Wave Life Sciences’ stock took another plunge after the company reported early results from its Huntington’s disease study. Cambridge, MA-based Wave Life Sciences (NASDAQ: [[ticker:WVE]]) earlier this week released topline data from its ongoing Phase 1b/2a PRECISION-HD2 trial evaluating WVE-120102. The experimental therapy is being developed for the potential treatment of Huntington’s disease (HD), a … Continue reading “Wave’s Huntington’s Drug Disappoints in Clinical Trial, Stock Sinks”
Category: National
Investors Go With Gut Ahead of Microbiome Therapy Boom, Says Seventure
French VC firm Seventure is raising over €200 million in a second dedicated fund focused on the microbiome and says the therapeutic space will mature within the next three years. In March, France’s Seventure announced it is looking to raise €200 million ($220 million) in its Health for Life Capital II fund dedicated to the … Continue reading “Investors Go With Gut Ahead of Microbiome Therapy Boom, Says Seventure”
Allergan Wins FDA Nod for First Migraine Pill in New Class of Medicines
The FDA on Monday approved a pill from Allergan to treat migraine in adults. The treatment, ubrogepant (Ubrelvy), is part of a new class of migraine medicines designed to block calcitonin gene-related peptide (CGRP), a protein thought to play a role in pain transmission. Ubrogepant is the first CGRP drug in tablet form to get … Continue reading “Allergan Wins FDA Nod for First Migraine Pill in New Class of Medicines”
Evotec CEO Discusses Ways to Make Drug R&D Faster and Better
Partnering and sharing data will improve the research and development process for all pharmaceutical industry stakeholders, of which there are more than ever, according to a top executive at Evotec. “Imagine that everyone would drill a hole into the ocean to dig for oil alone—it would be largely and heavily inefficient,” said Evotec CEO Werner … Continue reading “Evotec CEO Discusses Ways to Make Drug R&D Faster and Better”
Roche Puts Up Historic $1.15B for Sarepta Duchenne Gene Therapy Deal
Swiss biopharma Roche is betting more than $1 billion that Sarepta Therapeutics, which recently won FDA approval for its second RNA medicine for Duchenne muscular dystrophy, will get its lead gene therapy treatment across the finish line too. The deal, one of the biggest ever US biopharma licensing agreements, gives Roche the right outside of … Continue reading “Roche Puts Up Historic $1.15B for Sarepta Duchenne Gene Therapy Deal”
Healthcare AI Tech is Here, Now We Must Learn to Manage the Data
Pharma and software firms are actively embracing AI and machine learning, but governments may need to lead the way in accessing relevant data, says Molecular Health. Molecular Health, a software firm focused on transforming big data into analysis and prediction tools for healthcare companies, believes biotech is in the early days of using artificial intelligence … Continue reading “Healthcare AI Tech is Here, Now We Must Learn to Manage the Data”
Bio Roundup: Amarin’s Approval, FTC v. Illumina, a $120M Deal & More
As national politics dominates the news in the final days of the decade, we at Xconomy are wrapping up the year with some big moves by the FDA, including an approval of the first drug OK’d reduce heart risk in patients with high triglycerides who are already taking statins. Holidays don’t provide immunity to bad … Continue reading “Bio Roundup: Amarin’s Approval, FTC v. Illumina, a $120M Deal & More”
Biogen Strikes Alzheimer’s Deal, Paying $45M for Ionis Tau Drug Rights
When it comes to Alzheimer’s disease, Biogen isn’t putting all of its eggs in the same basket. In the latest development of a multi-drug and multi-disease collaboration, the company has licensed an experimental Ionis Pharmaceuticals compound that works in a different way than the therapies currently in its pipeline for neurodegenerative diseases. On Thursday, Ionis … Continue reading “Biogen Strikes Alzheimer’s Deal, Paying $45M for Ionis Tau Drug Rights”
Takeda Pays Turnstone $120M to Start Alliance on Viral Cancer Therapies
