.
