News
Key Takeaways:
  • Amgen announced that the U.S. Food and Drug Administration (FDA) has approved IMDELLTRA™ (tarlatamab-dlle) for the treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after platinum-based chemotherapy.
  • IMDELLTRA has received accelerated approval based on the encouraging response rate and duration of response (DoR) observed in clinical studies.
Key Takeaways:
  • Rila Therapeutics, a biotechnology company focused on addressing the burden of chronic fibrotic diseases, announced that the first cohort of subjects was dosed in a phase 1 trial of RLA-23174 in China in collaboration with it's development partner, Yingli Pharmaceutical. RLA-23174 is a first-in-class small molecule allosteric inhibitor of HIPK2 which inhibits TGF-β signaling to address FSGS and other fibrotic diseases.
Key Takeaways:
  • Verismo Therapeutics, a clinical-stage CAR T company developing the novel KIR-CAR platform technology, announced that it has received a notification from the US Food and Drug Administration (FDA) that the Phase 1 clinical trial of SynKIR™-310 for the investigation of NHL may proceed.
  • The Phase 1 trial, named CELESTIAL-301, will assess safety, tolerability, and preliminary efficacy of SynKIR™-310 in patients with relapsed/refractory (r/r) B-cell Non-Hodgkin Lymphomas (B-cell NHL), including Diffuse Large B Cell lymphoma (DLBCL), Follicular Lymphoma (FL), Mantle Cell Lymphoma (MCL), and Marginal Zone Lymphoma (MZL). 
Key Takeaways:
  • Sanofi and Novavax announce co-exclusive licensing agreement to co-commercialize COVID-19 vaccine and develop novel flu-COVID-19 combination vaccines
  • Agreement provides patients with broader access to a protein-based non-mRNA adjuvanted COVID-19 vaccine through combined commercial strength, from 2025 onwards
  • Accelerates potential for development of a novel flu-COVID-19 combination product based on authorized vaccines with demonstrated efficacy and tolerability, potentially offering patients enhanced convenience and protection


Key Takeaways:
  • Bluejay Therapeutics, a leader in the development of novel therapeutics, announced the successful closure of a $182 million Series C financing round.
  • This capital infusion will accelerate the clinical development of BJT-778, as the treatment for chronic hepatitis D (HDV).
  • The funds will also support the progression of additional promising candidates in Bluejay’s robust pipeline for the treatment for chronic hepatitis B.
Key Takeaways:
  • Fate Therapeutics, a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases, announced that the first patient with systemic lupus erythematosus (SLE) has been treated in the Phase 1 autoimmunity study of FT819, the Company’s off-the-shelf, CD19-targeted chimeric antigen receptor (CAR) T-cell program.
Key Takeaways:
  • bluebird bio, announced that it has completed the first commercial cell collection for LYFGENIA (lovotibeglogene autotemcel), a one-time gene therapy for the treatment of sickle cell disease in patients with a history of vaso-occlusive events.
  • LYFGENIA was approved by the FDA in December 2023 and is the most deeply studied gene therapy for sickle cell disease, with the longest follow-up in the field.
Key Takeaways:
  • Phanes Therapeutics, a clinical stage biotech company focused on innovative drug discovery and development in oncology, announced that it has entered into a clinical supply agreement with Roche to study PT217, its first-in-class bispecific antibody targeting DLL3 and CD47, in combination with Roche’s anti-PD-L1 therapy, atezolizumab, in patients with small cell lung cancer (SCLC), large cell neuroendocrine carcinoma of the lung (LCNEC) and extrapulmonary neuroendocrine carcinomas (EP-NECs).
Key Takeaways:
  • VYNE Therapeutics, a clinical-stage biopharmaceutical company developing proprietary, innovative and differentiated therapies for the treatment of immuno-inflammatory conditions, announced the clearance of its Investigational New Drug application (“IND”) by the U.S. Food and Drug Administration for VYN202, an oral BD2-selective BET inhibitor.
  • VYNE plans to initiate a first-in-human Phase 1a single ascending dose/multiple ascending dose (“SAD/MAD”) trial in healthy volunteers this quarter and expects to report top line results from the SAD/MAD trial in the second half of this year.
Key Takeaways:
  • Lyra Therapeutics, announced topline results from the Company’s Phase 3 ENLIGHTEN 1 trial evaluating LYR-210 for the treatment of chronic rhinosinusitis (CRS). ENLIGHTEN 1 did not meet its primary endpoint of demonstrating statistically significant improvement compared to sham control in the composite score of the three cardinal symptoms (3CS) of CRS (nasal obstruction, nasal discharge, facial pain/pressure) at 24 weeks.
