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GSK pays $100M to partner on a cancer drug with new twist on immunotherapy

GSK is partnering with Mersana Therapeutics on the development of an antibody-drug conjugate that is approaching Phase 1 testing. Unlike other ADCs that deliver a toxic drug payload, the Mersana drug is designed to activate the innate immune system to fight tumor cells.

Alnylam drug succeeds in heart study, setting stage for FDA filing and showdown with Pfizer

Alnylam Pharmaceuticals drug Onpattro, an FDA-approved treatment for nerve pain caused by hereditary transthyretin amyloidosis, now has Phase 3 data showing it can also help the much larger group of patients suffering heart problems from the rare protein disease. An FDA submission is planned, which would pit the Alnylam drug in competition with a blockbuster …

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FDA hits pause on Beam Therapeutics’ off-the-shelf cell therapy for blood cancers

Beam Therapeutics’ application to begin human testing of a cell therapy made with base-editing technology is now under an FDA clinical hold. In other clinical hold news, the FDA lifted a hold on a Celyad Oncology cell therapy clinical trial that was halted in March.

U.S. government places $1.74B order for Moderna’s new Covid-19 vaccine boosters

The U.S. government has signed a supply agreement securing 66 million doses of Moderna’s Covid-19 vaccine booster shots, which are designed to address the omicron subvariants driving new cases of infection. The agreement follows a similar supply agreement reached with Pfizer and BioNTech.

Novartis turns to algorithms developer Anumana to apply AI to cardio conditions

Novartis is partnering with algorithms startup Anumana to develop a new artificial intelligence-based solution for cardiovascular diseases. The goal is to find ways to detect heart conditions years before symptoms develop in order to enable earlier intervention.

Danger Ahead. Good

BY KIM BELLARD I saw a great quote by Alfred North Whitehead the other day: “It is the business of the future to be dangerous.” Now, I was a math major many years ago, so I know who Alfred North Whitehead was: the coauthor (with Bertrand Russell) of the Principia Mathematica, a landmark, three-volume treatise …

Danger Ahead. Good Read More »

Amgen joins Kernal Bio’s $25M financing as the startup brings mRNA 2.0 to cancer

Kernal Biologics has technology that enables the delivery of messenger RNA therapies to a wider range of destinations in the body. The Series A financing comes as the startup works to advance its lead immuno-oncology program toward its first clinical test.

With new gene therapy data in hand, Sarepta talks with FDA about approval pathways

Sarepta Therapeutics has more clinical data showing the safety and efficacy of its experimental gene therapy for Duchenne muscular dystrophy. A pivotal Phase 3 test is already underway and could post data next year but the company is also talking with regulators about the possibility of a submission under the accelerated approval pathway.

New data for Intellia’s CRISPR therapy shows potential for one-time treatment of a rare disease

An Intellia Therapeutics therapy that uses CRISPR to perform in vivo genomic edits has clinical data showing that the therapeutic effects continue for a year in patients. The biotech now plans to speak with regulators about the design for a pivotal clinical trial testing this therapy, a potential one-time treatment for a rare inherited disease.

GSK gets pneumococcal vaccine contender with $2.1B Affinivax acquisition

Pfizer and Merck have each won FDA approvals in the past year for new pneumococcal vaccines, but GSK aims to top both of them with its $2.1 billion acquisition of Affinivax. That biotech’s Phase 3-ready vaccine candidate addresses more bacterial strains than any pneumococcal vaccines currently available.

New Flagship Pioneering startup expands the protein universe to fuel drug discovery

ProFound Therapeutics emerged from stealth with technology that it claims has discovered tens of thousands of previously undiscovered proteins—many of them promising new drug targets. The Flagship Pioneering-founded startup is backed by $75 million to support its drug R&D along with plans to partner with large pharmaceutical companies.

CAR T without the toxicity: Kelonia gets $50M for a new take on cell therapy

Kelonia Therapeutics is developing cell therapies made by the in vivo engineering of a patient’s cells made possible by the targeted delivery of a genetic payload. The startup, newly out of stealth with $50M in funding, claims its approach would offer manufacturing and safety advantages over other cell therapy technologies.

FDA sounds caution on Foghorn cancer drug with a partial hold after patient death

The lead cancer drug candidate of Foghorn Therapeutics is now under a partial clinical hold following a patient death. According to the biotech, the patient may have experienced a side effect that is a known complication of leukemia drugs.

