Biotech News

Approved Wednesday for severe beta thalassemia, Zynteglo will test insurers’ willingness to pay for expensive one-time treatments. Its market launch is likely to be watched carefully by other gene therapy developers.

The first patient has been enrolled on a study to trial a treatment for relapsed or refractory metastatic colorectal cancer (R/R mCRC) in the U.S. Innovative Cellular Therapeutics (ICT), Inc. a clinical-stage biotechnology company developing a comprehensive portfolio of chimeric antigen receptor (CAR) T cell therapies made the announcement today (August 18). Tolerability The study is an open-label, single arm, multicenter phase 1 dose-escalation clinical trial evaluating the safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of the drug, GCC19CART, in patients with R/R mCRC in the U.S. Larry Lei Xiao, chief executive officer of ICT, said: “This is an important and exciting milestone for ICT. There is a significant unmet medical need for R/R mCRC patients. We believe that GCC19CART has the potential to provide significant clinical benefit to these patients.” Lead product GCC19CART is the company’s lead product candidate from the Company’s CoupledCAR technology. It has been tested in 35 patients in an Institutional Review Board-approved trial in China. Innovative Cellular Therapeutics is a private clinical stage biotechnology company headquartered in Rockville, The post First patient on Innovative Cellular Therapeutics’ study looking at treating colorectal cancer appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

The company plans to soon seek approval of a once-monthly preventive drug for hereditary angioedema, a disease other drugmakers are targeting with gene editing and RNA medicines.

Technology transfer offices are vital departments for commercializing an academic’s biotech innovations, yet they can also be resource-stretched and averse to risk. Here are some ways they can streamline their process and land more deals with investors and big pharma.     Technology transfer offices (TTOs) perform vital functions in universities by guiding academics through the process of patenting and commercializing their ideas. They also help in researching the surrounding market, closing deals with companies and leveraging networks in the industry. However, there is a lot of variability in the attitudes of TTOs toward the commercialization of intellectual property (IP), particularly in the U.K.  “Tech transfer offices, by and large, are pretty resource-stretched, overrun groups of people,” said Toby Reid, executive director at We Are Pioneer Group, a U.K.-based business incubator that houses and funds startup companies across sectors including technology, foodtech and biotechnology.  However, there are some major success stories where academics, TTOs and other partners have been able to create hard-hitting spinouts or licensing deals with big pharma companies. The post How technology transfer offices can navigate biotech commercialization appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

Curis, Inc., a biotech company focused on the development of innovative therapeutics for the treatment of cancer, says the U.S. Food and Drug Administration (FDA) has lifted the partial clinical hold on the TakeAim lymphoma phase 1/2 study of emavusertib after reviewing the data package submitted by Curis. “We are excited to announce that FDA has completed its review of the TakeAim Lymphoma study and has lifted the partial clinical hold. We are working with our clinical sites to quickly resume enrollment of new patients in this study in the third quarter,” said James Dentzer, president and CEO of Curis. Previously, Curis announced the FDA had placed separate partial clinical holds on the TakeAim Leukemia and TakeAim Lymphoma studies on April 4 and April 11, 2022, respectively.  The partial hold on the TakeAim leukemia study was issued by the FDA Division of Hematologic Malignancies 1 (DHM1), which regulates clinical studies in leukemia. The partial hold on the TakeAim lymphoma study, and the lifting of that hold, was issued by the FDA Division of Hematologic Malignancies 2 (DHM2), The post FDA lifts partial clinical hold on Curis lymphoma study appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

U.S. clinical-stage biopharma company 89bio, Inc. has completed enrollment in ENLIVEN, the phase 2b trial of pegozafermin for the treatment of non-alcoholic steatohepatitis (NASH). “We are very pleased to have successfully completed enrollment of 219 total patients in ENLIVEN, a well-powered trial that builds on positive data demonstrating pegozafermin’s broad metabolic effects and favorable safety/tolerability profile,” said Hank Mansbach, chief medical officer at 89bio.  “The impressive pace of enrollment is a testament to both the high level of engagement by our clinical trial sites and our team’s strategic execution and brings us one step closer to addressing the need for new treatment options for patients suffering from NASH. We see this as a significant milestone in the pegozafermin clinical program and, building on this momentum, we expect to report topline data from ENLIVEN in the first quarter of 2023.” Trial details ENLIVEN is a multicenter, randomized, double-blind, placebo-controlled phase 2b trial in biopsy-confirmed NASH patients with fibrosis stage 2 or 3. The trial enrolled a total of 219 patients who will receive either one of two weekly doses (15mg or 30mg) or an every two-week dose (44mg) of pegozafermin in a liquid formulation or placebo for 24 weeks. The post 89bio completes enrollment in NASH phase 2b trial appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