[Updated 12/20/19, 2:51 p.m. See below.] Turnstone Biologics has yet to test its engineered viruses in cancer patients but Takeda Pharmaceutical sees enough promise in the technology to pay $120 million up front to start a partnership developing the biotech’s lead therapeutic candidate. The Takeda (NYSE: [[ticker:TAK]]) payment is a combination of cash, near-term milestones, … Continue reading “Takeda Pays Turnstone $120M to Start Alliance on Viral Cancer Therapies”
Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options
Ring Therapeutics, a Flagship Pioneering spinout, launched Thursday with ambitious plans to expand the universe of vectors available for gene therapy delivery. Gene therapy, treatments intended to treat disease by inserting a gene instead of using drugs or surgery, has had a banner year, with the second ever such therapy approved this year in the … Continue reading “Ring Therapeutics Launches to Expand Gene Therapy Viral Vector Options”
ArcherDX Raises $55M to Help Physicians Pick Best Cancer Therapies
2020 will be a big year for ArcherDX, says its CEO on the heels of raising $55 million to advance its companion diagnostics platform—which is intended to make precision medicine an option for patients in earlier stages of disease. Headquartered in Boulder, CO, the company will use the Series C funds to expand its global … Continue reading “ArcherDX Raises $55M to Help Physicians Pick Best Cancer Therapies”
FTC Challenges PacBio Acquisition, Says Illumina is a “Monopolist”
About one year ago Illumina agreed to splash out $1.2 billion on Pacific Biosciences, a rival maker of DNA sequencing technology. Now the Federal Trade Commission is looking to block the deal, and says it was meant to steamroll a competitor and maintain Illumina’s “monopolist” position atop the industry. The San Diego-based genomics giant (NASDAQ: … Continue reading “FTC Challenges PacBio Acquisition, Says Illumina is a “Monopolist””
Zafgen’s Path Forward Is Reverse Merger With Chondrial Therapeutics
Zafgen stumbled in clinical trials testing its experimental therapies for metabolic diseases. Going forward, it will have a new name, new management, and a mitochondrial disorder drug from another company. Boston-based Zafgen (NASDAQ: [[ticker:ZFGN]]) has entered a merger agreement with Chondrial Therapeutics, a rare disease drug developer just entering the clinic. Chondrial shareholders will own … Continue reading “Zafgen’s Path Forward Is Reverse Merger With Chondrial Therapeutics”
Orbiting Organoids: Research in Space to Unveil New Neurodegeneration Insight
More than 250 miles above the Earth’s surface aboard the International Space Station, a first-in-kind study of neurodegenerative disease is expected to reveal never-before-seen cell interactions. The National Stem Cell Foundation (NSCF) is funding the study, which is the result of a bi-coastal collaboration between the New York Stem Cell Foundation (NYSCF) Research Institute and … Continue reading “Orbiting Organoids: Research in Space to Unveil New Neurodegeneration Insight”
ALS Fund Talks Investments and Strategy as it Kicks Off $100M Campaign
The ALS Investment Fund has teamed with the ALS Association and is looking to establish new partnerships as part of its efforts to raise $100 million and collate a diversified set of portfolio companies. Craig Boyce, managing partner of the ALS Investment Fund, tells Xconomy it would have been crazy to start the fund 25 … Continue reading “ALS Fund Talks Investments and Strategy as it Kicks Off $100M Campaign”
Triplet Therapeutics Unveils $59M to Try Hairpin Fix for Huntington’s
Huntington’s disease drug research has focused mainly on the genetic roots of the rare neurodegenerative disorder. Triplet Therapeutics is taking a different tack by targeting a DNA repair mechanism gone awry, an approach that CEO Nessan Bermingham says could offer a better way to treat that disease and potentially dozens of others. Cambridge, MA-based Triplet … Continue reading “Triplet Therapeutics Unveils $59M to Try Hairpin Fix for Huntington’s”
Organovo’s Demise Offers Path for Tarveda to Join the Public Markets
Organovo raised millions of dollars from investors to fuel its goal of developing 3D “bioprinted” human tissues for use in people with end-stage liver disease and other life-threatening illnesses. But the San Diego company fell short of bringing its lead regenerative medicine program into clinical trials, and now it is charting a course as a … Continue reading “Organovo’s Demise Offers Path for Tarveda to Join the Public Markets”
