Dec 1, 2023

In the news this week, biotech Arvinas raises $350 million PIPE American protein degradation company Arvinas has bagged $350 million in a private placement (PIPE). The company aims to use these funds to advance clinical and preclinical trials. Developing proteolysis targeting chimeras, its pipeline includes candidates that could potentially treat breast cancer, prostate cancer, solid tumors and neurological diseases. The fundraising was co-led by EcoR1 Capital and entities managed by RTW Investments, LP, with participation from investors like Adage Capital Partners LP, ArrowMark Partners, Avidity Partners, BB Biotech, Boxer Capital, Citadel Global Equities, and Great Point Partners, LLC, among others. Aro Biotherapeutics secures $41.5 million to advance Pompe disease candidate Genetic medicine company Aro Biotherapeutics, based in the U.S., has secured $41.5 million in a series B round. The funds will contribute towards the development of its Pompe disease candidate BX1100, which is currently in phase 1 stage. Pompe disease is a genetic condition wherein a build-up of glycogen, a complex sugar, occurs. This can lead to patients experiencing muscle weakness and breathing difficulties. A first-in-human trial has begun with BX1100, which is comprised of a CD71 receptor binding centyrin conjugated to a small interfering RNA (siRNA) that interferes with the expression of Gys1 mRNA, in order to reduce its levels. The funding round was led by Cowen Healthcare Investments, and investors like Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Northpond Ventures, Healthcap and BVF Partners, L.P., took part. Vivodyne to develop lab-grown human organs U.S.-based Vivodyne has closed a $38 million seed financing round. The funds will help advance the company’s discovery pipeline, which is powered by artificial intelligence (AI). In an effort to help identify therapeutic targets to predict patient responses, the company is creating human organ tissues. It has bioengineered more than 20 types of human organ tissues, to aid in the drug discovery process. “By testing drugs and life-saving biologics directly on these realistic human tissues at an unprecedented scale and resolution, we can improve the success rates of therapeutics entering clinical trials,” said Alex Morgan, partner at Khosla Ventures, the company that led the fundraising. Cradle to accelerate protein engineering with series A funds Netherlands-based machine learning company Cradle, specializing in protein engineering, has raised $24 million since exiting stealth last year. The funds obtained from the series A round will be used to accelerate protein optimization. It has 12 ongoing research and development projects, which focus on engineering enzymes, vaccines, peptides, and even antibodies. The round was led by Index Ventures, and Kindred Capital, as well as angel investors like the chief executive officer (CEO) of Recursion, and former CEO of Thomson Reuters who is currently the lead director of Merck, participated. Harness Therapeutics secures more than $5 million to treat neurodegenerative diseases U.K.-based Harness Therapeutics has secured £4 million ($5.07 million). So far, the company has gathered £17.6 million ($22.24 million), since its inception in 2019. These funds will be used to further develop its lead candidate that is designed to treat Huntington’s Disease, as it targets FAN1 nuclease, which is meant to modify disease progression. The company has received funding from life science investors like Takeda Ventures, The Dementia Discovery Fund and Epidarex Capital. The latest biotech news in mergers, acquisitions, and strategic partnerships Boehringer Ingelheim acquires T3 Pharma in $500 million buyout With a motive to expand its immuno-oncology portfolio, German multinational company Boehringer Ingelheim has acquired T3 Pharma, a company that focuses on therapeutic research in solid tumors. Founded in 2015, T3 Pharma had set out to develop a cancer therapy platform based on regulating the natural behaviors of live bacteria. The acquisition would hand over T3 Pharma’s platform that uses engineered Yersinia enterocolitica bacteria to deliver bioactive proteins directly into the tumor microenvironment, to Boehringer Ingelheim. In exchange, T3 Pharma will be eligible to receive up to 450 million CHF ($513 million) in payments. Syncona to snap up Freeline Therapeutics British biotech Freeline Therapeutics has been bought by life science investment company Syncona. This will