Adicet Aims to Advance CAR-T Therapies in Reverse Merger with resTORbio

Adicet Bio, a privately held preclinical biotech looking to move ahead its “off-the-shelf” CAR-T cell therapies, has struck a deal with struggling resTORbio to use its Nasdaq listing as a backdoor to the public markets. The companies on Wednesday announced an agreement to combine in a type of transaction sometimes referred to as a “reverse … Continue reading “Adicet Aims to Advance CAR-T Therapies in Reverse Merger with resTORbio”

Supernus Makes a Parkinson’s Play With $300M Deal for US WorldMeds Drugs

Supernus Pharmaceuticals is expanding its central nervous system (CNS) drug lineup through a deal to acquire four US WorldMeds products with applications in Parkinson’s disease. The products, three of them already on the market and one in late-stage development, comprise the CNS portfolio of Louisville, KY-based US WorldMeds. According to terms of the deal, Supernus … Continue reading “Supernus Makes a Parkinson’s Play With $300M Deal for US WorldMeds Drugs”

Taysha Launches With $30M to Bring Gene Therapy to CNS Disorders

Some former executives and investors behind the second gene therapy to win FDA approval are teaming up again. Their new company, Taysha Gene Therapies, is launching with $30 million in the bank and a plan to bring its first central nervous system (CNS) disorder program into human testing later this year. Taysha is focusing on … Continue reading “Taysha Launches With $30M to Bring Gene Therapy to CNS Disorders”

Aurinia Taps Cerecor’s Miller to Succeed Retiring Chief Financial Officer

Aurinia Pharmaceuticals (NASDAQ: [[ticker:AUPH]]) announced that Joe Miller would become its new chief financial officer, succeeding Dennis Bourgeault, who is retiring after more than 20 years as its CFO. Miller was most recently CFO and corporate secretary at Cerecor (NASDAQ: [[ticker:CERC]]). His prior experience includes roles at Sucampo Pharmaceuticals, which merged with Mallinckrodt (NYSE: [[ticker:MNK]]) in … Continue reading “Aurinia Taps Cerecor’s Miller to Succeed Retiring Chief Financial Officer”

At Rome Therapeutics, Kapeller Aims to Turn “Junk” Into New Drugs

A new biotech, Rome Therapeutics, launched Monday with $50 million and a plan to develop novel drugs based on new insights into repeats in the non-coding stretches of the human genome. Rome’s debut pulls back the curtain on what Rosana Kapeller, the company’s co-founder and CEO, has been up to since she left Nimbus Therapeutics … Continue reading “At Rome Therapeutics, Kapeller Aims to Turn “Junk” Into New Drugs”

Neurocrine Gets FDA Nod for Once-Daily “Add-On” Parkinson’s Pill

Many patients with Parkinson’s disease take a combination medication called levodopa/carbidopa to control their symptoms, but over time, experience “off” episodes in which the tremor, slowed movement, and difficulty walking that characterize the disorder occur. Now the FDA has approved a Neurocrine Biosciences (NASDAQ: [[ticker:NBIX]]) drug intended to be taken along with levodopa/carbidopa to extend … Continue reading “Neurocrine Gets FDA Nod for Once-Daily “Add-On” Parkinson’s Pill”

Erasca Lands $200M Mega-Round to Move Cancer Programs to the Clinic

Serial biotech entrepreneur Jonathan Lim has raised a whopping $200 million to advance multiple candidates at his latest cancer drug development venture, Erasca, into human testing. Lim (pictured) sold his previous company, Ignyta, to Roche for $1.7 billion. On Monday Erasca—a portmanteau of “erase” and “cancer”—announced it had completed a Series B financing round jointly … Continue reading “Erasca Lands $200M Mega-Round to Move Cancer Programs to the Clinic”

AgenTus VP Mark Exley Appointed Chief Scientific Officer of Imvax

Imvax announced that Mark Exley has joined the biotech as its chief scientific officer. Exley comes to Philadelphia-based Imvax from AgenTus Therapeutics, where he was vice president of cellular immunology. His experience also includes faculty positions at Harvard Medical School and the University of Manchester. Imvax’s lead candidate, a tumor cell vaccine called IGV-001, is … Continue reading “AgenTus VP Mark Exley Appointed Chief Scientific Officer of Imvax”