Flagship merges two of its biotechs to form cellular microenvironment-focused Sonata

Sonata Therapeutics is developing drugs that reprogram cellular microenvironments to send signals intended to have curative effects. Cancer is the initial focus of the biotech, which Flagship Pioneering formed by combining two of its preclinical portfolio companies.

Claiming Akebia agreement breach, Otsuka seeks to end anemia drug pact

Otsuka Pharmaceutical is ending its alliance with Akebia Therapeutics, a move that comes less than two months after the FDA rejected their partnered anemia drug, vadadustat, and asked for another clinical trial. Otsuka alleges unspecified breaches of the agreement by Akebia, and ending the alliance early could spare it from spending hundreds of millions of …

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PepGen IPO nabs $108M for muscular dystrophy drug with potential edge over Sarepta med

PepGen’s technology improves the delivery of a therapy to more tissue types and the clinical-stage biotech plans to use its IPO cash to continue developing its lead program for Duchenne muscular dystrophy. Meanwhile, eye products giant Bausch + Lomb returned to the public markets as a standalone company. Both the PepGen and Bausch + Lomb …

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Biogen gets in tune with MedRhythms to develop new digital therapeutic for MS

Biogen struck a deal that could bring a digital therapeutic to its multiple sclerosis product portfolio. The drugmaker is partnering with startup MedRhythms in the development of a therapy that combines sensors, software, and music to improve walking problems in multiple sclerosis patients.

Biogen pulls Alzheimer’s drug application in Europe as EMA calls data insufficient

Biogen withdrew an application seeking approval in Europe for Alzheimer’s disease drug Aduhelm, a move that follows a negative opinion on the therapy issued by a European Medicines Commission committee last December. The company was unable to persuade European regulators to change their minds about the drug.

Regenerative med biotech Satellite unveils tissue-based tech to restore organ function

Satellite Bio has emerged from stealth with technology for bioengineering tissue to restore organ function. The regenerative medicine startup, based on research from MIT and Boston University, is backed by $110 million in financing.

Cell therapy developer Be Bio hauls in $130M for pipeline in rare disease, cancer

Be Biopharma is developing cell therapies by engineering B cells to churn out therapeutic proteins with an initial focus on cancer and rare disease. The company’s Series B financing comes as competitors in the space also make progress with their engineered B cell therapies.

Final CMS decision limits coverage of Biogen’s Aduhelm to those in clinical trials

Biogen’s Alzheimer’s disease drug Aduhelm will only be covered for Medicare beneficiaries participating in a clinical trial. The final coverage determination will have the effect of limiting use of the drug, but the agency said its decision provides clarity on what drug companies need to show in order to secure Medicare coverage.

Safety concerns sink Akebia drug for anemia from chronic kidney disease

The FDA rejected Akebia Therapeutics drug vadadustat as a treatment for anemia caused by chronic kidney disease. According to the biotech, the agency cited safety concerns and asked for another clinical trial to show that the pill’s benefits outweigh its risks.

Celsius Therapeutics heats up with $83M and an IBD drug on path to the clinic

Single-cell analysis biotech Celsius Therapeutics has raised $83 million to advance its pipeline of drugs for autoimmune disorders and cancer. The startup’s lead autoimmune program, a TREM1-blocking antibody for inflammatory bowel disease, is expected to begin human testing in early 2023.

GSK joins LifeMine’s $175M funding as partners revive fungi as a drug discovery frontier

LifeMine Therapeutics, a company that analyzes fungal genomes to find molecules that have potential as new medicines, has raised $175 million in financing. Among the investors is GlaxoSmithKline, which is teaming up with the biotech startup in a multi-target drug discovery alliance.

AstraZeneca’s Evusheld Covid-prevention drug gets UK approval

Treatment aimed at people who cannot be vaccinated is boost to firm’s coronavirus portfolio AstraZeneca has received UK regulatory approval for its long-acting Covid-19 antibody treatment Evusheld in a boost to its coronavirus portfolio as the British-Swedish drugmaker targets greater drug development success at its new £1bn research lab in Cambridge. Aimed at preventing Covid …

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Novartis turns to Voyager Therapeutics to get gene therapies into the brain

Novartis is paying Voyager Therapeutics $53 million up front to begin a partnership focused on the discovery of capsids for central nervous system disorder gene therapies. Depending on the progress of the research, Voyager could earn up to $1.5 billion in milestone payments.

