The FDA issued a warning letter to Kaleido Biosciences alleging the company tested its experimental microbiome product in Covid-19 patients without submitting a formal clinical trial application. Kaleido claims the filing was not required because the product is a medical food, but the regulator doesn’t buy that argument.
A Florida orthopedic surgeon and designer of costly spinal surgery implants was arrested Tuesday and charged with paying millions of dollars in kickbacks and bribes to surgeons who agreed to use his company’s devices. Dr. Kingsley R. Chin, 57, of Fort Lauderdale, Florida, is the founder, chief executive officer and owner of SpineFrontier, a device …
A Roche drug that failed as a treatment for neurological disorders is now the lead program for Disc Medicines. CEO John Quisel said Roche’s clinical data showed the small molecule’s promise addressing a rare blood disorder and now the biotech startup has $90 million to advance that drug and another one into Phase 2 testing.
Atavistik Bio is one of several companies discovering and developing drugs that work by allostery, binding to less obvious sites of a target protein. Acting CEO John Josey said the startup aims to stand apart with its focus on understanding metabolic interactions, a path less trodden by others in allosteric drug discovery.
Though Gilead Sciences leads the group of companies developing immunotherapies that target the cancer protein CD47, Pfizer contends that molecules it is getting through the Trillium Therapeutics acquisition have safety and efficacy advantages that could make them the best in this emerging drug class.
For much of this summer, Sesen Bio said all of the FDA’s communications pointed toward regulatory approval for the biotech’s bladder cancer drug. Though Sesen characterized the regulator’s rejection of the fusion protein as surprising, CEO Tom Cannell also revealed an earlier sign that another clinical trial may be necessary.
The artificial intelligence-based technology of Iterative Scopes brings computer vision analysis to endoscopic images. The startup’s technology was initially developed to assist gastroenterologists in finding pre-cancerous polyps but CEO and founder Jonathan Ng said it’s also finding additional use helping pharmaceutical companies identify patients for clinical trials.
Patients taking new Alzheimer’s disease drug Aduhelm will be evaluated in a real-world study lasting up to five years. Biogen announced plans for the observational study, which is one of three post-marketing clinical programs planned to generate more data about the drug
The engineered viruses used to deliver gene therapies can spark complications and they can’t be re-dosed. Ring Therapeutics says viruses that evolved with humans can be better viral vectors and the startup has raised $117 million in new financing to continue its research.
Seres Therapeutics’ ambitions to treat ulcerative colitis with a microbiome drug were dealt a setback as its experimental therapy failed in Phase 2 testing. The company plans a “rigorous scientific analysis” to assess what happened and to determine the next steps for the program.
An amyotrophic lateral sclerosis drug from Amylyx Pharmaceuticals is being prepared for Phase 3 clinical testing on track to begin later this quarter. To support that research, the biotech has raised $135 million in financing.
Startup PAQ Therapeutics is developing drugs work like Pac Man, gobbling up components of a cell associated with disease. The biotech has closed $30 million in financing to continue its research, with a neurodegenerative disorder as its lead disease target.
By paying $125 million up front for rights to multiple sclerosis drug candidate orelabrutinib, Biogen secures a place among those companies trying to treat the disease by targeting a particular enzyme highly expressed in the central nervous system. But Biogen still has some catching up to do.
A hemophilia A patient in a Sigilon Therapeutics clinical trial developed a well-known complication to treatments for the blood disorder. The potential problem for Sigilon is that its cell therapy is designed to avoid triggering such immune responses.
The states, including Massachusetts and New York, agreed to drop opposition to the bankruptcy organization plan of the company, the maker of OxyContin.
The drug label for Biogen Alzheimer’s disease drug Aduhelm has been revised to clarify that its use is for patients with mild forms of the neurodegenerative disorder. The change aligns the prescribing information with the patient population studied in clinical trials.
July Fourth was not the celebration President Joe Biden had hoped for, as far as protecting more Americans with a coronavirus vaccine. The nation fell just short of the White House’s goal to give at least a first dose to 70% of adults by Independence Day. By that day, 67% of adult Americans had gotten …
Four years after a private equity acquisition took Parexel private, the contract research organization is being acquired by two private equity firms for $8.5 billion. It’s the latest in a series of acquisitions to hit the CRO sector this year.