Wave Stock Crashes After Decision to Halt Duchenne Drug Studies
Wave Life Sciences saw its stock price plunge nearly 60 percent Monday following the company’s decision to discontinue development of its investigational Duchenne muscular dystrophy drug, suvodirsen, after an interim analysis from a Phase 1 study. Cambridge, MA-based Wave (NASDAQ: [[ticker:WVE]]) was testing suvodirsen as a potential treatment for Duchenne, a genetic neuromuscular disorder that … Continue reading “Wave Stock Crashes After Decision to Halt Duchenne Drug Studies”
Cell Reprogramming Startup Bit Bio Inks Pact With Charles River
Charles River Laboratories International is making moves in the cell therapy space after signing on the cell reprogramming startup Bit Bio and announcing plans to acquire a biomaterials company for $380 million. The Wilmington, MA-based contract research organization (CRO) last week announced that it has entered into an exclusive discovery and safety services partnership with … Continue reading “Cell Reprogramming Startup Bit Bio Inks Pact With Charles River”
Axsome’s Depression Drug Meets Phase 3 Goals, FDA Filing Planned
An experimental Axsome Therapeutics drug has succeeded in improving depression symptoms in a late-stage study, and the company is now preparing to seek regulatory approval next year. Axsome (NASDAQ: [[ticker:AXSM]]) is testing its drug, AXS-05, as a treatment for major depressive disorder (MDD). The Phase 3 study enrolled 327 patients with moderate-to-severe major depressive disorder. … Continue reading “Axsome’s Depression Drug Meets Phase 3 Goals, FDA Filing Planned”
Building a Better 3D Bioprinter: GE Healthcare and ASLS Combine Tech
GE Healthcare Life Sciences and ASLS have teamed up to offer a new platform that can design, build, and image living 3D tissue models for drug discovery and development. ASLS, Advanced Solutions Life Sciences, is a subsidiary of Advanced Solutions headquartered in Louisville, KY. Its patented Angiomics technology enables users to create 3D tissue models … Continue reading “Building a Better 3D Bioprinter: GE Healthcare and ASLS Combine Tech”
Amarin’s Fish-Oil Pill Wins FDA Nod to Cut Heart Attack, Stroke Risk
A prescription pill derived from fish-oil received the regulatory nod Friday to expand its use as a supplementary treatment, alongside statins, to more patients at risk of heart attack or stroke. The FDA approved the Amarin Pharma drug, icosapent ethyl (Vascepa), for use as a secondary treatment for adults who are taking statins and have elevated … Continue reading “Amarin’s Fish-Oil Pill Wins FDA Nod to Cut Heart Attack, Stroke Risk”
Bio Roundup: Sarepta’s Approval, Merck M&A, Drug Price Bill & More
If you’re keeping track, FDA drug approvals have been on a tear in the past month with several decisions coming well ahead of their targeted dates. And then there’s Sarepta Therapeutics. Late Thursday, the FDA announced approval of the Cambridge, MA, biotech’s drug for Duchenne muscular dystrophy in patients who have a specific mutation. The … Continue reading “Bio Roundup: Sarepta’s Approval, Merck M&A, Drug Price Bill & More”
In an About-Face, FDA Approves Second Sarepta Muscular Dystrophy Drug
Sarepta Therapeutics received an early holiday gift Thursday—approval of golodirsen (Vyondys 53), its second Duchenne muscular dystrophy treatment, which the FDA rejected in August. The agency had dinged the application over potential side effects, namely a risk of infections from the “ports” used to infuse the drug, and kidney problems observed in animals in which … Continue reading “In an About-Face, FDA Approves Second Sarepta Muscular Dystrophy Drug”
miRagen Restructures to Focus on Fibrosis, Inks $20M Stock Deal
MicroRNA therapies developer miRagen Therapeutics is scaling back work on its most advanced cancer drug candidate and shifting resources to a compound being developed to treat fibrosis in the lungs. The pipeline shuffle will be accompanied by a corporate shakeup. Boulder, CO-based miRagen (NASDAQ: [[ticker:MGEN]]) is also laying off some of its staff. The company … Continue reading “miRagen Restructures to Focus on Fibrosis, Inks $20M Stock Deal”
Amgen’s Osteoporosis Drug Wins European Nod, With a Heart Warning