Sherlock Biosciences secures $80M to bring molecular diagnostics to the home

Diagnostics developer Sherlock Biosciences has advanced its molecular diagnostic capabilities, which can now produce faster results on low-cost devices. The startup will use its Series B round of funding to expand the reach of its technology, potentially placing it directly in the hands of consumers around the world.

Flagship’s Vesalius launches with $75M to take AI into realm of common diseases

Artificial intelligence is a powerful tool for rare disease drug discovery. The founders of Vesalius Therapeutics say AI and machine learning can also be applied to common diseases, and the startup has launched with $75 million in committed financing.

Startup hC Bioscience emerges with $24M to join pursuit of transfer RNA therapies

New startup hC Bioscience is developing therapies based on transfer RNA, molecules that can be leveraged to address disease-causing proteins. The biotech joins several companies that are developing tRNA therapies in this emerging class of genetic medicines.

Agios strategy shift pays off with FDA approval of first drug for rare anemia

Agios Pharmaceuticals has its third FDA-approved product, and the first under its new rare disease strategy. The regulatory decision also marks the first approved treatment for pyruvate kinase deficiency, a rare disease that leads to chronic anemia.

J&J partners with startup Remix, aiming to treat disease by “tuning” RNA

Several biotech companies are rising to the challenge of developing small molecules to drug RNA. Remix Therapeutics is joining the chase, but its different approach has drawn the interest of Johnson & Johnson, which has inked an R&D alliance spanning cancer and immunology.

Intellia’s Rewrite Therapeutics buyout brings new precision to gene editing

Gene-editing medicines developer Intellia Therapeutics is paying $45 million up front to acquire Rewrite Therapeutics. The startup’s technology brings Intellia another tool for its genetic medicines toolbox, one that could enable even more precise edits.

CMS proposes limited coverage of Biogen Alzheimer’s drug, only in clinical trials

The Center for Medicare and Medicaid Services proposes covering Biogen Alzheimer’s disease drug Aduhelm only for people enrolled in a clinical trial. In explaining this proposal, the agency cited the unclear benefit to patients as well as the documented safety risks associated with the drug, which can be best monitored within the context of a …

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Avrobio shifts gene therapy priorities as data surprise shelves lead program

Avrobio is stopping enrollment in a clinical trial for its Fabry disease program after new results showed variability in how the gene therapy was taken up by patients. No safety problems were reported, but the biotech said it now plans to focus its other gene therapy candidates for rare metabolic disorders.

FDA sets decision date for Amylyx Pharma ALS drug that improves neuron survival

The FDA has accepted Amylyx Pharmaceuticals’ submission for amyotrophic lateral sclerosis drug AMX0035 and it expects to issue a regulatory decision by late June. A lot will happen before then. Amylyx recently filed for an IPO and the company’s drug is slated for an advisory committee meeting ahead of an FDA decision.

Biogen projects confirmatory study for Alzheimer’s drug will take four years

Six months after Biogen was awarded regulatory approval for its Alzheimer’s disease drug Aduhelm, the company the company is laying out a timeline for the post-marketing study to confirm whether the drug works. The projected four-year study will enroll more than 1,300 patients and will include a placebo control arm.

Eli Lilly steers into Foghorn’s gene traffic control drugs with $380M pact in cancer

Eli Lilly is building its cancer drug pipeline via a research alliance with Foghorn Therapeutic, a biotech whose technology discovers drugs addressing targets in a cellular system that regulate the way genes are turned on and off. In paying its new partner $380 million up front, Lilly is also placing yet another bet on targeted …

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Curie Therapeutics unveils $75M to widen scope of radiopharmaceuticals for cancer

Radiopharmaceuticals deploy radiation to damage cancer DNA, but Curie Therapeutics sees these therapies opening the door to a wider range of ways to kill tumors. The startup has raised $75 million in Series A financing to advance its research.

Blueprint Medicines to buy cancer drug startup with potential edge over Takeda, J&J targeted therapies

Blueprint Medicines is paying $250 million up front to acquire Lengo Therapeutics, developer of targeted cancer therapies. The startup’s lead drug candidate is on track to begin human testing in non-small cell lung cancer, and Blueprint believes the small molecule has advantages over new targeted therapies from Johnson & Johnson and Takeda Pharmaceutical.

