Pacira Posts Topline Data From Knee Surgery Study With Its Flagship Pain Management Drug

 

 

• Pacira BioSciences Inc 
  PCRX+2.18%+ Free Alerts
   announced topline results from its Phase 3 study of EXPAREL as a single-dose femoral nerve block in the adductor canal for postsurgical regional analgesia in patients undergoing total knee arthroplasty. 
• EXPAREL achieved the study's primary endpoint demonstrating a statistically significant reduction in cumulative pain scores from 0 to 96 hours compared with bupivacaine HCl. 
• EXPAREL also achieved a statistically significant reduction in postsurgical opioid consumption through 96 hours compared with bupivacaine HCl.
• EXPAREL was well tolerated with a safety profile consistent with bupivacaine HCl.  
• Pacira plans to submit a supplemental marketing application to the FDA early next year seeking expansion of the EXPAREL label to include femoral nerve block in the adductor canal.
• The company plans to submit the full results from the Phase 3 study for presentation at future scientific conferences and publication in a peer-reviewed journal.

https://www.benzinga.com/general/biotech/22/09/28777377/pacira-posts-topline-data-from-knee-surgery-study-with-its-flagship-pain-management-drug

Roche doubles down on cytokines

 After disappointments with IL-2 projects, big pharma would be forgiven for giving this approach a swerve. Not so Roche, which today purchased Good Therapeutics for $250m up front to get its hands on that group’s PD-1-regulated IL-2 programme. It will not have gone unnoticed that Roche already has a PD-1-targeted IL-2 asset, RG6279, in phase 1. Perhaps the Swiss group is looking for something even more selective: Good has made much of the fact that so-called conditionally active therapeutics only work when bound to a target molecule. And improved selectivity could widen the therapeutic window for IL-2s, which have been dogged by toxicity. It will be interesting to see if any Good-originated projects can outdo RG6279, but presumably any showdown is some way off, with the former still preclinical. Meanwhile, Good will spin off a new company, Bonum Therapeutics, to work on assets with “other targets in immuno-oncology and beyond” – Good’s pipeline lists PD-L1 and CEA-regulated interferon-alpha as its next most-advanced projects, and the group has mentioned autoimmune diseases, metabolic disease and pain management as potential avenues outside cancer.

To read Evaluate Vantage’s latest report on the IL-2/IL-15 space, click here.

https://www.evaluate.com/vantage/articles/news/snippets-deals/roche-doubles-down-cytokines

Ionis looks average in amyloidosis

 In June Ionis said that its hereditary transthyretin-mediated amyloid polyneuropathy project eplontersen had hit in phase 3; today it gave the magnitude of transthyretin knockdown, and though it is statistically significant the effect is similar to that seen with the three approved drugs. More interestingly, it does not look as good as that seen with Intellia’s much earlier-stage rival project, NTLA-2001. Eplontersen’s safety looks clean, putting it ahead of Ionis’s approved product Tegsedi, but so does NTLA-2001’s, so Intellia could still be a threat. Ionis and its partner Astrazeneca intend to file an NDA by the end of 2022; Stifel believes eplontersen could seize a 30% share within the silencer market in the long term. This is currently split roughly 90%-10% between Alnylam’s Onpattro and Tegsedi.

Cross-trial comparison of therapies for hereditary transthyretin amyloid polyneuropathy
Drug	Description	Trial	Time point	Pbo-adj chg on mNIS+7	Pbo-adj chg on Norfolk QoL-DN	Mean chg in serum TTR
Onpattro (Alnylam)	IV TTR RNAi therapeutic, given every 3wk	Ph3 Apollo	18mth	-34	-21	84%
Amvuttra (Alnylam)	SC TTR RNAi therapeutic, given every 3mth	Ph3 Helios-A	9mth	-17	-16	83%
Tegsedi (Ionis)	SC TTR antisense, given once weekly	Ph2/3 (NCT01737398)	15mth	-20	-12	74%
Eplontersen (Ionis/AZ)	SC TTR antisense, given once monthly	Ph3 Neuro- TTRansform	8mth*	Hit  (p<0.0001)	Hit  (p<0.0001)	82% (p<0.0001)
NTLA-2001 (Intellia)	IV in vivo Crispr-edited project	Ph1 (NCT04601051)	6mth	Not given	Not given	93%**
TTR=transthyretin. mNIS+7=modified neuropathy impairment score +7. Norfolk QoL-DN=Norfolk quality of life questionnaire-diabetic neuropathy. *Interim data; final readout at 66wk. All eplontersen p values are comparisons with historical control. **With 1mg/kg dose. Source: drug labels, company releases. https://www.evaluate.com/vantage/articles/news/trial-results-snippets/ionis-looks-average-amyloidosis

After Disappointing Data, Imara Offloads It Sickle Cell Candidate

 