  • ENLIGHTEN 1 is one of two Phase 3 clinical trials evaluating LYR-210, a bioabsorbable sinonasal implant (7500µg mometasone furoate), as a six-month treatment for chronic rhinosinusitis (CRS).
Key Takeaways:
  • Edgewise Therapeutics, a leading muscle disease biopharmaceutical company, announced the dosing of the first patient in the Phase 2 CIRRUS-HCM trial of EDG-7500.
  • EDG-7500 is a novel oral, selective, cardiac sarcomere modulator, specifically designed to slow early contraction velocity and address impaired cardiac relaxation associated with HCM and other diseases of diastolic dysfunction.
Key Takeaways:
  • X4 Pharmaceuticals, a company driven to improve the lives of people with rare diseases of the immune system, announced that the U.S. Food and Drug Administration (FDA) has approved XOLREMDI™ (mavorixafor) capsules for use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.
Key Takeaways:
  • ADC Therapeutics announced that initial data from an investigator-initiated Phase 2 clinical trial evaluating ZYNLONTA® (loncastuximab tesirine-lpyl) demonstrated a high response rate in patients with relapsed/refractory (r/r) marginal zone lymphoma (MZL).
Key Takeaways:
  • Lantern Pharma, a leading artificial intelligence (AI) oncology drug discovery and development company, announced a strategic AI-driven collaboration with French biotechnology company, Oregon Therapeutics to optimize the development of its first-in-class protein disulfide isomerase (PDI)(1) inhibitor drug candidate XCE853 in novel and targeted cancer indications
  • Lantern will be leveraging its proprietary RADR® AI platform to uncover biomarkers and efficacy-associated signatures of XCE853 across solid tumors that can aid in precision development.
Key Takeaways:
  •  Regulus Therapeutics, a biopharmaceutical company focused on the discovery and development of innovative medicines targeting microRNAs announced, after review of all available safety data, the Company initiated screening in the of the fourth cohort of patients in the Phase 1b MAD study of RGSL8429 for the treatment of ADPKD.
  • Patients in the fourth cohort will receive an open label fixed dose of 300 mg of RGLS8429 every other week for three months.
Key Takeaways:
  • Astellas Pharma and Poseida Therapeutics announced that Xyphos Biosciences, Inc., (a wholly owned subsidiary of Astellas, "Xyphos") and Poseida have entered into a research collaboration and license agreement to develop novel convertibleCAR® programs by combining the innovative cell therapy platforms from each of the companies.
Key Takeaways:
  • Kazia Therapeutics, a biotechnology company specialising in oncology, announced that the Safety Review Team (SRT) of the EVT801 Phase 1 clinical trial has concluded that the primary and secondary objectives of stage 1 of the trial have successfully been met.
Key Takeaways:
  • Soleno Therapeutics, a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to diazoxide choline for the treatment of adults and children ages 4 years and older with genetically confirmed Prader-Willi syndrome (PWS) who have hyperphagia.
  • The designation reflects the Agency’s determination that, based on an assessment of the preliminary data from the Phase 3 clinical development program, diazoxide choline may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies.
Key Takeaways:
  • Memo Therapeutics AG , a late-stage biotech company developing a best-in-class BKV-neutralizing antibody for BKV infection in kidney transplantation, announces that it has successfully raised CHF 20 million as part of an extension of its Series C financing, bringing the total amount raised in the funding round to CHF 45 million.
Key Takeaways:
  • Bayer and the German-based life science company, Evotec, announced that they have updated the focus of their strategic collaboration to developing innovative precision treatments for cardiovascular diseases (CVDs).
Key Takeaways:
  • Genomics plc and GSK announced a new collaboration to explore the potential for using polygenic risk scores (PRS) in clinical trials, to improve understanding of disease risk and patient selection, which could support optimisation of trial design.
  • Genomics plc is a global leader in PRS-powered technologies and approaches and has developed extensive genetic databases and algorithms to understand the genetic component of diseases.
Key Takeaways:
  • Harmony Biosciences, announced the acquisition of Epygenix Therapeutics, Inc., accelerating its growth strategy by adding a rare epilepsy franchise to its expanding late-stage pipeline of innovative CNS assets.