‘Follow the science’: AstraZeneca unveils £1bn R&D centre

Anglo-Swedish pharmaceutical company has come a long way since it fought off a takeover bid in 2014 Little expense has been spared at the giant glass and steel structure that sprouts from a once-vacant plot of land on the outskirts of Cambridge. AstraZeneca’s £1bn new research and development centre houses 16 labs and 2,200 scientists, …

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AstraZeneca cuts the ribbon on its $1bn R&D lab in Cambridge

After years of delays and rising costs, AstraZeneca has formally unveiled its $1 billion R&D facility in Cambridge, said to be the biggest science lab of its kind in the UK. The Discovery Centre (DISC) based in the Cambridge Biomedical Campus – first given the green light in 2015 – is being officially opened today …

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Generate Bio adds $370M for big push toward programmable protein medicines

Generate Biomedicines applies artificial intelligence and machine learning to protein analysis, which it uses to program its protein therapies for particular applications. The startup now has $370 million in financing to rapidly scale up operations, with a goal of reaching the clinic within two years.

CRISPR-editing biotech Arbor Bio adds $215M for liver, CNS disease therapies

Arbor Biotechnologies raised new cash to keep up the pace in the chase for new genetic medicines. The MIT spinout applies artificial intelligence and machine learning to discover CRISPR enzymes applicable for gene-editing therapies; the Series B financing comes as programs in liver and central nervous system disorders move closer to human testing.

Flagship’s next genetic meds startup has $50M for transfer at a critical protein stop

Alltrna, a biotech startup founded by Flagship Pioneering, is developing a new type of medicines based on a form of RNA called transfer RNA, or tRNA. The company’s scientists say that this approach offers the potential to treat a wide range of diseases, both rare and common.

Biotech startup Marengo unveils $80M and a new way to get T cells to fight cancer

Apple Tree Partners’ latest cancer biotech company, Marengo Therapeutics, is using antibodies to selectively activate T cells to fight cancer. Led by former Merck KGaA executive Zhen Su, the startup is launching with $80 million in financing, which will support a drug pipeline that includes a lead program expected to reach the clinic in 2022.

ALS drugmaker steps up with FDA filing, but yet another biotech stumbles in clinic

Amylyx Pharmaceuticals has submitted its amyotrophic lateral sclerosis (ALS) drug for FDA review based on positive Phase 2 data after discussions with the agency over the summer. Meanwhile, a Clene Nanomedicine ALS drug failed its Phase 2 study, but the biotech points to encouraging results in a subgroup of patients.

Biogen ALS drug fails main goal of key test, but company points to other results

Biogen amyotrophic lateral sclerosis drug, tofersen, failed a Phase 3 clinical trial, but the drug showed improvement according to secondary and exploratory goals of the study. Based on those results, the company said it is talking with regulators to find a path forward for the drug.

Resilience turns to Harvard for biologics manufacturing tech, inks 5-year R&D pact

Biomanufacturing technologies company Resilience is partnering with Harvard in a deal that could see innovations from the university’s labs commercialized under new businesses. Resilience has committed $30 million in funding to support Harvard biomanufacturing research.

Atlas Venture-incubated Rectify lands $100M to transport drug hunt to new grounds

Rectify Pharmaceuticals is taking an approach to disease that led to a successful franchise of Vertex Pharmaceutical lung drugs and expanding it to more diseases in more organs in the body. The startup has launched with $100 million in financing.

Pyxis IPO leads way as four life science firms kick off 4Q, raising nearly $500M

Pyxis Oncology’s IPO raised $168 million as the biotech steers toward the clinic with drug candidates and technology licensed from Pfizer. Theseus Pharma, IsoPlexis, and Cognition Therapeutics also went public; combined the four companies raised nearly half a billion dollars.

Voyager Therapeutics’ strategy shift to gene therapy tech leads to a Pfizer alliance

The gene therapy pipeline of Voyager Therapeutics has hit several setbacks in the past year, but a strategy shift to its capsid technology is now showing promise. Pfizer has signed on as partner in a wager that Voyager’s tech holds the key to delivery of gene therapies for cardiovascular and neurological disorders.

Vedanta Bio guts out microbiome trial win to keep up with field, get BARDA cash

Vedanta Biosciences is developing its microbiome therapy with support from the Biomedical Advanced Research and Development Authority. Under terms of that contract, the positive Phase 2 results in preventing recurrent C. diff infection trigger a $23.8 million payment.

