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Updated: 3 hours 39 min ago

Sanofi, Pfizer & Bristol Drugs Among the 12 Approvals to Look Out for in Q4

Thu, 10/15/2020 - 10:23

The end of the year should see a flurry of new products reaching the market to break new commercial ground or shake up their respective therapeutic areas. Here, Scrip takes a look at 12 of the more interesting approvals expected in the coming months, with the help of analysts at Biomedtracker.

Supernus Pharmaceuticals’ SPN-812
Indication: Attention Deficit Hyperactivity Disorder

PDUFA Date: 8 November NDA – First Review

Supernus Pharmaceuticals, Inc. should hear by 8 November whether the US Food and Drug Administration will approve SPN-812 (viloxazine hydrochloride), its novel non-stimulant treatment for the treatment of children and adolescents... Read more »

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Vertex Stops Test of Rare Disease Drug, Dimming Pipeline Prospects

Thu, 10/15/2020 - 08:46

Vertex Pharmaceuticals is halting work on an experimental drug for a rare protein deficiency after safety problems were reported in Phase 2 testing.

The study was evaluating the small molecule, VX-814, as a treatment for alpha-1 antitrypsin deficiency, which can lead to liver and lung problems. After the markets closed Wednesday, Vertex (NASDAQ: VRTX) disclosed that elevated liver enzymes—eight times greater than normal—-were observed in four of the patients who received the drug i. The placebo-controlled study enrolled approximately 50 patients.

The liver enzyme levels are resolved or are resolving but Vertex also said that its analysis of how VX-814... Read more »

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Codiak Bio’s IPO Brings In $82M as Exosome Clinical Trials Begin

Wed, 10/14/2020 - 07:59

Codiak BioSciences now has $82.5 million to fund clinical tests of a new class of medicines based on exosomes, tiny bubbles formed by cells that the company harnesses to carry therapeutic cargo.

Late Tuesday, Codiak priced its offering of 5.5 million shares at $15 apiece, the midpoint of its projected $14 to $16 per share price range. Shares of the Cambridge, MA-based biotech are set to begin trading Wednesday on the Nasdaq under the stock symbol “CDAK.”

Exosomes are extracellular vesicles that function like a messaging system between cells. Laden with proteins, genetic material, and other substances, these vesicles transport molecules... Read more »

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Roche Enlists Dyno to Bring AI to Next-Generation Liver, CNS Gene Therapies

Wed, 10/14/2020 - 03:00

Roche made a splash with its acquisition of Spark Therapeutics, whose Luxturna, a gene therapy for an inherited form of vision loss, was the first such therapy to win FDA approval. Now the Swiss pharmaceutical giant is looking to bolster its capabilities in liver and central nervous system (CNS) disorder gene therapies and it’s turning to Dyno Therapeutics to help.

Dyno, a two-year-old startup based in Cambridge, MA, has never brought a gene therapy to the market, nor does it currently have its own drug pipeline. What it does have is a platform technology for engineering the viruses that ferry gene... Read more »

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Despite Phase 2 Failure, Gossamer Bio Sees a Path for Drug in Asthma

Tue, 10/13/2020 - 14:28

Gossamer Bio’s lead drug, an experimental treatment for asthma and allergies, has failed in two mid-stage clinical trials but the biotech sees enough in the data to pursue further development in a severe form of asthma and it wants to talk with regulators about advancing the compound to a larger Phase 3 study.

The Gossamer drug, GB001, was tested as a treatment for eosinophilic asthma, a form of the respiratory disorder characterized by high levels of white blood cells called eosinophils. According to the San Diego biotech, 10 to 15 percent of patients who have this form of asthma are treated... Read more »

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Dewpoint Therapeutics Taps Ex-Sanofi Executive Ameet Nathwani as CEO

Tue, 10/13/2020 - 06:35

Ameet Nathwani has been appointed CEO of Dewpoint Therapeutics. He is also joining the Boston company’s board of directors. Earlier this year, Nathwani left his chief medical officer and chief digital officer roles at Sanofi (NYSE: SNY) as part of an executive team shakeup. His experience also includes 12 years at Novartis (NYSE: NVS) in a number of senior roles. Dewpoint is developing drugs that target components of cells called biomolecular condensates. The biotech has research and development partnerships with Bayer and Merck (NYSE: MRK).

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SQZ Biotech Lines Up an IPO on the NYSE to Fund Cell Therapy R&D

Mon, 10/12/2020 - 14:39

SQZ Biotechnologies, which already has an alliance with Roche focused on developing new cancer cell therapies, is now looking to sell shares to the public to finance its research of potential new treatments for both cancer and infectious disease.

In paperwork filed with securities regulators late last week, SQZ set a preliminary $75 million goal for its IPO. The Watertown, MA-based biotech has applied for a listing on the New York Stock Exchange under the stock symbol “SQZ.”