ADC Therapeutics Resubmits IPO to Fuel Cancer Drug Filing, Launch Plans

Last fall, ADC Therapeutics (ADCT) concluded the time just wasn’t right for an IPO. Six months later, with its lead drug in a pivotal test and plans being drawn up for a regulatory submission and potential commercialization, the cancer drug developer has determined it needs cash now. ADCT refiled its IPO plans late Friday. The … Continue reading “ADC Therapeutics Resubmits IPO to Fuel Cancer Drug Filing, Launch Plans”

Bio Roundup: Fast FDA Approvals, Biogen Delays, ORIC’s IPO & More

COVID-19’s spread has clamped down on a lot of economic activity, including the work of developing and testing new medicines. But the FDA isn’t showing signs of slowing down, at least when it comes to drugs filed for accelerated approval. The regulator signed off on three such drugs in the past week. These approvals are … Continue reading “Bio Roundup: Fast FDA Approvals, Biogen Delays, ORIC’s IPO & More”

Drug Review Times Not Shortened Much by Single Expedited Review Designation

Expedited programs usually shorten a US Food and Drug Administration new drug application assessment, but on average, maybe not as much as their reputation would suggest. A Government Accountability Office (GAO) analysis found that expedited assessment programs generally are significant variables affecting assessment times, but less so than perhaps stakeholders might expect. When all else is equal, … Continue reading “Drug Review Times Not Shortened Much by Single Expedited Review Designation”

ORIC Pharma Gets $120M in IPO to Advance Drug-Resistant Cancer Therapies

ORIC Pharmaceuticals followed in the footsteps of the other biotechs that have managed to go public this month, selling more shares than it expected for a haul of $120 million. The South San Francisco, CA-based company—whose name is an acronym for “overcoming resistance in cancer”—priced its IPO late Thursday, offering 7.5 million shares at $16 … Continue reading “ORIC Pharma Gets $120M in IPO to Advance Drug-Resistant Cancer Therapies”

Nitrome Bio Bags $38M to Prep Parkinson’s Program for the Clinic

Nitrome Biosciences is looking to leverage its research into a class of enzymes involved in the aggregation of a protein characteristic of Parkinson’s disease into a treatment for that and other age-related diseases. This week the San Francisco-based startup raised $38 million, a Series A financing that it anticipates will allow it to ready its … Continue reading “Nitrome Bio Bags $38M to Prep Parkinson’s Program for the Clinic”

Affinivax Lands $120M to Take MAPS Tech Into New Vaccine Territory

Affinivax has a lead program with early clinical data showing the potential to best a pharmaceutical giant’s blockbuster vaccine. The startup now has $120 million in financing to see just how much further its technology can go. The Series B round of funding announced Thursday was led by Viking Global Investors. Cambridge, MA-based Affinivax develops … Continue reading “Affinivax Lands $120M to Take MAPS Tech Into New Vaccine Territory”

Life Science a VC Bright Spot in Q1, But Startups May Soon Struggle

Even as the novel coronavirus has derailed daily life and business operations, the life sciences sector continues to see companies make public debuts and ink both financing and partnership deals. In venture capital, US deal activity in the first quarter tallied 2,300 financings totaling $34.2 billion, according to the latest report by the National Venture … Continue reading “Life Science a VC Bright Spot in Q1, But Startups May Soon Struggle”

Biogen Pushes Finalized Filing for Alzheimer’s Drug to Third Quarter

Biogen last year said it would ask the FDA to review its potential Alzheimer’s disease treatment, aducanamab, sometime in “early” 2020. The submission is being made under a rolling review, in which sections of an application are filed as they are completed rather than all at once. Now the Cambridge, MA-based company says the regulatory … Continue reading “Biogen Pushes Finalized Filing for Alzheimer’s Drug to Third Quarter”

Immunomedics Triple Negative Breast Cancer Drug Wins Early FDA Nod

An Immunomedics therapy for an aggressive form of breast cancer won FDA approval on Wednesday. It’s the company’s first regulatory nod, and it covers a product expected to become a blockbuster seller. Morris Plains, NJ-based Immunomedics (NASDAQ: [[ticker:IMMU]]) developed the drug, sacituzumab govitecan (Trodelvy), as a treatment for treat triple negative breast cancer. This form … Continue reading “Immunomedics Triple Negative Breast Cancer Drug Wins Early FDA Nod”