Fresh off its first FDA approval, Apellis Pharmaceuticals is teaming up with Beam Therapeutics, a partnership that will use Beam’s base-editing technology to develop new therapies for complement system disorders. Apellis has committed to pay Beam $75 million to kick off the alliance.
Intellia Therapeutics has early clinical trial data showing that its CRISPR-based therapy for a rare disease can edit genes inside the body safely and effectively. The trial is ongoing, but the preliminary results suggest that Intellia’s approach could offer a cure.
Lyell Immunopharma and Verve Therapeutics are still preclinical, but they’re bring novel genetic approaches to the treatment of cancer and cardiovascular disease. Now the two companies can claim two of the biggest biotech IPOs of 2021 so far.
CAMP4 Therapeutics is developing a new kind of RNA therapy that treats disease by upregulating gene expression. The startup has raised $45 million as CEO Josh Mandel-Brehm steers toward clinical trials expected to begin next year in liver and brain diseases.
A Sage Therapeutics depression drug that failed a pivotal study in 2019 has positive preliminary results from a new Phase 3 clinical trial, but the data also showed the therapy’s effects waned over time. Questions about zuranolone’s durability are relevant to Biogen, which committed $1.5 billion to Sage in large part due to the drug’s …
Senda Biosciences is developing drugs based on an understanding of intersystems biology—the way that humans interact with bacteria and plants. The startup, founded by Flagship Pioneering, has added $55 million to advance its three lead programs to clinical testing next year.
Valo Health is going public in a SPAC merger. Less than nine months removed from its formal launch, Valo is now revealing more about its artificial intelligence platform technology, which it plans to use to make the firm the first “digitally native” pharmaceutical company.
Biogen is still discussing with the FDA the clinical trial design to confirm the benefit offered by its newly approved Alzheimer’s disease drug, Aduhelm. According to a timeline set by the regulator, that clinical trial is not expected to be complete until 2029.
A Biogen Alzheimer’s drug that clears amyloid plaque from the brain has been granted accelerated approved by the FDA. The drug, which will be marketed as Aduhelm, is the first new FDA-approved Alzheimer’s treatment since 2003.
MorphoSys is acquiring Constellation Pharmaceuticals and its late-stage myelofibrosis drug in a deal that values the epigenetics biotech at $1.7 billion. Germany-based MorphoSys will finance the acquisition with cash from a separate $2 billion deal with drug royalty acquirer Royalty Pharma.
Moderna released preliminary clinical data showing its Covid-19 vaccine produced efficacy in adolescents that was comparable to what was seen in adults. The company plans to submit applications to regulators in early June seeking expansion of emergency authorization of the vaccine to include this younger age group.
Biogen is reaching across the Charles River, striking up a gene therapy manufacturing alliance with synthetic biology firm Ginkgo Bioworks. Ginkgo will use its technology to accelerate and make more efficient the process of manufacturing the AAV viral vectors used in gene therapies.
BlueRock Therapeutics, a Bayer subsidiary, is teaming up with Opsis Therapeutics and Fujifilm Cellular Dynamics in an R&D pact focused on developing stem cell therapies for eye diseases. BlueRock is paying its new partners $30 million up front to kick off the alliance.
Bristol Myers Squibb already has a cancer program targeting the TIGIT protein, but in acquiring rights to Agenus’s drug, the pharma giant adds a bispecific antibody that preclinical research suggests has the potential to be the best in this drug class. BMS agreed to pay $200 million up front for global rights.
The FDA approved Apellis Pharmaceuticals drug pegcetacoplan (Empaveli) as a treatment for a rare blood disorder that is currently treated with medicines sold by Alexion Pharmaceuticals, a biotech being acquired by AstraZeneca. But Apellis’s drug beat Alexion drug Soliris in a head-to-head test, and the smaller company aims to steal market share from its larger …
A Biogen gene therapy for a rare, inherited eye disorder that leads to blindness has failed a key clinical trial. The preliminary results are the latest blow to the company’s efforts to expand its scope to gene therapies.