Key Takeaways:
  • Addex Therapeutics, a clinical-stage biopharmaceutical company focused on developing a portfolio of novel small molecule allosteric modulators for neurological disorders, announced top-line data from a Phase 2 epilepsy study evaluating adjunctive ADX71149 (JNJ-40411813) administration in patients with focal onset seizures with suboptimal response to levetiracetam or brivaracetam.
  • The Phase 2 study did not achieve statistical significance for the primary endpoint of time for patients to reach baseline seizure count when ADX71149 was added to standard of care.
Key Takeaways:
  • Variantyx, a leader in molecular diagnostics, announced that it has secured an additional $36 million in funding from its portfolio of investors which includes Peregrine Ventures, Pitango HealthTech, New Era Capital Partners and Bosch Ventures.
Key Takeaways:
  • Lykos Therapeutics, a company dedicated to transforming mental healthcare, announced the completion of a European Phase 2 study (MP18), an open-label, multicenter feasibility study of investigational midomafetamine (MDMA) capsules used in combination with psychological intervention ("MDMA-assisted therapy") for adults with post-traumatic stress disorder ("PTSD").
  • Having completed the pivotal clinical Phase 3 studies in the United States, the new drug application ("NDA") for Lykos' investigational midomafetamine capsules is currently under review by the U.S. Food and Drug Administration ("FDA").
Key Takeaways:
  • Cidara Therapeutics, a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) immunotherapies designed to save lives and improve the standard of care for patients facing serious diseases, announced that it has entered into a definitive agreement with Johnson & Johnson to reacquire the exclusive global development and commercial rights to CD388, which is in development for the prevention of all strains of influenza A and B.
Key Takeaways:
  • United Therapeutics Corporation, a public benefit corporation, announced the world’s first successful transplant of a UThymoKidney™, which the company produced, into a living person on April 12, 2024.
  • The first-ever transplant of a xenothymokidney into a living human recipient
  • The first-ever combined mechanical heart pump and organ transplant
  • The first-ever xenotransplant into a living human using only FDA-approved immunosuppressive medicines
Key Takeaways:
  • Xaira Therapeutics launched on a mission to help re-engineer the way we discover and develop medicines through the end-to-end application of emerging AI technologies.
  • A joint incubation by ARCH Venture Partners and Foresite Labs, Xaira launched with more than $1 billion of committed capital from lead investors ARCH Venture Partners and Foresite Capital, joined by F-Prime, NEA, Sequoia Capital, Lux Capital, Lightspeed Venture Partners, Menlo Ventures, Two Sigma Ventures, the Parker Institute for Cancer Immunotherapy (PICI), Byers Capital, Rsquared, and SV Angel, among others. 
  • Xaira brings together three core elements: advanced machine learning research, expansive data generation to power new models, and robust therapeutic product development.
Key Takeaways:
  • Vertex Pharmaceuticals and TreeFrog Therapeutics announced that Vertex has obtained an exclusive license to TreeFrog’s proprietary cell manufacturing technology, C-StemTM, to optimize production of Vertex’s cell therapies for type 1 diabetes (T1D).
  • TreeFrog and Vertex will collaborate to scale-up TreeFrog’s process to produce and amplify cells for Vertex’s T1D therapies.
Key Takeaways:
  • GSK announced the US Food and Drug Administration (FDA) accepted the supplemental Biologics License Application (sBLA) for Jemperli (dostarlimab) in combination with standard-of-care chemotherapy (carboplatin and paclitaxel) to expand treatment to all adult patients with primary advanced or recurrent endometrial cancer.
  • This would include patients with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumours.
Key Takeaways:
  • Abeona Therapeutics announced a regulatory update for prademagene zamikeracel (pz-cel). The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to the Company’s Biologics License Application (BLA) for pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB).
  • At the Late Cycle Review Meeting and in a subsequent information request, the FDA noted that certain additional information needed to satisfy Chemistry Manufacturing and Controls (CMC) requirements must be satisfactorily resolved before the application can be approved.
Key Takeaways:
  • Ipsen and Skyhawk Therapeutics announced the signing of an exclusive worldwide collaboration to discover and develop novel small molecules that modulate RNA for rare neurological diseases.
  • The agreement includes an option pursuant to which Ipsen would acquire exclusive license for the worldwide rights to develop successful development candidates (DC).
Key Takeaways:
  • Spinogenix, a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for SPG601 for the treatment of people with Fragile X Syndrome (FXS).
  • SPG601 works by restoring synaptic function to address core symptoms of FXS.