Synthorx’s Shawver Joins Silverback Therapeutics as New Top Exec

Veteran biotech executive Laura Shawver is taking over as CEO at Silverback Therapeutics, joining the cancer drug developer as it is poised to move its first compound into the clinic. The Wednesday announcement comes three months after French firm Sanofi (NYSE: [[ticker:SNY]]) closed its $2.5 billion acquisition of La Jolla, CA-based Synthorx, the biotech Shawver … Continue reading “Synthorx’s Shawver Joins Silverback Therapeutics as New Top Exec”

Why 2020 Is Pivotal for Microbiome Drug R&D (Or Maybe 2021, Given COVID-19)

The start of a potentially big biomedical breakthrough is blooming within all of us. Drug research aiming to turn bacteria into microbiome therapies now spans programs aiming to address gut disorders, autism, cancer, and more. Given the number of late-stage and mid-stage studies expected to report data in 2020—assuming they stay on track amid the … Continue reading “Why 2020 Is Pivotal for Microbiome Drug R&D (Or Maybe 2021, Given COVID-19)”

BARDA Director Rick Bright Moves to NIH to Head COVID-19 Testing R&D

Rick Bright, who joined the Biomedical Advanced Research and Development Authority (BARDA) in 2010, has suddenly left his role as director of the federal agency, which is playing a key role in advancing medical countermeasures against COVID-19. BARDA formed in 2006; Bright (pictured) succeeded founding director Robin Robinson to the position in late 2016. The … Continue reading “BARDA Director Rick Bright Moves to NIH to Head COVID-19 Testing R&D”

Cara Therapeutics Itching Drug Scratches Off Goals of Phase 3 Test

A Cara Therapeutics drug developed to treat the severe itching experienced by patients receiving hemodialysis has met the main goal of a second late-stage study, setting up the experimental medicine for regulatory filings in the US and Europe. Cara (NASDAQ: [[ticker:CARA]]) is testing its injectable drug, difelikefalin (Korsuva), in patients who have moderate-to-severe chronic kidney … Continue reading “Cara Therapeutics Itching Drug Scratches Off Goals of Phase 3 Test”

New COVID-19 Special Reports Explore Clinical Research During the Pandemic

Two new special reports on Xconomy Insight provide an expert view on how companies can sustain clinical trials during the COVID-19 crisis. “Decentralized Clinical Trials: The Call for a New Paradigm” and “Sustaining Clinical Research During the Crisis” offer the latest trends and technologies companies can use to keep business running as smooth as possible. … Continue reading “New COVID-19 Special Reports Explore Clinical Research During the Pandemic”

War Paint to Bone Paint: MIT Spinout Aims to Transform Regenerative Meds

Driven by 20 years in the military, the founder of Theradaptive hopes his firm’s regenerative “paint” technology platform can transform bone repair, spinal fusion, and sports medicine. The firm, spun out of MIT, has a computational platform it says turns recombinant proteins into material-binding variants that behave like a paint. This can then be used … Continue reading “War Paint to Bone Paint: MIT Spinout Aims to Transform Regenerative Meds”

Unlearn Adds $12M for “Digital Twins” That Accelerate Clinical Trials

Speeding up and bringing down the cost of clinical trials would be a huge boon to pharma companies and patients alike. San Francisco-based startup Unlearn.AI says it has developed proprietary technology that may help accomplish just that. On Monday the company announced it raised $12 million from investors who believe it stands out among the … Continue reading “Unlearn Adds $12M for “Digital Twins” That Accelerate Clinical Trials”

FDA Approves Incyte Drug, First Ever for Rare Bile Duct Cancer

The FDA has granted speedy approval for an Incyte drug developed to treat cholangiocarcinoma, a rare cancer affecting the bile ducts. The regulatory nod for the drug, pemigatinib (Pemazyre), comes more than a month earlier than the anticipated May 30 decision date and it makes the pill the first FDA-approved treatment for cholangiocarcinoma. The decision … Continue reading “FDA Approves Incyte Drug, First Ever for Rare Bile Duct Cancer”

