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X4 Pharmaceuticals Announces Fast Track Designation Granted by the FDA to Mavorixafor for the Treatment of WHIM Syndrome

X4 Pharmaceuticals Announces Fast Track Designation Granted by the FDA to Mavorixafor for the Treatment of WHIM Syndrome

BOSTON, Oct. 08, 2020 (GLOBE NEWSWIRE) — X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel therapies targeting diseases resulting from dysfunction of the CXCR4 pathway, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to its lead asset, mavorixafor, for the treatment of adult patients with WHIM (Warts, Hypogammaglobulinemia, Infections, and Myelokathexis) syndrome, a rare, inherited, primary immunodeficiency disease caused by genetic mutations in the CXCR4 receptor gene.

Mavorixafor is a potential first-in-class, once-daily, oral, small molecule antagonist of chemokine receptor CXCR4, currently being investigated in a global pivotal Phase 3 clinical trial, 4WHIM, for the treatment of WHIM syndrome.

“The Fast Track Designation of mavorixafor for the treatment of WHIM syndrome further recognizes WHIM as a serious condition with a clear unmet need for an effective and potentially disease-modifying therapy,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We recently showed the potential therapeutic benefit of mavorixafor in a Phase 2 clinical trial in WHIM, with significant increases in white blood cell counts and reductions in infection rates and wart burden. We look forward to continuing to demonstrate mavorixafor’s clinical utility in WHIM syndrome in our ongoing Phase 3 clinical trial and working with the FDA to potentially bring this promising therapy to patients as soon as possible.”

The Fast Track Designation aims to facilitate the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs. Through the Fast Track program, X4 will be eligible for more frequent meetings with the FDA to discuss the drug’s development plan, protocols and clinical data that would support mavorixafor’s potential approval for WHIM.

In addition to Fast Track

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Overnight Health Care: Regeneron asks for emergency authorization of coronavirus treatment Trump received | McConnell says he hasn’t visited White House in two months due to coronavirus

Overnight Health Care: Regeneron asks for emergency authorization of coronavirus treatment Trump received | McConnell says he hasn’t visited White House in two months due to coronavirus

Welcome to Thursday’s Overnight Health Care.



Donald Trump wearing a suit and tie: Overnight Health Care: Regeneron asks for emergency authorization of coronavirus treatment Trump received | McConnell says he hasn't visited White House in two months due to coronavirus | Employer-sponsored health insurance premiums rise 4 percent


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Overnight Health Care: Regeneron asks for emergency authorization of coronavirus treatment Trump received | McConnell says he hasn’t visited White House in two months due to coronavirus | Employer-sponsored health insurance premiums rise 4 percent

Regeneron filed for emergency authorization of its antibody COVID-19 treatment drug, just hours after President Trump claimed it basically cured him. Mitch McConnell hasn’t been to the White House in months, and a new analysis shows Americans’ job-based health care is continually getting more expensive.

We’ll start with Regeneron:

Regeneron asks for emergency authorization of coronavirus treatment Trump received

Biotech company Regeneron late Wednesday applied for emergency authorization for an experimental antibody treatment praised by President Trump.

“Subsequent to our discussions with regulatory authorities, we have submitted a request to the U.S. Food and Drug Administration for an Emergency Use Authorization (EUA) for our REGN-COV2 investigational antibody combination for COVID-19,” the company said in a news release.

The move came just hours after the president praised the efficacy of the treatment in a short video message posted on Twitter.

“They gave me Regeneron, it’s called Regeneron,” Trump said in the five-minute video Wednesday afternoon. “It was unbelievable. I felt good immediately. I felt as good three days ago as I do now.”

Why it matters: Trump was taking several drugs for his illness, so it’s not clear which helped him feel better. He claimed he has the “emergency use authorization all set,” but the FDA is supposed to make decisions based on science and not demands from the president. Regeneron’s drug is still undergoing clinical trials, and while early results seem promising, the company has not released data to back up its claims.

Read more here.

McConnell says he hasn’t visited White

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Regeneron Shares Surge After Trump Praises COVID Treatment

Regeneron Shares Surge After Trump Praises COVID Treatment

Regeneron Pharmaceuticals  (REGN) – Get Report shares surged higher in pre-market trading Thursday after President Donald Trump praised its coronavirus antibody treatment and the drugmaker said it will seek emergency use authorization from the Food & Drug Administration. 

Regeneron said its REGN-COV2 treatment, a combination of two monoclonal antibodies, is designed to prevent the infectivity of SARS-CoV-2, the virus that causes COVID-19. The group has around 50,000 doses available for public use and said it would have doses available for “300,000 patients in total within the next few months” if the Emergency Use Authorization (EUA) is approved.