An osteoporosis drug developed by Amgen now has marketing approval in Europe, but it carries a warning that the medicine can raise the risk of cardiovascular problems. Romosozumab (Evenity) treats postmenopausal woman who have osteoporosis, a disease that weakens bones, making them susceptible to fracture. The antibody drug, given as a monthly injection, was developed … Continue reading “Amgen’s Osteoporosis Drug Wins European Nod, With a Heart Warning”
Aspen Neuro Bags $6.5M to Test Parkinson’s Disease Stem Cell Therapy
Nearly nine years ago Jeanne Loring and her colleagues at Scripps Research debuted a test that leveraged advances in genomics and data science to determine, without testing in animals, whether human stem cells were “pluripotent,” or able to become any type of cell in the body. Being able to prove that has become increasingly important … Continue reading “Aspen Neuro Bags $6.5M to Test Parkinson’s Disease Stem Cell Therapy”
Iterum’s Antibiotic for Super Bugs Falls Short in Phase 3, Shares Sink
Iterum Therapeutics’ bid to bring patients a new antibiotic that’s safer than currently available drugs has fallen short of the main efficacy goal of a late-stage clinical trial. But the company is pinning its hopes on another Phase 3 study that will report data early next year. Iterum (NASDAQ: [[ticker:ITRM]]) reported preliminary Phase 3 data … Continue reading “Iterum’s Antibiotic for Super Bugs Falls Short in Phase 3, Shares Sink”
Flagship’s Cellarity Aims to Advance Cell Behavior-Based Therapeutics
The latest startup to emerge from Flagship Pioneering aims to change drug discovery by starting the process with a clear understanding of cell behavior. Cellarity—like many other startups looking to revamp the slow, costly process of developing new medicines—is using machine-learning tools to do so, leveraging computational advances to advance a new way of thinking … Continue reading “Flagship’s Cellarity Aims to Advance Cell Behavior-Based Therapeutics”
KalVista Pharma Eye Drug Partnered With Merck Flunks Phase 2 Test
A KalVista Pharmaceuticals eye drug at the center of a research collaboration with Merck has failed a mid-stage clinical trial. KalVista (NASDAQ: [[ticker:KALV]]) developed its drug, KVD001, to treat diabetic macular edema (DME), a complication of diabetes that can lead to blindness. On Monday, Cambridge, MA-based KalVista said patients treated with the drug, given as … Continue reading “KalVista Pharma Eye Drug Partnered With Merck Flunks Phase 2 Test”
Zentalis Steps Out With $147M Raised to Develop “Tried and True” Small Molecules
Zentalis Pharmaceuticals is banking on small molecules for cancer—leaving the “cool technologies” aside for a “tried and true” approach, says the CEO, who wants to let the science speak for itself. Since it was founded five years ago, the company has moved three compounds into the clinic. How? By operating under the radar. “If you … Continue reading “Zentalis Steps Out With $147M Raised to Develop “Tried and True” Small Molecules”
Scripps Research, AbbVie Expand Drug Discovery Deal Beyond Cancer
The Scripps Research Institute and AbbVie have agreed to broaden an existing drug discovery agreement focused on cancer cell therapies to now encompass treatments for other kinds of disease. Scripps’s early-stage drug discovery division, Calibr, last year inked a big partnership with North Chicago, IL-based AbbVie (NYSE: [[ticker:ABBV]]) to develop potential treatments for solid cancer … Continue reading “Scripps Research, AbbVie Expand Drug Discovery Deal Beyond Cancer”
Perceptive Unveils New $210M Fund to Invest in Early-Stage Biotechs
Perceptive Advisors, an investment firm with a long track record of life science investing across all points of development, is now stepping up to pump money into companies at the earliest stages with a new $210 million venture capital fund. The Perceptive Xontogeny Venture Fund (PXV) is led by Chris Garabedian (pictured above, center), a … Continue reading “Perceptive Unveils New $210M Fund to Invest in Early-Stage Biotechs”
Eying Cancer Combos, Sanofi to Splash Out $2.5B on San Diego’s Synthorx
