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Friday, June 17, 2022

Capricor: One-Year Efficacy Results from Muscular Dystrophy Program

  Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of a broad spectrum of diseases, today announced that the Company will present one-year, safety and efficacy results from its HOPE-2 open-label extension study with lead asset CAP-1002 for treating Duchenne muscular dystrophy (DMD) at this year’s Parent Project Muscular Dystrophy (PPMD) Annual Conference.

The details are as follows:

  • Dr. Linda Marbán, CEO, will present the one-year open label extension results via a late-breaking session on June 25, 2022, at 5:05 p.m. MT.

  • Capricor will also deliver a clinical update as part of PPMD’s “Novel Approaches for Combatting Duchenne” session on June 25, 2022, at 11:00 a.m. MT.

KAZIA: US FDA AWARDS ORPHAN DRUG DESIGNATION (ODD) TO PAXALISIB

  Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, is pleased to announce that the United States Food and Drug Administration (FDA) has awarded Orphan Drug Designation (ODD) to Kazia's paxalisib for the treatment of atypical rhabdoid / teratoid tumors (AT/RT), a rare and highly-aggressive childhood brain cancer.

https://finance.yahoo.com/news/us-fda-awards-orphan-drug-120000433.html

'Long Covid' risk lower with Omicron study

 Last winter's Omicron variant was less likely to cause lingering symptoms, known as "long Covid", a UK investigation, in The Lancet, suggests.

The King's College London team looked at data from nearly 100,000 people who logged their Covid symptoms on an app.

Just over 4% of those infected during the Omicron wave had logged long Covid symptoms, compared with 10% of those infected in the preceding, Delta, wave.

But as far more were infected during the Omicron wave, the total was higher.

In fact, the much bigger number of new infections during the Omicron wave "entirely trumped" the variant's potential lower risk of long Covid, Kevin McConway, emeritus professor of applied statistics, at The Open University, said.

"Anyway, you don't really have any choice about which virus variant you might be infected with," he said.


"What's more, nothing in these findings tells us what might happen with a different new variant, in terms of long Covid risk."

The researchers tried to take into account other variables, such as how long ago someone had been vaccinated against Covid, but it is impossible to be certain the difference between variants caused the difference in long Covid numbers.


Lead researcher Dr Claire Steves said: "The Omicron variant appears substantially less likely to cause long Covid than previous variants - but still, one out of every 23 people who catches Covid-19 goes on to have symptoms for more than four weeks.

"Given the numbers of people affected, it's important that we continue to support them at work, at home and within the NHS."

Officials estimate long Covid has affected at least two million people in the UK.

Addex ends lead program

  Addex Therapeutics Ltd (SIX: ADXN, Nasdaq: ADXN), a clinical-stage pharmaceutical company pioneering allosteric modulation-based drug discovery and development, announced today that it has terminated the Phase 2b/3 study evaluating dipraglurant as a potential treatment for dyskinesia associated with Parkinson’s disease (PD-LID) due to the slow recruitment of patients. This has been attributed to the consequences of COVID-19 related patient concerns about participation in clinical studies as well as staffing shortages and turnover within study sites.

https://finance.yahoo.com/news/addex-terminates-dipraglurant-phase-2b-050000406.html

AstraZeneca may be eyeing a takeover of Mereo

 AstraZeneca is rumoured to be considering a bid to take control of its longstanding partner Mereo BioPharma, according to a report in The Times, which doesn’t provide the source of the speculation.

The UK-based but US-listed biotech licensed rights to AZ’s orally-active neutrophil elastase inhibitor alvelestat for the rare disease alpha-1 antitrypsin deficiency (AATD) five years ago, and has since advanced the project into a phase 2 trial which is due to read out in the second half of this year.

The licensing deal took place as AZ was trimming down its pipeline to focus its R&D activities on the core areas of cardiology, diabetes and neurology, and recovering from a series of patent expiries that hit its revenues. Since then it has bounced back dramatically, thanks to fast-growing drugs for cancer and heart disease.

AZ may be interested in re-acquiring rights to alvelestat, which cleared a phase 2 test in AATD last month, albeit with some questions about the data, given its push into rare diseases that resulted from its takeover of Alexion last year.

It’s also possible that the big pharma is interested in Mereo‘s other clinical-stage candidates, such as its anti-TIGIT antibody etigilimab which is being tested for cancer alongside PD-1/PD-L1 inhibitors.

AZ already has a presence in TIGIT however with its bispecific antibody programme and a partnership with Arcus pairing its TIGIT drug domvanalimab with AZ’s PD-L1 inhibitor Imfinzi (durvalumab) in a phase 3 trial.

Meanwhile, and enthusiasm for the category has been dampened by the failure in clinical trials of Roche’s tiragolumab, currently leading the pack.

Mereo’s other main candidate is Novartis cast-off setrusumab for osteogenesis imperfecta, an inherited bone disorder, but that asset is tied up into an alliance with bone disease specialist Ultragenyx.

Its remaining programmes are anti-DLL4/VEGF bispecific navicixizumab for ovarian cancer – already out-licensed to OncXerna – aromatase inhibitor leflutrozole for male infertility associated with hypogonadotropic hypogonadism, and acumapimod, a p38 MAP kinase inhibitor for chronic obstructive pulmonary disorder (COPD).

The rumour has emerged as Mereo has just been notified by the Nasdaq that the price of its American Depository Shares has failed to maintain the minimum price of $1 over the last 30 days, putting it at risk of delisting.

The company’s ADS rose around 16% pre-market on the back of the rumour to 80 cents, after losing almost 40% of their value in the last month.

https://pharmaphorum.com/news/rumour-mill-says-astrazeneca-may-be-eyeing-a-takeover-of-mereo/

Cytokinetics gets extended PDUFA date

 Cytokinetics, Incorporated (Nasdaq: CYTK) today announced that, in response to a request from the U.S. Food and Drug Administration (FDA), the company recently provided additional pharmacokinetic analyses of omecamtiv mecarbil related to the company’s new drug application (NDA). After an initial review of the company’s submission, FDA has communicated that the additional data provided constitutes a major amendment to the NDA and has extended the PDUFA date by three months to February 28, 2023 to provide time for a full review of the submission.

https://finance.yahoo.com/news/cytokinetics-provides-regulatory-omecamtiv-mecarbil-113000740.html

AbbVie: SKYRIZI Receives FDA Approval for Crohn's

  Third approved indication for SKYRIZI (risankizumab-rzaa) is supported by safety and efficacy data from two induction and one maintenance clinical trials evaluating SKYRIZI in moderately to severely active Crohn's disease, ADVANCE, MOTIVATE and FORTIFY1-4

- As early as week 4 in the induction studies, clinical response and clinical remission were achieved by significantly more subjects treated with SKYRIZI versus placebo, as were co-primary endpoints of endoscopic response and clinical remission at week 12 and week 521-4

Crohn's disease is chronic, systemic and progressive; most patients experience unpredictable symptoms that have a significant impact on daily life5-8

https://finance.yahoo.com/news/skyrizi-risankizumab-rzaa-receives-fda-120000225.html