President Trump was injected with the treatment last week, according to his physicians, and he claimed in a video released late Wednesday to “feel good immediately” and suggested that “it just me me better. I call that a cure.” 

Regeneron shares were marked 4.6% higher in pre-market trading Thursday to indicate an opening bell price of $619.00 each, a move that would extend the stock’s six-month gain to around 20.8%.

Regeneron’s REGN-COV2, is undergoing a jointly run trial with the National Institute of Allergy and Infectious Diseases, of 2,000 people, as well as a separate study of 2,900 coronavirus patients who are in hospitals and ambulatory settings.

Earlier on Wednesday, Eli Lilly & Co. filed an EUA application with the FDA trial data showing that the experimental antibody, Y-CoV555, met both primary and secondary endpoints. 

The drugmaker had earlier noted that the treatment, which is being manufactured in a joint arrangement with Amgen Inc.  (AMGN) – Get Report. reduced the need for hospitalization and emergency-room visits among patients with moderate coronavirus symptoms.

Eli Lilly said it’s studying several neutralizing antibodes for the prevention and treatment of COVID-19, either in combination with other drugs or as a so-called monotherapy,

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Corbus Pharmaceuticals Announces Phase 2b Study of Lenabasum for Treatment of Cystic Fibrosis …

Corbus Pharmaceuticals Announces Phase 2b Study of Lenabasum for Treatment of Cystic Fibrosis …

  • Study did not meet primary endpoint of reducing rate of pulmonary exacerbations
  • Lenabasum treatment had a favorable safety profile and was well-tolerated
  • Data to be presented at the upcoming North American Cystic Fibrosis Conference Oct. 7-23

Norwood, MA, Oct. 06, 2020 (GLOBE NEWSWIRE) — Corbus Pharmaceuticals Holdings, Inc. (NASDAQ: CRBP) (“Corbus” or the “Company”), a clinical-stage drug development company pioneering transformative medicines that target the endocannabinoid system, today announced topline results from its 28-week Phase 2b study of lenabasum in patients with cystic fibrosis (CF). The study enrolled patients in the U.S., Canada and Europe at high risk for recurrent pulmonary exacerbations (PEx). Subjects received lenabasum or placebo added to their background treatments for CF.

The CF-002 Phase 2b trial did not meet the primary endpoint of a statistically significant reduction in rate of new PEx per subject per 28 weeks. Lenabasum treatment had a favorable safety profile and was well-tolerated.

The topline data will be presented at the upcoming virtual North American Cystic Fibrosis Conference (NACFC), taking place October 7-23, 2020.

Barbara White, M.D., Chief Medical Officer and Head of Research of Corbus, said, “We are very disappointed that the study did not meet the primary endpoint. We look forward to providing more details of study results starting tomorrow at NACFC. We thank the participants, the staff at study sites, the Cystic Fibrosis Foundation, and the European Cystic Fibrosis Society Clinical Trials for their support and partnership throughout this study.”

Yuval Cohen, Ph.D., Chief Executive Officer of Corbus, said, “We are immensely grateful to the Cystic Fibrosis Foundation for their invaluable support of this program from its inception. It has been a privilege to work with the CF community throughout this development program.”

Phase 2b CF-002 Trial Design

CF-002 was a multinational Phase 2b study evaluating the efficacy and

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Regeneron Pharmaceuticals Stock Shoots Up 8% on Trump Treatment

Regeneron Pharmaceuticals Stock Shoots Up 8% on Trump Treatment



a close up of a bottle: A doctor holds a coronavirus 2019-nCoV Blood Sample


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A doctor holds a coronavirus 2019-nCoV Blood Sample

Regeneron Pharmaceuticals (NASDAQ:REGN) stock is taking off on Monday following news that its treatment for the novel coronavirus was used on President Donald Trump.



a person posing for the camera: A doctor holds a coronavirus 2019-nCoV Blood Sample


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A doctor holds a coronavirus 2019-nCoV Blood Sample

Regeneron Pharmaceuticals is working on an antibody treatment for the coronavirus that has yet to get approval from the U.S. Food and Drug Administration (FDA). The treatment was used on President Trump, who tested positive for the virus last week.

Even though the treatment doesn’t have FDA approval, that could change in the near future. Analysts are predicting it will receive emergency use authorization from the FDA in the coming days to treat patients suffering from the virus. That would be a massive boost to Regeneron Pharmaceuticals stock, reports MarketsInsider.

Gallery: 7 ‘A-Rated’ Aggressive Biotech Stocks to Gamble On (InvestorPlace)

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