A gene therapy drug approved by the U.S. Food and Drug Administration (FDA) has been made accessible for people with beta-thalassemia. Zynteglo, a betibeglogene autoemcel, also known as beti-cel, treats adults and pediatric patients with the disease who need regular red blood (RBC) transfusions. The condition is passed down from one or both parents through their genes and it means the body makes less hemoglobin than normal. bluebird bio recently released details of its U.S commercial infrastructure to support rapid access to the treatment including an outcomes based contract offering and a patient support program. Blood cell transfusion Tom Klima, chief commercial officer at bluebird bio, said: “As the first and only FDA-approved gene therapy for people with beta-thalassemia who require regular red blood cell transfusions, and with the potential to untether patients from a lifelong, burdensome treatment regimen, Zynteglo is setting the standard for what a one-time therapy can deliver. “We have developed an equally innovative commercial infrastructure in collaboration with payers and providers, The post Patients with beta-thalassemia able to access first approved gene therapy appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

Geoffrey Porges, who has spent nearly two decades researching biotechs at SVB Securities and AllianceBernstein, will now oversee and seek to grow the drug discovery specialist's network of partnerships.

Australia’s Carina Biotech has raised A$7.5 million (US$5.2 million) at first close of its current funding round. Venture capital business Tenmile was the cornerstone investor, with support from existing investors. The capital raise enables Carina to complete its Investigational New Drug (IND) enabling studies and initiate a CAR-T clinical trial of its LGR5 CAR-T cell for patients with advanced colorectal (bowel) cancer. “Colorectal cancer is Australia’s second deadliest cancer and its incidence is rising in people under the age of 50. Many younger people are diagnosed at advanced stages of the disease with very poor prognoses. This is a lethal cancer for all Australians, young and old, that we want to be able to treat. CAR-T therapy is currently used for the treatment of blood cancers and it has proved to be curative,” said CEO Deborah Rathjen. “We are very excited to be able to bring this personalized form of immunotherapy embedded with Carina’s proprietary CAR-T technology to patients with solid cancers.” Tenmile executive chair, The post Carina Biotech raises funds for colorectal cancer research appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

Hereditary angioedema (HAE), a condition that causes recurrent attacks of severe swelling, could be prevented by a monoclonal antibody after positive top line phase 3 results were reported. Garadacimab, biotech company CSL’s investigational monoclonal antibody inhibiting factor XIIa, is being developed as a long-term preventive treatment for patients with HAE. Efficacy objectives The study met its primary and secondary efficacy objectives and also demonstrated favorable safety and tolerability. CSL aims to begin filing with global health authorities at the end of the current fiscal year for full approval. The multicenter, double-blind, randomized, placebo-controlled, parallel-arm study (known as VANGUARD) evaluated the efficacy and safety of monthly subcutaneous garadacimab administration in the prevention of HAE attacks compared to the placebo for six months. Full results from the study will be presented at an upcoming scientific congress and published in a peer-reviewed journal.  Bill Mezzanotte, chief medical officer for CSL said: “These results underscore our belief that garadacimab has the potential to become a transformative first-in-class therapy for people living with HAE, The post CSL gets positive results for preventive treatment in patients with hereditary angioedema appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

A collaboration called The Project to Accelerate New Treatments for Tuberculosis (PAN-TB) was announced yesterday (August 17). The joint development agreement (JDA) will support the progression of two investigational tuberculosis (TB) combination treatment regimens into phase 2 clinical development. The collaboration is made up of organizations contributing five antimicrobial agents. Active pulmonary TB Together they will evaluate whether the regimens, which combine registered products and new chemical entities (NCEs), can effectively treat all forms of active pulmonary TB using substantially shorter treatment durations than existing drug regimens, with the goal of identifying a regimen suitable for phase 3 development. TB is a major global cause of illness, disability and catastrophic household costs, and is one of the leading causes of death from an infectious disease worldwide, responsible for an estimated 1.5 million deaths per year. A shorter drug regimen that can treat both drug-susceptible and drug-resistant forms of TB in potentially three months or less could provide a significant benefit to both patients and health systems and may overcome the need for accompanying drug-resistance testing. The post Collaboration to advance investigational tuberculosis drug treatments appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

Singapore biotech company TurtleTree has announced production of LF+, the world’s first sustainable bovine lactoferrin created using precision fermentation technology. A key part of the company’s ongoing work with cultivated dairy, precision fermentation uses microbial hosts for the mass production of ingredients. Compared to traditional methods, precision fermentation offers far greater efficiency and volume, enabling viable production at commercial scale. In pioneering the use of this technology for lactoferrin and its inclusion in food, TurtleTree said it will now accelerate its development efforts for LF+ in order to achieve a commercial launch in 2023. TurtleTree chief strategist, Max Rye, said: “Looking at recent events like the infant formula shortage in the US and the disruptions in food supply arising from COVID-19, we felt that we could no longer sit on the sidelines. With LF+, we see a way to contribute towards better nutrition in the here and now while our longer-term dairy projects remain in the pipeline. The hope is to ultimately take a proactive approach in creating the world we want to see and genuinely make a difference during a time of need.” The post TurtleTree produces first precision fermentation sustainable lactoferrin appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

The federal government will begin the transition as early as this fall, clearing the way for drugmakers to control sales and distribution of their medicines.