Exo Therapeutics adds $78M as cancer, inflammation drugs march toward clinic

Biotech startup Exo Therapeutics aims to overcome the challenges of drugging proteins by targeting exosites, locations that modulate enzyme activity. The company has four small molecule drug candidates for cancer and inflammation, and the company plans to use the $78 million in new financing to advance them toward human testing.

Anji Pharma adds $70M for clinical trials, expansion of ‘hub and spoke’ biz model

Cross-border biotech Anji Pharma closed $70 million in Series B funding to advance a drug pipeline that includes metabolic and cancer drugs. The biotech’s business model of finding promising drugs and forming subsidiaries to develop them is similar to approaches taken by Roivant Sciences and BridgeBio Pharma, among others.

Biogen and Samsung land FDA approval for biosimilar to blockbuster Roche drug

The FDA has approved the first biosimilar that references the blockbuster Roche drug Lucentis. The Biogen and Samsung BioLogics joint venture that developed the biologic product have approval to treat three eye conditions that lead to vision loss.

Startup testing video-based treatment for ‘lazy eye’ touts Phase 3 results

Luminopia, a startup with a digital therapeutic for amblyopia, or lazy eye, recently published the results of a phase 3 study showing its TV-based treatment can improve vision in kids’ non-dominant eye. Patients don a headset where images are altered to make them use both eyes. 

Blood disorder biotech Disc Medicine gets $90M to bring two drugs into Phase 2

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.

A biotech startup’s hunt for elusive disease targets scores $60M for drug R&D

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.

Boots launches on-demand delivery trial with Deliveroo

Customers can order medicines for mild ailments, makeup, toiletries and more from 14 stores The pharmacy chain Boots will become the latest large retailer to make its products available through Deliveroo, as the takeaway delivery company aims to expand beyond restaurant orders. Stores in London, Birmingham, Edinburgh and Nottingham will be among the 14 initially …

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Pfizer makes big move in blood cancers with $2.3B Trillium acquisition

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.

Sesen Bio says rejected bladder cancer drug may need new clinical trial

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.

Eli Lilly joins $30M Series A financing for startup bringing AI analysis to endoscopy

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.

Biogen and Eisai unveil details for five-year, real-world Aduhelm clinical trial

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

Ring Therapeutics rounds up $117M to find better viral vectors for gene therapy

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 microbiome drug flunks Phase 2 test in ulcerative colitis

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.

Amylyx Pharmaceuticals raises $135M as ALS drug heads to pivotal clinical trial

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.

PAQ Therapeutics scores $30M to unleash “Pac Man” drugs on neuro disease

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.

Biogen reaches across globe and pays $125M to land its next new MS drug

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.

Sigilon hemophilia A cell therapy trial placed on hold after patient complication

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.

UK supercomputer Cambridge-1 to hunt for medical breakthroughs

Computer – the most powerful in Britain – will use AI to find new cures and deepen understanding of diseases The UK’s most powerful supercomputer, which its creators hope will make the process of preventing, diagnosing and treating disease better, faster and cheaper, is operational. Christened Cambridge-1, the supercomputer represents a $100m investment by US-based …

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Apellis taps Beam’s base-editing tech to broaden autoimmune disease scope

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’s early CRISPR trial data validate a drug pipeline and the gene-editing field

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, Verve IPOs raise a combined $692M as both biotechs steer toward clinical trials

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.

Northpond, 5AM Ventures lead $45M funding for CAMP4’s trek to new RNA therapies

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.

While Sage Therapeutics sees a window for depression drug, market is skeptical

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 …

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Pioneering a new realm of biology, startup Senda Bio expands financing to $98M

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.

MorphoSys to buy Constellation Pharma in a $1.7B bet on epigenetic cancer drugs

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’s Covid vaccine shows efficacy in younger teens; regulatory filing are next

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 teams up with Ginkgo Bioworks in gene therapy manufacturing pact

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.

Bayer’s BlueRock Therapeutics widens cell therapy scope to the eye under new pact

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.

Biogen adds to neuro drug pipeline with acquisition of stroke drug from TMS

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.

Andreessen Horowitz leads $100M Series A round in Dyno and its gene therapy tech

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 gets $17M for new take on electrical therapy for epilepsy, depression

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.

Biotech IPO wave rolls on as Werewolf, Vaccitech raise $230M for clinical trials

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.