Seattle Genetics Gets Early FDA Nod for Breast Cancer Combo Drug

Seattle Genetics on Friday received its third FDA approval, a regulatory nod for a breast cancer drug for patients with an especially aggressive form of the disease, four months ahead of schedule. The Bothell, WA-based biotech earned the earlier-than-expected decision for its oral drug tucatinib (Tukysa), when taken in combination with the chemotherapies trastuzumab and … Continue reading “Seattle Genetics Gets Early FDA Nod for Breast Cancer Combo Drug”

COVID-19 Vax Efforts Aim to Balance Speed, Efficacy in Race to Market

If Peter Marks had a magic wand to perfect the sophisticated process that takes a new vaccine from idea to reality, the US could have a way to prevent some people from COVID-19 infection in nine months to one year. In the real world, the journey to a vaccine OK’d under emergency guidelines is more … Continue reading “COVID-19 Vax Efforts Aim to Balance Speed, Efficacy in Race to Market”

Lantern Pharma IPO File Illuminates Plan to “Rescue” Failed Cancer Drugs

A growing number of drug developers are embracing artificial intelligence as a tool for finding new medicines. Lantern Pharma is betting the technology can also resurrect failed cancer drugs and it is now preparing an initial public stock offering to support their development. Lantern filed its IPO paperwork late Thursday. The Dallas-based company set a … Continue reading “Lantern Pharma IPO File Illuminates Plan to “Rescue” Failed Cancer Drugs”

Bio Roundup: Alnylam’s Billions, COVID-19 Vaccines, Moma Debuts & More

The public markets are moving erratically, but Alnylam Pharmaceuticals showed this week that selling equity isn’t the only way for biotech companies to raise cash. Late last year Alnylam (NASDAQ: [[ticker:ALNY]]) began exploring potential royalty deals for inclisiran, a drug that uses RNA interference to lower cholesterol. Alnylam had sold the drug’s development and commercialization … Continue reading “Bio Roundup: Alnylam’s Billions, COVID-19 Vaccines, Moma Debuts & More”

Neurology at Forefront of FDA’s Complex Innovative Trial Design Pilot

The coronavirus pandemic has frozen many clinical trials and forced others to adapt in the face of overwhelmed healthcare systems around the world. When activity resumes, trials could look different, with more remote monitoring and decentralized enrollment. They could reflect some of the design evolutions that the US Food and Drug Administration and sponsors have … Continue reading “Neurology at Forefront of FDA’s Complex Innovative Trial Design Pilot”

RDMD Lands $14M to Expand Reach of Rare Disease Research Platform

Thousands of diseases occur so infrequently that few data are readily available about the patients who have them. RDMD, which launched in 2018 to gather such data to spur more drug research for those patients, has raised $14 million to add 20 more diseases to its platform. The San Francisco-based startup operates a two-sided network. … Continue reading “RDMD Lands $14M to Expand Reach of Rare Disease Research Platform”

UroGen Gets FDA Nod for Low-Grade Upper Tract Urothelial Cancer Drug

The FDA on Wednesday approved a drug developed by UroGen Pharma to treat low-grade upper tract urothelial cancer, which rarely spreads but often recurs and can cause kidney damage in some patients. Urothelial cancer is the most common type of bladder cancer. Upper tract urothelial cancer (UCTC), however, affects the lining of the urinary system, … Continue reading “UroGen Gets FDA Nod for Low-Grade Upper Tract Urothelial Cancer Drug”

With Harvard Tech and $5M, PIC Therapeutics Takes Aim at Cancer

Some of the targets in cancer research are visible and well known, but they’re hard to hit with new drugs all the same. PIC Therapeutics is developing medicines aiming for one particularly elusive group of proteins, and the startup’s early research has attracted financial support from some prominent names in the life sciences industry. PIC … Continue reading “With Harvard Tech and $5M, PIC Therapeutics Takes Aim at Cancer”