SQZ (pronounced “squeeze”) is developing technology intended to improve cell therapy. Current cell therapies get the therapeutic cargo into a cell via an... Read more »

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“Opioid Stacking” Concerns Lead FDA to Reject Avenue Therapeutics Drug

Mon, 10/12/2020 - 08:15

An opioid that Avenue Therapeutics reformulated for treating post-surgical pain in hospital settings has been rejected by the FDA due to safety concerns.

The Avenue (NASDAQ: ATXI) drug is an intravenous formulation of tramadol, an opioid that first reached the US market 25 years ago in pill form. Now New York-based Avenue is trying to offer an alternative to painkillers used after surgical procedures, as well as an improvement over an intravenous formulation of tramadol that is already approved for use in Europe. But that improvement may have unwittingly led to the basis for the FDA rejection.

In Europe, intravenous... Read more »

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FDA Panel Vote Weighs in Favor of Alkermes Psyche Drug, With Caveats

Fri, 10/09/2020 - 18:03

An Alkermes antipsychotic treatment designed to offer the benefits of a commonly prescribed medicine, but without the weight gain side effect, won the backing of an FDA advisory committee on Friday. But even some of the panel members who supported the drug qualified their votes, expressing concern about what the drug could mean for patients taking opioid painkillers.

The vote of the independent panel isn’t binding, but the FDA will consider the body’s votes and comments as it evaluates the drug developed by Dublin, Ireland-based Alkermes (NASDAQ: ALKS), which also maintains operations in Waltham, MA. The agency is expected to... Read more »

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Bio Roundup: CRISPR Kudos, Bristol Myers’s Buy, RNAi Alliance & More

Fri, 10/09/2020 - 08:08

It’s October: a time of days growing shorter, leaves changing color, and phone calls in the wee hours that rouse scientists from slumber notifying them they’ve won the highest honor in their fields.

This week, the 2020 Nobel Prize in physiology or medicine was awarded to Harvey Alter, Michael Alton, and Charles Rice, whose research led to the discovery of the hepatitis C virus. Their work paved the way for blood tests and new medicines that can cure the infection. In chemistry, the Nobel was awarded to Emmanuelle Charpentier and Jennifer Doudna for their discovery of CRISPR/Cas9, a system that... Read more »

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Biogen’s AdComm Meeting Will Test FDA’s “Substantial Evidence” Flexibility

Thu, 10/08/2020 - 12:16

A US Food and Drug Administration advisory committee meeting on Biogen, Inc.’s Alzheimer’s drug aducanumab will offer some insight into the amount of flexibility the agency and its external experts are willing to exercise for neurodegenerative diseases with high unmet need, particularly in the wake of a December 2019 guidance document on the quantum and type of data capable of satisfying the “substantial evidence” standard.

On 6 November, the Peripheral and Central Nervous System Drugs Advisory Committee will consider the benefit/risk profile of aducanumab, an anti-amyloid-beta antibody infusion intended for treatment of Alzheimer’s, according to a notice due to publish in... Read more »

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In RNAi Move, Takeda Fronts $300M to Team Up on Arrowhead Liver Drug

Thu, 10/08/2020 - 10:11

Arrowhead Pharmaceutical’s gene silencing treatment for a rare liver disease will move through the regulatory process and potential commercialization with the muscle of Takeda Pharmaceutical behind it.

Takeda (NYSE: TAK) has agreed to pay $300 million up front in a partnership on Arrowhead (NASDAQ: ARWR) drug ARO-AAT as a treatment for alpha-1 antitrypsin-associated liver disease. According to terms of the agreement announced Thursday, Arrowhead and Takeda will co-develop the drug, which is currently in Phase 2/3 testing.

In addition to the upfront cash, Pasadena, CA-based Arrowhead could earn up to $740 million in milestone payments. If the drug is... Read more »

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BIO Launches ‘Biotech Votes’ Campaign to Encourage Registration and Informed Voting

Thu, 10/08/2020 - 06:41

Science is the way out of this pandemic—and scientific breakthroughs are on the ballot on November 3. To help you get ready for Election Day, BIO’s initiative Biotech Votes has resources to help you get registered and get informed.

First, make sure you’re registered to vote. Check out this handy form to verify your voter information, find out your polling location, or request a mail-in ballot.

Then, get informed. This Biotech Votes 2020 Election Preview Report is an in-depth analysis of the races that matter the most, from the top of the ticket down the ballot to Senate, House, and state... Read more »

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Scribe Therapeutics Sets Out to Write CRISPR Gene Editing’s Next Chapter

Tue, 10/06/2020 - 13:02

The first generation of CRISPR-based therapies showed how the technology can edit DNA, making genetic fixes to unwanted mutations. But those treatments use the CRISPR system as scientists found it in nature, which means it still has limitations when applied to humans, says Benjamin Oakes, CEO of Scribe Therapeutics.