AUM LifeTech, Inc., a Philadelphia-based preclinical stage biotech company, has received a Small Business Innovation Research (SBIR) grant from the National Cancer Institute (NCI) of the National Institutes of Health (NIH) to advance its preclinical program in lung cancer immunotherapy using a non-viral RNA-targeting gene therapy approach.  “This grant will help advance our preclinical immunotherapy program in lung cancer and provide us the next critical steps towards IND enabling studies,” said Veenu Aishwarya, founder and CEO of AUM LifeTech, Inc. AUM LifeTech will use the funds to advance the development of its new form of cancer immunotherapy, which involves targeting the messenger RNA (mRNA) of a gene, FOXP3, and thereby inhibiting the function of immune cells, called T-regulatory (Treg) cells.  Tregs are known to have an immunosuppressive function and, in some instances, prevent the body from fighting cancer and lead to cancer progression. The functions of Tregs are critically dependent upon their expression of FOXP3.  AUM LifeTech’s FANA Antisense Oligonucleotide (FANA ASO) RNA silencing approach allows efficient and selective  The post AUM LifeTech secures $400,000 grant for lung cancer immunotherapy program appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

MiAlgae, a Scottish biotech start-up, has secured new investment of £2.3 million ($2.8 million) to help drive the next stage of its growth journey. Established in 2016, MiAlgae has created a solution to producing a sustainable source of omega-3 for animal and human consumption.  Championing the circular economy, the company has developed a novel biotechnology platform that uses low-value coproducts from the food and drink industry as a feedstock to initially grow microalgae rich in omega-3 oils. Plans are already in place to commercialize other high-value compounds and pigments through expansion of the platform. The Conduit Impact Fund, managed by Ascension led the round, investing £900,000 ($1.1 million), with new investor SIS Ventures putting forward £350,000 ($421,560).  Having already previously invested, Equity Gap, Old College Capital and Scottish Enterprise all committed to additional funding, totalling more than £1m ($1.2 million). The investment builds on the £850k ($1 million) of grant funding recently secured from Zero Waste Scotland and Scottish Enterprise. Expansion plans The £2.3m investment will support the company in its growth plans which include the completion of a commercial demonstrator facility near Stirling, The post Scottish biotech startup secures funding for sustainable omega-3 production appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a cost of $2.8 million in the U.S. 

The French drugmaker’s shares fell by more than 6% after the once promising treatment, called amcenestrant, failed its second major trial this year.

The COVID-19 vaccine maker has appointed James Mock, currently PerkinElmer’s CFO, as its next finance head, three months after ousting Jorge Gomez from the role. 

August 18 2022: the clinical stage of ITM’s lead candidate was corrected to phase 3. Radiopharmaceuticals have been a relatively niche area of cancer therapy for decades. Steffen Schuster, the CEO of Germany’s ITM Group, outlines how the space is heating up, and how much nuclear medicine centers still need to grow. There are many areas in oncology research that are generating investor excitement, including immuno-oncology, DNA damage repair and protein degradation. Another emerging area with huge potential is the treatment of cancer using drugs that carry radioactive molecules, known as radiopharmaceuticals. “This space is hot right now,” said Steffen Schuster, CEO of the German firm ITM Group. “​​You see acquisitions in the field and you see the money flowing in, in terms of venture capital, private equity and growth capital.” Some notable acquisitions in the last decade include Novartis’ takeover of Advanced Accelerator Applications and Endocyte, and Bayer’s purchase of Noria Therapeutics and its subsidiary PSMA Therapeutics. Radiopharmaceuticals typically consist of a tumor-hunting drug that is chemically attached to a radioactive molecule called a radioisotope. The post The challenges of developing radiopharmaceuticals for cancer appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

A biopharma company advancing therapies for acute and chronic gastrointestinal (GI) complications has announced today (August 17) the closing of its previously announced underwritten public offering for $13.8 million. An underwritten public offering means a public offering in which the common stock is offered and sold on a firm commitment basis through one or more underwriters. Gross proceeds U.S.-based Palisade Bio gross proceeds were before deducting underwriting discounts and commissions and offering expenses, including full exercise of the underwriter’s over-allotment option to buy additional common stock shares and warrants. The offering was made up of 49,360,000 shares of common stock, 1,460 shares of series B convertible preferred stock, 55,200,000 series 1 warrants with an exercise price of $0.25 per share and a term of one year following the issuance date, and 55,200,000 series 2 warrants with an exercise price of $0.25 per share and a term of five years following the issuance date. Stockholder approval The price per share of common stock, series 1 warrant and series 2 warrant is $0.25. The post Palisade Bio announces closing of $13.8M upsized underwritten public offering appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