Cerevance Raises $45M to Advance Brain Disease Research & Development

Cerevance, a biotech focused on developing new treatments for brain diseases, has added $45 million to its coffers in a Series B financing round from investors including the venture capital arm of Google parent company Alphabet and billionaire philanthropist Bill Gates. On Tuesday the Boston-based company said the money would allow it to continue identifying … Continue reading “Cerevance Raises $45M to Advance Brain Disease Research & Development”

GSK and Sanofi Team Up on COVID-19 Vaccine, Aim for Clinic This Year

[Updated, 3:13 p.m. See below.] Pharmaceutical giants GlaxoSmithKline and Sanofi are teaming up on COVID-19 research, aiming to combine their respective technologies in a new vaccine that could start clinical trials in coming months. The full details of the agreement, which was announced Tuesday, are still being finalized. But the companies say the vaccine would … Continue reading “GSK and Sanofi Team Up on COVID-19 Vaccine, Aim for Clinic This Year”

Arcturus Poised to Move Liver Drug, COVID-19 Vaccine Into Clinic

Messenger RNA drug developer Arcturus Therapeutics is ready to kick off its first-ever human trials this year, beginning clinical evaluation of its lead drug candidate and testing a vaccine designed to target the novel coronavirus. The San Diego biotech on Monday announced it had received the FDA’s OK to enter into the clinic with ARCT-810, … Continue reading “Arcturus Poised to Move Liver Drug, COVID-19 Vaccine Into Clinic”

Japan’s Kyowa Kirin Pays MEI Pharma $100M in Blood Cancer Drug Pact

MEI Pharma is expanding a 2018 deal with Japanese biopharma Kyowa Kirin giving the company the development and commercialization rights to one of its investigational blood cancer drugs in that country. Kyowa has agreed to pay $100 million up front for rights to MEI-401 outside of the United States, and to jointly develop and—if approved—promote … Continue reading “Japan’s Kyowa Kirin Pays MEI Pharma $100M in Blood Cancer Drug Pact”

More Trust, Collaboration in Pharma Needed to Reap Benefits of Data Science

Limited data isn’t an issue when it comes to how to best use computational tools to better prevent disease and treat patients—but how to more efficiently analyze existing information is. Industry veterans with experience in life sciences and data science floated their ideas about how the pharmaceutical industry could better plug in to the “big … Continue reading “More Trust, Collaboration in Pharma Needed to Reap Benefits of Data Science”

Blackstone Pays Alnylam $1B to Grab a Stake in RNAi Drug Inclisiran

Seven years ago Alnylam Pharmaceuticals sold rights to one of its preclinical drugs, gaining upfront cash and $180 million in potential milestone payments, plus royalties, for a therapy taking a novel approach to lowering cholesterol. On Monday, the company announced the sale of half of the royalties it’s owed to private equity firm Blackstone for … Continue reading “Blackstone Pays Alnylam $1B to Grab a Stake in RNAi Drug Inclisiran”

AstraZeneca Wins First FDA Drug Nod for NF1, a Rare Genetic Disorder

An AstraZeneca drug designed to block tumor growth is now approved as a treatment for neurofibromatosis type 1 (NF1), a rare inherited disorder that leads to tumors on nerves throughout the body. The FDA’s Friday decision for the drug, selumetinib (Koselugo), makes it the first approved treatment for NF1. It’s also the latest twist in … Continue reading “AstraZeneca Wins First FDA Drug Nod for NF1, a Rare Genetic Disorder”

Akero Heads to Top of Class in NASH; Genfit Says Pandemic Won’t Delay Data

Akero’s FGF21 analog yields 63 percent to 72 percent relative hepatic fat reduction in Phase 2a study; the company awaits biopsy data hoping to see a fibrotic benefit. Also, Genfit says COVID-19 pandemic should not significantly delay its Phase 3 NASH readout. FGF21 analogs comprise one of the more crowded classes in nonalcoholic steatohepatitis (NASH) … Continue reading “Akero Heads to Top of Class in NASH; Genfit Says Pandemic Won’t Delay Data”

Biotech Roundup: GSK’s Ante, Shakeup at Sage, Telehealth’s Rise & More

The COVID-19 pandemic has stopped enrollment in many clinical trials and postponed the start of others but there is still life in the life sciences sector. The coronavirus research is expected. It’s the other activity that might come as a surprise. Two biotech companies have gone public in the past week; each upsized its offering and … Continue reading “Biotech Roundup: GSK’s Ante, Shakeup at Sage, Telehealth’s Rise & More”