enable Freeline Therapeutics to pursue the development of its AAV gene therapy candidate for Gaucher disease. “Given the compelling data that we have seen from the first two patients treated with FLT201, we are committed to advancing FLT201 as expeditiously as possible, and we believe we will be better positioned to do that as a privately held, Syncona-backed company than we could, by continuing as a publicly traded company in the current environment,” said Michael Parini, CEO of Freeline. As part of the buyout, Freeline will receive $28.3 million upfront as well as up to $15 million in secured convertible debt financing. Avidity Biosciences and Bristol Myers Squibb in cardiovascular partnership Avidity Biosciences will receive $100 million upfront from Bristol Myers Squibb as the two collaborate on creating a cardiovascular program of up to five targets. Avidity could potentially receive up to $2.2 billion in milestone payments in exchange for Bristol Myers Squibb’s access to its antibody oligonucleotide conjugates (AOC) platform technology. The platform combines the technology of monoclonal antibodies and the potency of oligonucleotide therapies to broaden treatment options for people. This partnership to build a cardiovascular program is separate from Avidity’s own discovery pipeline that targets rare skeletal muscle conditions and rare cardiac muscle diseases. AnaptysBio to acquire BDCA2 modulator antibody portfolio from Centessa Pharmaceuticals Immunomodulators are designed to treat autoimmune and inflammatory diseases by modifying the immune system so it works more effectively. American company AnaptysBio is expanding its immune cell modulator portfolio by acquiring British biotech Centessa’s blood dendritic cell antigen 2 (BDCA2) modulator antibodies. This will include its lead asset CBS004, a monoclonal antibody for the treatment of autoimmune conditions. As part of the agreement, CBS004 will be renamed to ANB101 next year. The candidate is being considered for an investigational new drug (IND) application by the company. The deal will see AnaptysBio pay Centessa Pharmaceuticals $7 million upfront. Boehringer Ingelheim and IBM in AI collaboration Multinationals Boehringer Ingelheim and IBM have begun a partnership that will allow Boehringer to access IBM’s AI models to discover antibodies. As antibodies are at the forefront of treating infectious diseases and even cancer, this accelerates the antibody discovery process through in-silico methods, which relies on IBM’s foundation model technologies. “We are thrilled to now bring IBM’s multimodal foundation model technologies to Boehringer, a leader in the development and manufacturing of antibody-based therapies, to help accelerate the pace at which Boehringer can create new therapeutics,” said Alessandro Curioni, vice president of Accelerated Discovery at IBM Research, in a press release on Wednesday. The latest biotech news in clinical trials 89bio sees encouraging results in NASH trial American biotech 89bio has announced that its nonalcoholic steatohepatitis (NASH) candidate obtained promising results in a phase 1b/2a trial. The drug candidate pegozafermin is a fibroblast growth factor 21 (FGF21) – a hormone that modulates drivers of NASH – analog. 63% of the patients who were administered pegozafermin achieved 2-point or greater improvement in NAS score without worsening of fibrosis, which means that the trial met the primary endpoint. While there were no serious adverse events that were drug-related, according to the press release, one treatment-related discontinuation took place. “We are very pleased with the full data from our phase 1b/2a study showing promising efficacy and safety and the encouraging histology results in this cohort further support pegozafermin as a promising drug for the treatment of NASH,” said Hank Mansbach, chief medical officer of 89bio. A phase 2b trial for pegozafermin is ongoing. Exonate’s phase 1b/2a diabetic retinopathy trial reaps success Exonate, a pharmaceutical based in England, has found that its lead ophthalmology candidate EXN407 met all endpoints in a phase 1b/2a trial for patients with diabetic macular oedema. Diabetic macular oedema is a condition where blood vessels leak fluid into the retina in the eye, which could lead to blindness. Exonate’s results are moving EXN407 towards potentially being the first topical treatment for the disease. The therapy, which is a small molecule inhibitor of splice factor kinase SRPK1, was found to be safe and