An early access program for semiconductor synthesized DNA has been opened by UK-based gene synthetic biology company, Evonetix. Evonetix’s DNA synthesis capability brings together semiconductor chip design and proprietary, thermally controlled synthesis chemistry, integral to the company’s future gene synthesis platforms. Silicon chip The company’s proprietary process utilizes a silicon chip to control the synthesis of DNA at many thousands of independent thermally controlled reaction sites, or ‘pixels’, on the chip surface. Matt Hayes, founder and chief technology officer at Evonetix, said: “We have been working on optimizing the different elements of our technology ready to share DNA with researchers. “Reaching this point in our development is a key milestone, paving the way for the expansion in scale that only semiconductor-based technologies can achieve.” Long DNA Combined with the company’s patented chemistry, this approach enables the accurate synthesis of thousands of sequences on a single chip to meet the demand for complex libraries and assembly of long DNA, addressing the DNA supply bottleneck in the field of synthetic biology. The post Researchers invited to apply for DNA synthesized by Evonetix’s platform appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

The European Investment Bank (EIB) is providing €22 million ($22.4 million) in financing to Ryvu Therapeutics, a Polish clinical-stage drug discovery and development company focusing on novel small molecule therapies that address unmet medical needs in oncology.  The funding is being provided under the EIB’s venture debt instrument, which is tailored to the specific financing needs of high-growth innovative companies. The European Fund for Strategic Investments, part of the Investment Plan for Europe, is backing this funding with a guarantee. The EIB’s support will help Ryvu finance its development pipeline of new cancer treatments from discovery through to clinical trials. Ultimately, Ryvu aims to address the clinical limitations of current treatments in oncology and provide patients with access to innovative therapies for haematologic and solid tumors. EIB vice-president Teresa Czerwińska said: “Financing projects within the field of research, development and innovation is one of the EIB’s top priorities and is especially relevant today, as the world suffers from the health crisis caused by the pandemic. The post Ryvu Therapeutics bags €22M from EIB to work on new cancer treatments appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

Trial results position the company to seek approval for indolent systemic mastocytosis, which executives have painted as a “multibillion dollar” market opportunity. Even so, shares fell by double digits in trading Wednesday.

AGC Biologics has announced its partnership with supplier of human mesenchymal stem/stromal cells (hMSCs), RoosterBio Inc. The partnership, the companies say creates an end-to-end solution for the development and production of hMSCs and exosome therapeutics using RoosterBio’s well-established cell and media products and process development services together with AGC’s cell and gene therapy manufacturing capabilities. RoosterBio, that also supplies engineered media and bioprocess development series, has means that include genetic engineering of cells and exosomes to express therapeutic targets, upstream processing in both 2D flask and bioreactor systems, downstream purification to achieve desired purity and potency and comprehensive analytical characterization of the resulting formulated cell or exosome therapy. Outstanding Tim Kelly, chief executive officer of RoosterBio, said: “AGC Biologics has an outstanding track record of success in the manufacture of clinical and commercial advanced therapies and an ever-expanding global network to meet the needs of our customers. “Biopharmaceutical companies striving to develop engineered cell and exosome therapies require proven, flexible technologies paired with reliable and scalable manufacturing solutions.  The post New partnership delivers clinically proven products and manufacturing processes for advanced therapies appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.

Sanofi says it is discontinuing the global clinical development program of amcenestrant, an investigational oral selective estrogen receptor degrader (SERD).  The decision was based on the outcome of an interim analysis of the phase 3 AMEERA-5 trial evaluating amcenestrant in combination with palbociclib compared with letrozole in combination with palbociclib in patients with estrogen receptor-positive (ER+)/human epidermal growth factor receptor 2-negative (HER2-) advanced breast cancer. An independent data monitoring committee found amcenestrant in combination with palbociclib did not meet the prespecified boundary for continuation in comparison with the control arm and recommended stopping the trial. No new safety signals were observed. Trial participants will be transitioned to letrozole in combination with palbociclib or another appropriate standard of care therapy, as determined by their physician. Sharing results The company will continue to review the data and plans to share the results with the scientific community in the future. All other studies of amcenestrant, including in early-stage breast cancer (AMEERA-6), will be discontinued. John Reed, The post Sanofi drops amcenestrant clinical development program appeared first on Labiotech.eu. © Labiotech UG and Labiotech.eu. Unauthorized use and/or duplication of this material without express and written permission from this site’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Labiotech UG and Labiotech.eu with appropriate and specific direction to the original content.