Pfizer, BioNTech Say mRNA COVID-19 Vaccine Trials May Start This Month

Multiple COVID-19 vaccine candidates that use a new technology called messenger RNA are now being readied for clinical trials that Pfizer and BioNTech plan to start in coming weeks. The partnership pairs the mRNA vaccine technology of BioNTech (NASDAQ: [[ticker:BNTX]]) with the vaccine and clinical trial experience of Pfizer (NYSE: [[ticker:PFE]]). The companies said Thursday … Continue reading “Pfizer, BioNTech Say mRNA COVID-19 Vaccine Trials May Start This Month”

Tango Adds $60M in CRISPR Hunt for Synthetically Lethal Cancer Drugs

CRISPR is key to the cancer drugs that Tango Therapeutics is developing, but the startup isn’t out to edit disease-causing genes. Tango uses the technology as a tool for finding drug targets, in research that has yielded a pipeline of compounds. Now it has $60 million to continue that work. The Series B round of … Continue reading “Tango Adds $60M in CRISPR Hunt for Synthetically Lethal Cancer Drugs”

Investors Bet on Next-Gen Telehealth as COVID-19 Fuels Adoption

Talking to a healthcare provider from the safety of one’s own home make more sense than ever today. Tyto Care offers a way to do so, plus a device that allows consumers to perform exams and transmit video, images, and sounds to their provider. This week the telehealth company doubled its outside investment to fuel … Continue reading “Investors Bet on Next-Gen Telehealth as COVID-19 Fuels Adoption”

Gilead Heightens Inflammatory Disease Focus With Second Genome Microbiome Deal

Gilead Sciences has signed an agreement with South San Francisco biotech Second Genome to use its microbiome drug development expertise to advance the compounds it is evaluating in inflammatory disease and to identify new ones. “There is a growing body of evidence that the microbiome plays an important role in disease progression and treatment response … Continue reading “Gilead Heightens Inflammatory Disease Focus With Second Genome Microbiome Deal”

Organovo Future Unclear After Shareholders Reject Tarveda Merger

A planned merger between struggling 3-D “bioprinter” Organovo and Tarveda Therapeutics, a privately held cancer drug developer looking to join the public markets through a reverse merger, has fallen through. Organovo (NASDAQ: [[ticker:ONVO]]) management announced in mid-December that its board of directors and Tarveda’s had approved the companies’ combination. But Organovo shareholders, who under the … Continue reading “Organovo Future Unclear After Shareholders Reject Tarveda Merger”

Roche Pays $190M to Start RNA Drug Alliance With Arrakis Therapeutics

Some diseases have proven difficult to address using conventional tools of drug discovery that target disease-causing proteins. Roche is placing a $190 million bet on Arrakis Therapeutics, a startup whose technology aims for RNA—carriers of the genetic instructions that make these proteins in the first place. The cash, an upfront payment to Waltham, MA-based Arrakis, … Continue reading “Roche Pays $190M to Start RNA Drug Alliance With Arrakis Therapeutics”

Keros Therapeutics Raises $96M in Upsized IPO to Fuel Clinical Tests

The financial markets may remain volatile but Keros Therapeutics (NASDAQ: [[ticker:KROS]]) managed to raise more money in its public market debut than it had initially planned. Late Tuesday, the Lexington, MA-based company offered 6 million shares for $16 apiece, the high end of its projected price range. Keros had previously planned to offer 5 million … Continue reading “Keros Therapeutics Raises $96M in Upsized IPO to Fuel Clinical Tests”

Inovio Begins Experimental COVID-19 Vaccine Tests in Healthy Volunteers

Inovio Pharmaceuticals, one of the biotechs developing a vaccine to prevent COVID-19, has received the FDA’s OK to start testing its candidate in healthy volunteers. The Plymouth Meeting, PA-based company, which also conducts research in San Diego, plans to enroll as many as 40 adults across two trial locations. Inovio (NASDAQ: [[ticker:INO]]) said Monday it … Continue reading “Inovio Begins Experimental COVID-19 Vaccine Tests in Healthy Volunteers”