• Imara Inc 
  IMRA+42.7%+ Free Alerts
   has agreed to divest tovinontrine (IMR-687) and all other assets related to its PDE9 program to Cardurion Pharmaceuticals Inc.
• In addition to $250,000 previously paid by Cardurion upon execution of a non-binding term sheet, the aggregate purchase price consists of an upfront payment of $34.75 million and $60 million as milestone payments.
• In case of termination, the company would be obligated to pay a fee of $1.5 million Cardurion. 
• In April, Imara posted interim analyses of its Ardent Phase 2b trial of tovinontrine (IMR-687) in sickle cell disease (SCD) and Forte Phase 2b trial in beta-thalassemia. 
• No meaningful benefit was observed in transfusion burden in either tovinontrine group compared to the placebo in the beta-thalassemia study.
• Imara announced discontinuing the Ardent and Forte trials and further developing tovinontrine in sickle cell disease and beta-thalassemia.

https://www.benzinga.com/general/biotech/22/09/28775626/after-disappointing-data-imara-offloads-it-sickle-cell-candidate-shares-surge

Praxis Precision Medicines to Start PRAX-222 Study After FDA Clearance

 Praxis Precision Medicines Inc. said Wednesday it plans to start its PRAX-222 clinical study for the treatment of pediatric patients with early-seizure-onset SCN2A developmental and epileptic encephalopathy.

The biopharmaceutical company said the U.S. Food and Drug Administration has cleared the Investigational New Drug application for the initial dose cohort of PRAX-222.

Following collection of the safety and efficacy data from the first cohort of patients in the study, the data will be evaluated and submitted to the FDA to seek authorization for further dose escalation.

Praxis also said it intends to start a PRAX-562 Phase 2 study for the treatment of pediatric patients with SCN2A and SCN8A DEEs outside of the U.S. before the end of 2022, after the FDA placed a clinical hold on its second IND application for PRAX-562.

PRAX-562 has been dosed in over 130 healthy volunteers in completed and continuing studies, including 66 in the U.S. under an initial IND for adult rare headache conditions. Following the clearance of the initial IND last year, Praxis completed the chronic and juvenile toxicology programs and submitted a second IND to the FDA.

Praxis has begun discussions with the FDA to provide clarification about the pre-clinical and clinical data packages in relation to the clinical hold correspondence. Topline results for the PRAX-562 Phase 2 study for the treatment of pediatric patients with SCN2A and SCN8A DEEs are expected in 2023.

https://www.marketscreener.com/quote/stock/PRAXIS-PRECISION-MEDICINE-113902330/news/Praxis-Precision-Medicines-to-Start-PRAX-222-Study-After-FDA-Clearance-41712482/

Amylyx Pharmaceuticals Shares Halted Ahead of FDA AdCom Meeting

 Trading in shares of Amylyx Pharmaceuticals Inc. is halted Wednesday ahead of a key U.S. Food and Drug Administration advisory committee meeting on the company's AMX0035 drug candidate.

The FDA in July said it would reconvene the meeting to focus on additional analyses of study data that Amylyx had submitted on the proposed treatment for amyotrophic lateral sclerosis, a progressive neurodegenerative disease also known as Lou Gehrig's disease that robs patients of their ability to move and speak.

The FDA's advisory panel of neuroscience experts voted 6-to-4 in March that a study hadn't provided sufficient evidence that AMX0035 works, raising fears that the agency would reject the drug.

However, the FDA extended its target action date on AMX0035 to Sept. 29 from June 29 to allow more time to review the additional analyses, raising renewed hopes for the drug, which in June won approval from Canadian health authorities.

Those hopes took a hit last week after the FDA posted briefing documents for Wednesday's meeting that showed continued concerns about the reliability of the drug's study data.

FDA reviewers called some of Amylyx's new analyses "questionable" and noted that the Cambridge, Mass., company is conducting a new Phase 3 study with results expected in late 2023 or early 2024, which they said puts the agency "in a challenging situation of potentially making a regulatory decision that may not be subsequently aligned with the results of the ongoing study."

The advisory committee meeting is slated for noon ET.

https://www.marketscreener.com/quote/stock/AMYLYX-PHARMACEUTICALS-I-131408793/news/Amylyx-Pharmaceuticals-Shares-Halted-Ahead-of-FDA-AdCom-Meeting-41715706/

SpringWorks Expands Collaboration with GSK in Multiple Myeloma

 - SpringWorks to Receive $75 Million Equity Investment with Potential for an Additional $550 Million in Milestone Payments -

- SpringWorks to Supply Nirogacestat for GSK’s Global Blenrep Development Program and to Make Nirogacestat Commercially Available in Markets Where a Combination with Blenrep is Approved -

- SpringWorks to Continue Retaining Full Global Commercial Rights to Nirogacestat

https://finance.yahoo.com/news/springworks-announces-expansion-global-non-135500749.html