Synthorx is working to design new cytokine therapies for cancer using an “extended” genetic alphabet that could help avoid the shortcomings associated with earlier versions. Now French biopharma Sanofi has struck a deal to add the startup to its oncology business in a transaction that values the company at about $2.5 billion. The move is … Continue reading “Eying Cancer Combos, Sanofi to Splash Out $2.5B on San Diego’s Synthorx”
Aurinia Plans FDA Filing After Lupus Nephritis Drug Hits Phase 3 Goals
Aurinia shares spiked last week after voclosporin, an immunosuppressant being tested in patients with lupus nephritis, met its primary and secondary endpoints in a Phase 3 study. Aurinia Pharmaceuticals (NASDAQ: [[ticker:AUPH]]) is testing its drug, voclosporin, as a treatment for lupus nephritis (LN), which can occur when lupus, an autoimmune disorder, causes the immune system … Continue reading “Aurinia Plans FDA Filing After Lupus Nephritis Drug Hits Phase 3 Goals”
Merck Makes a Big Blood Cancer Bet With $2.7B Deal for ArQule
Merck is acquiring cancer drug developer ArQule for $2.7 billion, a cash deal that brings it a promising early-stage compound that could challenge a blockbuster leukemia drug from AbbVie. According to terms of the agreement announced Monday, a Merck (NYSE: [[ticker:MRK]]) subsidiary will pay $20 for each share of ArQule (NASDAQ: [[ticker:ARQL]]), a 107 percent … Continue reading “Merck Makes a Big Blood Cancer Bet With $2.7B Deal for ArQule”
AstraZeneca Taps Gatehouse Bio to Identify Potential Drug Targets
After joining AstraZeneca’s Boston Bio Hub incubator last year, Gatehouse Bio is now teaming up with the pharma company to identify potential drug targets in respiratory and cardiovascular diseases. Under the collaboration, expected to last multiple years, Gatehouse will use its sRNAlytics platform to identify small RNA (sRNA) signatures, which the company says help in … Continue reading “AstraZeneca Taps Gatehouse Bio to Identify Potential Drug Targets”
3 Takeaways From the Resurrection of Biogen’s Alzheimer’s Drug
The name of the experimental drug was heard so frequently in hotel hallways at the conference that it sounded like an incantation: aducanumab, aducanumab, aducanumab. Regardless of the eventual FDA verdict on Biogen’s once-bagged, now-revived anti-amyloid antibody, data from clinical trials of the treatment have inarguably altered the course of drug discovery and development for … Continue reading “3 Takeaways From the Resurrection of Biogen’s Alzheimer’s Drug”
Acadia Looks to Expand Drug Beyond Parkinson’s Disease Psychosis
Acadia Pharmaceuticals revealed late-stage clinical data this week that could support bringing its drug for psychosis to a broader group of patients. The Acadia (NASDAQ: [[ticker:ACAD]]) drug pimavanserin (Nuplazid) is currently approved for treating psychosis associated with Parkinson’s disease. But the San Diego-based company has also been testing the drug in dementia-related psychosis (DRP), which … Continue reading “Acadia Looks to Expand Drug Beyond Parkinson’s Disease Psychosis”
FDA Puts Partial Hold on Tests of Ipsen Drug Acquired in $1B Deal
Two clinical trials testing an Ipsen drug in rare connective tissue disorders have been placed on partial hold after safety concerns about chronic dosing of the compound emerged. The partial hold applies to patients under 14 years of age in Phase 2 and Phase 3 tests of the drug, palovarotene. But the Paris-based company, which … Continue reading “FDA Puts Partial Hold on Tests of Ipsen Drug Acquired in $1B Deal”
Bio Roundup: Biogen at CTAD, Sage’s Stumble, Astellas’s $3B Offer & More
It’s been a roller-coaster year for those hoping for new drugs to treat Alzheimer’s disease, a leading cause of death with rising treatment costs and no clear answer yet as to its cause. Clearing proteins, such as amyloid and tau, was once seen as the top contender for stopping or slowing neurodegeneration. Costly failures forced … Continue reading “Bio Roundup: Biogen at CTAD, Sage’s Stumble, Astellas’s $3B Offer & More”
Biogen Points to Enrollment and Dosing to Explain Alzheimer’s Results
Biogen revealed detailed results from its Alzheimer’s trial to a jam-packed room of eager conference attendees this week—and while some described it as a major advance in the field, others were less convinced. Samantha Budd Haeberlein, Biogen’s vice president of clinical development, took the stage Thursday morning in San Diego at Clinical Trials on Alzheimer’s … Continue reading “Biogen Points to Enrollment and Dosing to Explain Alzheimer’s Results”