  • The Phase 2a trial will evaluate the neurophysiological and clinical effects of single-dose SPG601 and placebo in adult men with FXS.
Key Takeaways:
  • AstraZeneca’s FASENRA® (benralizumab) is approved by the US Food and Drug Administration (FDA) for add-on maintenance treatment for patients with severe asthma aged 6 to 11 with an eosinophilic phenotype.
  • FASENRA was first approved in 2017 as an add-on maintenance for the treatment of severe eosinophilic asthma (SEA) in patients aged 12 and older.
Key Takeaways:
  • Shinobi Therapeutics, a biotechnology company developing a new class of immune evasive iPS cell therapies, announced a partnership with Panasonic Holdings Corp and Kyoto University's Center for iPS Cell Research and Application (CiRA).
  • Through this strategic collaboration, the organizations aim to engineer a novel manufacturing platform to produce iPS-T cell therapies more efficiently and at lower cost than is possible with currently available technology.
Key Takeaways:
  • Outrun Therapeutics, the E3 ligase inhibitor and protein stabilisation specialist, exits from stealth following a $10m seed financing round.
  • Outrun’s platform has enabled it to rapidly build a pipeline of highly selective, small molecule, first-in-class E3 ligase inhibitors, as well as identify novel E3 ligase targets across multiple disease areas.
  • Outrun’s lead programme is focused on hard-to-treat solid tumours.
Key Takeaways:
  • Pathios Therapeutics , a biotech company focused on the development of first-in-class therapies for cancer, today announced that the company has raised USD $25 million in the first close of a Series B financing.
  • Proceeds from the raise will be used to support the continued development of Pathios’ unique approach to cancer immunotherapy focused on the inhibition of GPR65, a novel target that has been genetically associated with a range of immunologically-mediated diseases.
Key Takeaways:
  • Sage Therapeutics, announced topline results from PRECEDENT, a double-blind, placebo-controlled Phase 2 study of the investigational oral medicine dalzanemdor (SAGE-718) in people with mild cognitive impairment (MCI) in Parkinson’s Disease (PD).
  • The PRECEDENT Study did not meet its primary endpoint of demonstrating statistically significant difference from baseline in participants treated with once-daily dalzanemdor versus placebo on the Wechsler Adult Intelligence Scale Fourth Edition-IV (WAIS-IV) Coding Test score at Day 42. Dalzanemdor (SAGE-718) was generally well-tolerated, and there were no new safety signals observed.
Key Takeaways:
  • Ventus Therapeutics, a clinical-stage biopharmaceutical company utilizing its proprietary structural biology and computational chemistry platform, ReSOLVETM, to develop differentiated small molecule therapeutics, announced results from its Phase 1 clinical trial of VENT-02, a novel, oral, brain-penetrant NLRP3 inhibitor.
  • The Phase 1 trial evaluated the pharmacodynamics, pharmacokinetics, safety, and tolerability of VENT-02 across a broad range of single and multiple ascending doses in adult healthy volunteers.
Key Takeaways:
  • Alvotech and Teva Pharmaceuticals, announced that the U.S. Food and Drug Administration (FDA) has approved SELARSDI (ustekinumab-aekn) injection for subcutaneous use, as a biosimilar to Stelara®, for the treatment of moderate to severe plaque psoriasis and for active psoriatic arthritis in adults and pediatric patients 6 years and older.
  • Under the strategic partnership between Teva and Alvotech, Teva is responsible for the exclusive commercialization of SELARSDI in the United States.
Key Takeaways:
  • Neumora Therapeutics, a clinical-stage biopharmaceutical company with a therapeutics pipeline consisting of seven clinical and pre-clinical brain disease programs, announced that the Phase 1 trial of NMRA-266 has been placed on clinical hold by the U.S. Food and Drug Administration (FDA). NMRA-266 is a positive allosteric modulator (PAM) of the M4 muscarinic receptor and is part of the Company’s M4 PAM franchise.
Key Takeaways:
  • Phanes Therapeutics, a clinical stage biotech company focused on innovative drug discovery and development in oncology, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to PT217 for the treatment of patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression following platinum chemotherapy with or without a checkpoint inhibitor.
  • PT217 was also granted orphan drug designation for the treatment of small cell lung cancer by the FDA in 2022.
Key Takeaways:
  • Seaport Therapeutics, a clinical-stage biopharmaceutical company that is charging a proven path in neuropsychiatry, announced the closing of a $100 million oversubscribed Series A financing round.