Alnylam FDA submission aims to expand biotech’s franchise of gene-silencing drugs

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 Pharma inks another R&D alliance to expand its gene-editing ambitions

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.

Regulatory progress for two biotechs stalls as FDA requests more information

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.

Relay Therapeutics pays $85M for startup with a new AI tech for drug discovery

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.

Amylyx Pharma’s ALS drug needs a Phase 3 study to support FDA submission

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.

Biotech startup Repertoire adds $189M to expand array of immune medicines

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.

Aveo Oncology’s kidney cancer drug gets FDA nod, 8 years after stinging rejection

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.

Neurocrine nixes Parkinson’s gene therapy from Voyager Theraeutics alliance

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.

Moderna’s Covid-19 vaccine shows antibody, T-cell responses a month after second dose in interim Phase I data

The company published interim data from the Phase I study of mRNA-1273 in 40 older and elderly adults, showing responses comparable to those seen in younger adults. The vaccine is being tested in a 30,000-participant Phase III trial.

Foundation Medicine to launch liquid biopsy companion diagnostic following FDA approval

The company announced Wednesday the FDA approval of its liquid biopsy test, the second to win an agency nod in less than a month, saying it would launch the product on Friday. The FDA acquired the first ever liquid biopsy companion diagnostic, Guardant Health’s Guardant360 CDx, on Aug. 10.

J&J to buy Momenta Pharmaceuticals for $6.5B

The drugmaker emphasized the full global rights it would acquire to Momenta’s lead asset, nipocalimab, which it is developing for several autoantibody-driven diseases and that in certain indications could have peak sales of more than $1 billion.

Moderna secures more than $1.5B federal government deal to provide Covid-19 vaccine

The agreement, which includes 100 million doses of mRNA-1273 and an option to acquire 400 million more, brings the total amount of money the biotech company has received from the federal government for its vaccine efforts to nearly $2.5 billion.

Biogen to get speedy FDA review for Alzheimer’s drug despite not using voucher to request it

Biogen and Eisai said the FDA had accepted and given priority review to their application for aducanumab. The drug will undergo an advisory committee meeting at an undisclosed date. Biogen’s stock rose 10%, but analysts had mixed opinions.

BMS, bluebird refile FDA approval application for CAR-T therapy in myeloma

The companies’ resubmission of their application Wednesday seeking approval for idecabtagene vicleucel was in line with the timeline they provided in May, when the FDA sent a refuse-to-file letter in response to their initial submission.

Thrive raises $257M Series B round for registration study of blood test for early cancer detection

The study design is still being finalized, and the company is thus not yet divulging too many details. However, CEO David Daly said it would be significantly larger than the previous DETECT-A, multi-center and would include both men and women.

Moderna snags another $472M from BARDA, launches Phase III Covid-19 vaccine trial

The company had received $483 million from BARDA in April to fund the development of the vaccine, mRNA-1273, through Phase III, but that amount was based on a smaller anticipated number of participants than the 30,000 it now plans to enroll into the trial, named COVE.

Biogen to launch Phase IV study of spinal muscular atrophy drug in post-gene therapy patients

The company plans to enroll 60 patients who have received its drug, Spinraza, following suboptimal response to Novartis’ Zolgensma, which was approved last year. The trial, RESPOND, is anticipated to start enrolling in the first quarter of 2021, pending regulatory approval.

Moderna publishes Phase 1 Covid-19 vaccine data showing robust immune responses

Evaluation of durability is still under evaluation, but the responses to mRNA-1273 show neutralizing antibody titers multiple times higher than those of recovered patients. Additionally, T-cell responses suggest potentially lower risk of enhanced respiratory disease.

Roche pays Blueprint $675M upfront for ex-U.S. development, commercialization rights to cancer drug

Under the deal, Blueprint will additionally be eligible for more than $900M in milestone payments plus royalties for the RET inhibitor pralsetinib. The deal includes exclusive rights for Roche in ex-U.S. markets other than China and shared rights in the U.S.

Biogen, Eisai complete submission of controversial Alzheimer’s drug. The ball is in FDA’s court now.

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.

‘The wondrous map’: how unlocking human DNA changed the course of science

Thanks to the success of the Human Genome Project, 20 years ago this week, scientists can track biology and disease at a molecular level Twenty years ago this week, an international group of scientists announced it had put together the first genetic blueprint of a human being. After 10 years of effort, the team – …

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