Biogen paid $18 million to acquire a drug that TMS Co. of Japan tested in patients with acute ischemic stroke. The acquisition follows the report of preliminary mid-stage data showing the drug led to no cases of a potentially fatal complication associated with currently available stroke therapies.
Incubated by Flagship Pioneering, biotech firm Laronde is developing endless RNA, or eRNA, a new class of medicines that goes beyond messenger RNA-based therapies. Flagship is backing the startup $50 million in Series A financing.
It’s 7:30 a.m. on a school day. Two parents are racing to get their three young children dressed, fed, packed for the day, into coats and out the door when 6-year-old Hallel runs downstairs, crying. This story is part of a partnership that includes WBUR, NPR and KHN. It can be republished for free. Ari, …
Dyno Therapeutics’ technology for designing viral vectors that deliver gene therapies has led to partnerships with Novartis, Sarepta Therapeutics, and Roche. With the Series A financing, the startup plans to expand its technology to address more tissue types, and potentially add more partners.
Neuroelectrics is planning a pivotal test of its wearable medical device, which delivers brain electrical stimulation to treat epilepsy. The startup’s Series A round of funding was led by Morningside Ventures.
Affinia Therapeutics, which has technology that could deliver gene therapies to more tissue types in the body, raised the Series B financing as it looks ahead to clinical testing. The biotech is the latest company to close a substantial round of funding for gene therapy technology.
Vaccitech and Werewolf Therapeutics joined the public markets, raising a combined $230 million. The vaccine developer and the cancer immunotherapy biotech will apply the IPO proceeds toward clinical development of their respective pipelines.
Seasonal allergy sufferers have a plethora of choices for treating the itching and redness affecting their eyes. An experimental Aldeyra Therapeutics drug that takes a new approach to blocking inflammation triggered by allergens has met the goals of a pivotal study and the biotech now plans to discuss the data with the FDA.
In March 2020, just weeks into the covid-19 pandemic, the incident command center at Brigham and Women’s Hospital in Boston was scrambling to understand this deadly new disease. It appeared to be killing more Black and brown patients than whites. For Latinos, there was an additional warning sign: language. This story is part of a …
Alnylam Pharmaceuticals has filed for FDA approval of vutrisiran, which could become the biotech’s fourth drug that works by RNA interference. Meanwhile the company is responding to a federal subpoena seeking documents related to the company’s marketing and promotion of its first RNAi drug, Onpattro.
Vertex Pharmaceuticals is collaborating with Obsidian Therapeutics to develop up to five new gene editing therapies. Obsidian’s technology precisely controls protein expression, a capability the partners plan to apply to several undisclosed serious diseases.
The FDA placed a clinical hold on a KalVista Pharmaceuticals drug being readied for mid-stage studies in a rare, inherited disorder. Meanwhile, the FDA has some questions about the historical data Y-mAbs used as the control in a clinical trial for its antibody cancer drug.
Antibody therapies are seen as one way to overcome the variants that continue to fuel the pandemic. With its Series C financing, Adagio Therapeutics, led by CEO Tillman Gerngross, will proceed with a pivotal clinical trial of an antibody that has neutralized SARS-CoV-2 variants in lab and animal tests.
Relay Therapeutics has its own technology that applies artificial intelligence and machine learning techniques to drug discovery. Its $85 million acquisition of ZebiAI gives it another technology that applies ML to DNA-encoded libraries and predicts the small molecules with the potential to drug proteins.
Thermo Fisher Scientific sells laboratory equipment used in drug research and it also offers manufacturing services for experimental and commercialized drugs. By acquiring PPD, Thermo Fisher says it will gain capabilities in the growing global market of clinical trial services.
Amylyx’s ALS drug met the goals of a mid-stage study, paving the way for regulatory submissions in Europe and Canada. But the FDA is requiring data from a larger Phase 3 clinical trial and that study is slated to begin later this year.
Tango Therapeutics’ SPAC merger will infuse the biotech with $353 million to fund clinical trials for its cancer drugs based on synthetic lethality. Tango’s lead program is on track to reach the clinic next year.