Key Takeaways:
  • Vertex Pharmaceuticals and Alpine Immune Sciences, a biotechnology company focused on discovering and developing innovative, protein-based immunotherapies, announced that the companies have entered into a definitive agreement under which Vertex will acquire Alpine for $65 per share or approximately $4.9 billion in cash.
  • The transaction was unanimously approved by both the Vertex and Alpine Boards of Directors and is anticipated to close later this quarter.
Key Takeaways:
  • Century Therapeutics, an innovative biotechnology company developing induced pluripotent stem cell (iPSC)-derived cell therapies in immuno-oncology and autoimmune and inflammatory diseases, announced plans to expand clinical development for its lead program, CNTY-101, a CD19-targeting iNK cell therapy, into additional autoimmune disease indications. CNTY-101 is currently being evaluated in a clinical trial in B-cell malignancies (ELiPSE-1) as well as a planned clinical trial in systemic lupus erythematosus (SLE) (CALiPSO-1), which is on track to be initiated in the first half of 2024.
  • Century’s increased research and development activities in autoimmune diseases are further supported by a $60 million private placement of its common stock to certain institutional investors.
Key Takeaways:
  • Allay Therapeutics, a clinical-stage biotechnology company pioneering ultra-sustained analgesic products to transform post-surgical pain management and recuperation, announced the results from a Phase 2B clinical study of its lead investigational product ATX101.
  • ATX101 achieved sustained post-surgical pain relief for up to four weeks for patients following total knee arthroplasty (replacement, or TKA surgery) and outperformed a standard of care (SOC) active comparator, bupivacaine, on durability of effect, use of opioids to support pain relief and the potential for patients to return to day-to-day activities earlier.
Key Takeaways:
  • Eliem Therapeutics and Tenet Medicines, a development-stage private biotechnology company, announced that the companies have entered into a definitive acquisition agreement whereby Eliem has agreed to acquire Tenet.
  • The combined company plans to focus on advancing TNT119, a potentially best-in class anti-CD19 antibody, designed for a broad range of autoimmune diseases, including systemic lupus erythematosus, immune thrombocytopenia and membranous nephropathy.
Key Takeaways:
  • Nuvation Bio , a late-stage, global biopharmaceutical company tackling some of the greatest unmet needs in oncology by developing differentiated and novel therapeutic candidates, announced the completion of the acquisition of AnHeart Therapeutics Ltd. (AnHeart), a late-stage, global biopharmaceutical company developing novel precision therapies for people with cancer. With the acquisition’s completion,
  • AnHeart is now a wholly-owned subsidiary of Nuvation Bio.
Key Takeaways:
  • Basilea Pharmaceutica Ltd, a commercial-stage biopharmaceutical company committed to meeting the needs of patients with severe bacterial and fungal infections, announced that the US Food and Drug Administration (FDA) approved ZEVTERA® (ceftobiprole medocaril sodium for injection), for the treatment of adult patients with Staphylococcus aureus bloodstream infections (bacteremia) (SAB), including those with right-sided infective endocarditis, and adult patients with acute bacterial skin and skin structure infections (ABSSSI) and for adult and pediatric patients (3 months to less than 18 years old) with community-acquired bacterial pneumonia (CABP).
Key Takeaways:
  • Tome Biosciences, Inc., the programmable genomic integration (PGI) company, announces the issuance of US Patent 11,952,571 for “Systems, Methods, and Compositions for Site-Specific Genetic Engineering Using Programmable Addition via SiteSpecific Targeting Elements (PASTE)” by the US Patent and Trademark Office.
  • This patent further protects Tome’s foundational PGI technology, integrase-mediated PGI (I-PGI), describes a system capable of performing I-PGI in a mammalian cell, and is exclusively licensed to Tome from MIT.
Key Takeaways:
  • This press release is for U.S. audiences only 
  • Blue Earth Therapeutics, a Bracco company and emerging leader in the development of innovative next generation therapeutic radiopharmaceuticals, announced results from a series of preclinical analyses designed to identify synergistic combinations of known anticancer drugs with 177Lu‐rhPSMA‐10.1 radioligand therapy, and from a preclinical efficacy analysis of the lead novel drug combination for the treatment of prostate cancer.
  • Results from a systematic in vitro screen identified MEK inhibitor cobimetinib as a lead candidate with potential for synergistic combination with 177Lu‐rhPSMA‐10.1, and the preclinical efficacy analysis showed enhanced therapeutic effect of this drug combination when compared to the untreated control and to the single agents alone.

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