The Daily Biotech Pulse: Addex Terminates Parkinson's Study, Expanded Use Of Rhythm's Obesity Drug, FDA Approval For AbbVie's Skyrizi In Crohn's Disease

Zinger Key Points
  • Global Cord Blood Corporation's Cellenkos has received an FDA nod for its Investigational New Drug application.
  • Sierra Oncology has submitted a marketing application to the FDA for momelotinib for myelofibrosis (bone marrow cancer).

Here's a roundup of top developments in the biotech space over the last 24 hours:

Stocks In Focus

Addex Stops Parkinson's-Associated Dyskinesia Trial

Addex Therapeutics Ltd ADXN has terminated the Phase 2b/3 study of dipraglurant for dyskinesia associated with Parkinson's disease (PD-LID) due to slow recruitment of patients.

The company plans to advance its preclinical portfolio toward the clinic and pursue strategic collaborations for selected programs.

Shares were down 52.47% at $1.59 Friday morning. 

Rhythm's Setmelanotide Scores FDA Approval For Expanded Use In Bardet-Biedl Syndrome

The FDA approved Rhythm Pharmaceuticals Inc's RYTM Imcivree (setmelanotide), a melanocortin-4 receptor (MC4R) agonist, for patients with Bardet-Biedl syndrome (BBS).

With today's approval, Imcivree is indicated for chronic weight management in patients 6 years old and older with monogenic or syndromic obesity due to proopiomelanocortin, proprotein convertase subtilisin/kexin type 1, leptin receptor deficiency or BBS.

Rhythm Pharma Secures Non-Dilutive Financing Of $100M

Rhythm Pharmaceuticals Inc RYTM entered into a Revenue Interest Financing Agreement with HealthCare Royalty Partners for a total investment of $100 million

Rhythm intends to use the proceeds to support global commercialization efforts for its newly approved Imcivree (setmelanotide) and ongoing clinical development.

Shares are rallying 20.26% to $4.69.

FDA Approves AbbVie's Skyrizi For Crohn's Disease

The FDA has approved AbbVie Inc's ABBV Skyrizi (risankizumab-rzaa) moderately to severely active Crohn's disease (CD).

The approval marks the first and only specific interleukin-23 inhibitor for treating adults with CD.

The dosing regimen for Skyrizi is 600 mg intravenous infusion over at least one hour at week zero, week four and week eight followed by 360 mg self-administered by subcutaneous injection (SC) with an on-body injector at week 12 and every eight weeks after that.

A 180 mg self-administered SC maintenance dose option remains under review by the FDA.

ObsEva's Linzagolix Approved In Europe For Uterine Fibroids 

The  European Commission has approved ObsEva SA's OBSV Yselty (linzagolix) to manage moderate to severe symptoms of uterine fibroids (UF) in adult women.

The company says Yselty is the only approved oral GnRH antagonist to offer flexible dosing options, with and without additional hormonal therapy, for women suffering from UF.

The US application is under FDA review, with a PDUFA target action date of Sept. 13. 

Alpha Tau Receives Radioactive License To Allow Production In Main Manufacturing Floor

The Israeli Ministry of Environmental Protection has granted Alpha Tau Medical Ltd DRTS an active radioactive license for the use and possession of Thorium-228 and Radium-224, radioactive materials utilized in the production of the Alpha DaRT sources. 

This license will enable the company to achieve a nearly 3x increase in production capacity to yield approximately 90,000 Alpha DaRT sources per year. 

Alpha DaRT is designed to enable highly potent and conformal alpha-irradiation of solid tumors by intratumoral delivery of radium-224 impregnated sources.

FDA Signs Off Global Cord Blood Subsidiary's Early Stage Blood Cancer Trial

Global Cord Blood Corporation's CO Cellenkos Inc has received an FDA nod for its Investigational New Drug application to initiate a Phase 1b study of CK0804.

The study will assess CK0804 as an add-on therapy to ruxolitinib in patients with myelofibrosis who experience a suboptimal response to ruxolitinib. 

Sierra Oncology Files US Application For Bone Marrow Cancer Candidate

Sierra Oncology Inc SRRA has submitted a marketing application to the FDA for momelotinib for myelofibrosis (bone marrow cancer).

The company expects the FDA to respond whether this submission is accepted within 60 days. Assuming the submission is accepted and subsequent approval is granted, the commercial launch of momelotinib is anticipated in 2023.

Click here to access Benzinga's FDA Calendar.

Kazia Therapeutics' Pediatric Brain Cancer Candidate Secures FDA Orphan Drug Tag

The FDA has granted Orphan Drug Designation to Kazia Therapeutics Limited's KZIA paxalisib for atypical rhabdoid/teratoid tumors, a rare and highly aggressive childhood brain cancer.

Shares are trading 8.63% higher at $5.73. 

Clinical Readouts/Presentations

Scholar Rock Holding Corp SRRK: 24-month data from the Phase 2 TOPAZ trial at Cure SMA Research & Clinical Care Meeting.

Offerings

Scholar Rock Holding Corp SRRK announced a registered direct offering of 16.3 million shares and prefunded warrants to purchase 25.5 million shares and accompanying warrants to purchase 10.5 million at $4.90 per share for total gross proceeds of approximately $205 million.

On The Radar

PDUFA Dates

AdComm Meeting for Acadia Pharmaceuticals Inc's ACAD pimavanserin for hallucinations and delusions associated with Alzheimer's disease psychosis.

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