FDA Rejects Enzyvant’s Regenerative Therapy for Rare Immune Disorder
[Corrected 12:27 p.m. See below.] The FDA has rejected an experimental regenerative therapy that Enzyvant developed to restore immune system function in babies born with a rare disorder. The agency cited manufacturing issues, according to the company. Enzyvant received the notification, formally known as a complete response letter, on Wednesday, CEO Rachelle Jacques tells Xconomy. … Continue reading “FDA Rejects Enzyvant’s Regenerative Therapy for Rare Immune Disorder”
Sage Therapeutics Depression Drug Fails in Phase 3, Shares Fall 60%
An experimental Sage Therapeutics drug intended to alleviate depression symptoms quickly failed to beat a placebo in a pivotal study, the company reported Thursday. Cambridge, MA-based Sage (NASDAQ: [[ticker:SAGE]]) has been testing its drug, SAGE-217, as a treatment for the symptoms of major depressive disorder. Unlike many depression drugs, which need weeks to take effect … Continue reading “Sage Therapeutics Depression Drug Fails in Phase 3, Shares Fall 60%”
As Alzheimer’s Conference Kicks Off, All Eyes Are on Biogen’s Drug
It’s the “era of neuroscience”; an industry “turning point”; and “an amazing time”—superlatives were flowing freely as the 12th annual Clinical Trials on Alzheimer’s Disease congress (CTAD) kicked off in rainy San Diego late Wednesday afternoon. The atmosphere felt thick with anticipation as attendees massed in the downtown hotel where the conference is taking place, … Continue reading “As Alzheimer’s Conference Kicks Off, All Eyes Are on Biogen’s Drug”
6 Experimental Alzheimer’s Disease Diagnostics Projects Get Funding Boost
Scientists gathered this week in San Diego at the annual Clinical Trials on Alzheimer’s Disease Congress are focused on ways to treat the neurodegenerative disease, a task that continues to vex those working to develop potential medicines. Others are working on an intimately related problem: early detection of the disease and related dementias. On Wednesday … Continue reading “6 Experimental Alzheimer’s Disease Diagnostics Projects Get Funding Boost”
Civetta Therapeutics Unveils a Propeller Protein Plan, $53M in Funding
New biotech company Civetta Therapeutics has closed a $53 million Series A financing round to develop drugs that target “propeller proteins” as a way of treating cancers and other diseases. Propeller proteins are found throughout nature, and the circularly arranged “blades” of these proteins commonly serve as active sites to which molecules can bind. Bruce … Continue reading “Civetta Therapeutics Unveils a Propeller Protein Plan, $53M in Funding”
FDA Green Lights Sight Diagnostics’ Finger-Prick Blood Test
Sight Diagnostics today announced it has received US FDA 510(k) clearance to use its patented OLO analyzer, which performs a complete blood count using two drops of blood. The FDA clearance gives hospital-run laboratories, diagnostic providers, and outpatient clinics the go-ahead to use the device, which captures around 1,000 images that are then interpreted by … Continue reading “FDA Green Lights Sight Diagnostics’ Finger-Prick Blood Test”
Digital Health Survivors & Innovative Incumbents Must Team Up for True Disruption
Editor’s note: This is Part 2 of a two-part post about innovation in digital health, co-authored by Rob Coppedge, CEO of Echo Health Ventures. Read Part 1 here. The Path Forward Between Echo Health Ventures and Blue Cross and Blue Shield of North Carolina, we work deeply on both sides of these partnerships and have perspective … Continue reading “Digital Health Survivors & Innovative Incumbents Must Team Up for True Disruption”
Roche Notches FDA Nod in Lung Cancer, Amps Up Competition With Merck
A Roche immunotherapy has won an additional FDA approval as a first-line treatment for non-small lung cancer, bolstering the product’s competitive stance versus one from Merck. The FDA approved atezolizumab (Tecentriq) for adults whose non-squamous non-small cell lung cancer (NSCLC) is metastatic, meaning it has spread, and does not have the EGFR or ALK genetic … Continue reading “Roche Notches FDA Nod in Lung Cancer, Amps Up Competition With Merck”