Scribe aims to develop gene-editing therapies that improve CRISPR’s efficacy, safety, and deliverability to target cells. Oakes says Scribe can achieve these goals with technology that imbues a therapy with whatever attributes will make it best suited for a particular application. More than taking what nature provides, Scribe engineers molecules to what... Read more »

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Federation Bio Bags $50M to Engineer New Approach to Microbiome Drugs

Tue, 10/06/2020 - 04:30

Scientists at microbiome therapy developers often talk about the importance of community, how the bugs in our gut act in concert in addition to their individual roles. That’s important for microbiome health and for microbe-based therapies, says Emily Drabant Conley, CEO of startup Federation Bio.

Conley’s company is developing therapies that build on the understanding of the microbiome community. It’s also going a step further by engineering some bacteria to take on new roles fighting disease. The biotech now has $50 million to advance its most advanced program toward human testing for a rare metabolic disorder.

South San Francisco-based Federation Bio is... Read more »

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Bristol Myers to Buy MyoKardia, FDA-Ready Heart Disease Drug for $13.1B

Mon, 10/05/2020 - 09:43

Bristol Myers Squibb is bolstering its cardiovascular drug lineup with a $13.1 billion deal to acquire MyoKardia, a company whose experimental therapy for a rare type of heart disease is on track for an FDA submission and is projected to become a blockbuster seller, if approved.

According to financial terms announced Monday, Bristol (NYSE: BMY) has agreed to pay $225 cash for each share of MyoKardia (NASDAQ: MYOK), a 61 percent premium to the Brisbane, CA-based company’s closing stock price on Friday. Shares of MyoKardia opened at $220.68 per share on Monday.

The key piece of the MyoKardia (NASDAQ:... Read more »

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Bristol Myers’s Hirawat on Pipeline Progress, Clinical Trial Diversity

Fri, 10/02/2020 - 11:12

As the one-year anniversary of Bristol-Myers Squibb Company’s $74bn acquisition of Celgene Corporation approaches, Scrip spoke with chief medical officer Samit Hirawat about the big pharma’s achievements during a challenging period in which the COVID-19 pandemic threatened to derail clinical trials and a national spotlight on racial inequality shined a light on the industry’s lack of diversity.

Key products, such as the PD-1 inhibitor Opdivo (nivolumab), delivered positive results in important studies and won new approvals since the BMS-Celgene merger closed in November. However, Bristol promised Celgene shareholders an additional payout if three key product candidates were approved by certain dates... Read more »

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Bio Roundup: MESO’s Miss, Pfizer’s Cancer Bet, Vaccine Promise & More

Fri, 10/02/2020 - 07:19

Amid the bombast of Tuesday’s presidential debate, viewers caught some discussion about the response to the coronavirus pandemic and efforts to develop a COVID-19 vaccine. In the crosstalk, the president made an unsupported claim that we are weeks away from having vaccines.

It’s true that some pharmaceutical companies are already manufacturing their COVID-19 vaccine candidates for broader use. This production ensures that vaccine product is ready for distribution upon receiving a regulatory decision. But companies undertake this “at-risk” manufacturing because they don’t yet know how well their vaccines will work or if they’ll be approved. The industry is pushing back, even... Read more »

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FDA Lifts Solid Bio Clinical Hold, Duchenne Study Cleared to Resume

Thu, 10/01/2020 - 09:42

Solid Biosciences has answered the FDA’s outstanding questions about its Duchenne muscular gene therapy, clearing a clinical trial to now resume after patient complications last year led the agency to halt the study for a second time.

Cambridge, MA-based Solid Bio (NASDAQ: SLDB) said Thursday that it expects dosing of patients in the Phase 1/2 study testing its experimental gene therapy, SGT-001, will start again in the first quarter of 2021. When it does, the maximum weight of patients in the study will be lowered, a change intended to boost safety. The therapy is dosed according to a patient’s weight.... Read more »

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Genfit Restructuring Cuts Staff by 40%, Creates Two New Subsidiaries

Wed, 09/30/2020 - 16:53

Genfit is halting all work on its lead drug in the fatty liver disorder nonalcoholic steatohepatitis (NASH) and implementing a corporate restructuring that will cut 40 percent of its staff, splitting the remaining workforce into two distinct business units.

The announcement after the market close on Wednesday follows the May failure of the drug, elafibrinor, in a pivotal NASH study. At the time, the company attributed the surprising result to a higher than anticipated placebo response. Genfit (NASDAQ: GNFT) pledged to review the data to see if there was a path forward in NASH.

Speaking on a conference call... Read more »

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