With two cancer programs already making progress, Repertoire Immune Medicines will use the Series B financing to expand its immune synapse research to autoimmune disorders and infectious disease. CEO John Cox said his startup’s approach could advance immune medicines beyond the scope of currently available therapies.
Microsoft will buy up healthcare AI partner Nuance for $19.7 billion. Nuance makes speech-recognition tools for clinical documentation, as well as security tools.
RA Capital led Ventus Therapeutics’ Series B financing, which the startup will use to develop its pipeline of medicines for “undruggable” disease targets. CEO Marcelo Bigal said the cash also gives Ventus the flexibility to consider an IPO.
Mobidiag offers multiplex testing, which is the ability to test for multiple pathogens from a single test sample. Hologic said the acquisition will enable the company to enter the acute care test market, which is projected to grow in a post-pandemic world.
Virtual primary care startup Firefly Health plans to launch a benefit plan after raising $40 million in new funding. The company currently offers virtual and in-person access to a primary care team.
The FDA increased the maximum number of doses permitted for each vial of Moderna’s Covid-19 vaccine, a change intended to make more doses available. The regulator also authorized storage changes for the vaccines at vaccination sites.
Omega Therapeutics has raised more than $210 million in the past eight months for R&D of medicines that regulate genes. The company’s lead therapeutic candidate addresses a validated but elusive cancer target.
Alexandra Sierra carried boxes of food to her kitchen counter, where her 7-year-old daughter, Rachell, stirred a pitcher of lemonade. This story also ran on USA Today and GateHouse Media. It can be republished for free. “Oh, my God, it smells so good!” Sierra, 39, said of the bounty she’d just picked up at a …
In two pivotal studies years apart, Aveo Oncology’s drug tivozanib did not help kidney cancer patients live longer overall compared to the standard of care. The FDA approved still approved it, the latest development in a long saga that includes several regulatory stumbles.
The FDA decision gives Sarepta Therapeutics its third approved drug for Duchenne muscular dystrophy. The accelerated approval requires the biotech to conduct additional clinical testing to confirm the drug’s benefit.
The Neurocrine decision follows a clinical hold placed on the Parkinson’s program in December. It’s Voyager’s second loss of an R&D partner in the past year; AbbVie previously ended an alliance aiming to develop gene therapies for Parkinson’s and Alzheimer’s.
Cuando los hospitales de todo el país luchan contra la nueva ola de la pandemia, no son camas ni ventiladores lo que escasean. Son las personas que cuidan de los enfermos. Pero existe mano de obra altamente calificada de médicos, enfermeras y otros trabajadores de salud, con formación en el extranjero, que no se aprovecha …
Despite lower sales, lack of profits and heavy R&D spending, biotech companies that went public between 1997 and 2016 reached similar market caps as non-biotech companies, according to an academic study.
The latest funding brings total investment in the company to $750 million. The money has gone, in part, toward licensing of promising drug candidates in breast, lung and other cancers.
In August, Robert Pettigrew was working a series of odd jobs. While washing the windows of a cellphone store he saw a sign, one that he believes the “good Lord” placed there for him. “Facing eviction?” the sign read. “You could be eligible for up to $3,000 in rent assistance. Apply today.” It seemed a …
Leslie Cutitta said yes, twice, when clinicians from Massachusetts General Hospital in Boston called asking whether she wanted them to take — and then continue — extreme measures to keep her husband, Frank Cutitta, alive. The first conversation, in late March, was about whether to let Frank go or to try some experimental drugs and …
It’s a familiar moment. The kids want their cereal and the coffee’s brewing, but you’re out of milk. No problem, you think — the corner store is just a couple of minutes away. But if you have COVID-19 or have been exposed to the coronavirus, you’re supposed to stay put. Even that quick errand could …
The company said it is on track to start its 30,000-participant Phase III trial this month and has finished manufacturing enough supplies of the vaccine to do so.
The FDA has 60 days to decide whether or not to accept the application. An analyst wrote that the agency accepting it with a priority or standard review may signal whether the agency is seriously considering approval or has continued reservations about the amyloid beta-targeting drug’s clinical benefit.