tolerable. Now, the company will press forward with a phase 2 study set to begin next year. Roivant and Priovant fail systemic lupus erythematosus trial A phase 2 study led by American companies Roivant and Priovant for the oral candidate brepocitinib, failed to meet its primary endpoint in the autoimmune disease systemic lupus erythematosus (SLE), which was meant to achieve a systemic lupus erythematosus responder index change of 4 (sri-4) at week 52. While the trial saw some of the highest SRI-4 responder rates ever observed in a lupus study in the active arm of the trial, it also saw the “highest placebo response rate observed in any significant SLE study.” The company is ending studies in SLE, however, will continue to evaluate brepocitinib, as it has established a positive safety and tolerability profile. In fact, Priovant will soon release results from another phase 2 study brepocitinib in non-infectious uveitis, a chronic eye inflammatory condition. argenx fails autoimmune disease trial, leads to slump in stocks argenx failed to meet the primary endpoint in a trial evaluating VYVGART Hytrulo in adults with primary immune thrombocytopenia (ITP). ITP is an autoimmune bleeding disorder that is characterized by an abnormally low platelet count. The candidate could not attain a sustained platelet count response in chronic ITP patients. Only about 14% of patients demonstrated a sustained platelet count response compared to 16.2% in the placebo group. Secondary endpoints were not met either. This led to a drop in stock prices when it hit the news on Tuesday. Nevertheless, VYVGART Hytrulo, a combination of a human IgG1 antibody fragment, recombinant human hyaluronidase PH20 (rHuPH20), and Halozyme’s drug delivery technology, maintained a favorable safety and tolerability profile. The latest biotech news in science Melanoma and lung cancer drug shows therapeutic potential in cutaneous squamous cell carcinoma A drug that was developed to treat melanoma and lung cancer, has shown that it could even possibly treat cutaneous squamous cell carcinoma (CSCC), a common kind of skin cancer. Researchers at the University of Turku in Finland, have found that the drug plixorafenib is capable of inhibiting the activity of various pathways, like the Ras signaling pathway and the TGF-b pathway, that lead to CSCC manifestation. They used 3D cell culture models of cutaneous squamous cell carcinoma tumors to identify the therapeutic changes with plixorafenib. “Our results show that plixorafenib is a very promising candidate for a clinical trial for treating local or metastatic cutaneous squamous cell carcinoma,” said Veli-Matti Kähäri, Professor of Dermatology at the University of Turku, who co-led the study, in a press release last week. Could a newfound molecular pathway halt lung cancer? Lung cancer is the leading cause of cancer-related deaths across the globe, accounting for the highest mortality rates among both men and women, according to the World Health Organization (WHO). Scientists at Tulane University in the U.S. have discovered that the tumor suppressor protein RBM10 can stop the growth of lung cancer. This is done by destabilizing c-Myc, which is a protein that enables the proliferation of cancer cells when it is overexpressed. Moreover, the study identified that a mutant form of RBM10 fails to suppress the c-Myc protein, and so instead of inhibiting lung cancer, it promotes tumor growth. “Hopefully, we can design a molecule to specifically target the mutant, since that’s a special structure not existing in the normal tissue,” said senior study author Dr. Hua Lu, at the Reynolds and Ryan Families Chair in Translational Cancer at the Tulane University School of Medicine. “If we can convert this mutant, we can hopefully make it suppress c-Myc’s cancer-causing activity.” New drugs to treat ovarian cancer developed A new drug has been developed to treat ovarian cancer. Scientists at the Shanghai Institute of Organic Chemistry of the Chinese Academy of Sciences in China, have created small molecule inhibitors of CPSF3, aimed at disrupting the enzyme activity of CPSF3. Evaluating these inhibitors gives insight into how they could help establish CPSF3-dependent transcription termination as a key mechanism that could be targeted to treat ovarian cancer. The inhibitors displayed target specificity in the study, which was published in Science Advances, last week. Online partnering event with academia Partner with academic leaders around sustainable materials research