Novartis iptacopan shows benefit in blood disorder paroxysmal nocturnal hemoglobinuria

-- First-in-class, oral, targeted factor B inhibitor iptacopan substantially 
      reduced both intra- and extravascular hemolysis when given as monotherapy 
      in a Phase II study of anti-C5 naïve paroxysmal nocturnal 
      hemoglobinuria (PNH) patients1 
 
   -- New results are promising for potential use of iptacopan as monotherapy 
      in PNH, a rare and life-threatening blood disorder2,3; results from a 
      previous Phase II study showed iptacopan substantially improved 
      hematological response as add-on to standard-of-care (eculizumab)4 
 
   -- The FDA has granted Breakthrough Therapy Designation to iptacopan for 
      PNH5; it also has received orphan drug designation for PNH from both the 
      FDA and EMA6 
 
   -- Iptacopan is also in development for several rare renal conditions with 
      complement system (part of the innate immune system) involvement, 
      targeting a key driver of these diseases7,8 
 
   -- Recently presented Phase II data showed iptacopan reduced proteinuria and 
      stabilized kidney function in IgA nephropathy (IgAN)9, and improved 
      estimated glomerular filtration rate (eGFR) slope and stabilized kidney  

function in C3 glomerulopathy (C3G)10

https://www.marketscreener.com/quote/stock/NOVARTIS-AG-9364983/news/Press-Release-nbsp-Novartis-investigational-oral-therapy-iptacopan-LNP023-shows-benefit-as-monot-35577171/ 

SIEMENS HEALTHINEERS: Credit Suisse gives a Buy ratin

 In a research note published by Max Yates, Credit Suisse advises its customers to buy the stock. The target price is still set at EUR 55.

https://www.marketscreener.com/quote/stock/SIEMENS-HEALTHINEERS-AG-42379342/news/SIEMENS-HEALTHINEERS-nbsp-Credit-Suisse-gives-a-Buy-rating-35578395/

Gilead : FDA Oks Epclusa, Expanding Pediatric Indication in Chronic Hepatitis C

 Gilead Sciences Inc. (GILD) said that the U.S. Food and Drug Administration has approved new formulation of Epclusa, expanding pediatric indication to treat children ages 3 and older with Chronic Hepatitis C.

The company noted that the new oral pellet formulation enables treatment in early childhood with pangenotypic, panfibrotic regimen.

The FDA approved a New Drug Application for two strengths of an oral pellet formulation of Epclusa (sofosbuvir 200 mg/velpatasvir 50 mg and sofosbuvir 150 mg/velpatasvir 37.5 mg) developed for use by younger children who cannot swallow tablets. The recommended dosage of Epclusa in children ages 3 years and older is based on weight.

Treatment with Epclusa for 12 weeks was approved in patients without cirrhosis or with compensated cirrhosis (Child-Pugh A), and in combination with ribavirin for patients with decompensated cirrhosis (Child-Pugh B or C). Sofosbuvir/velpatasvir is the only protease inhibitor-free, pangenotypic HCV regimen approved for patients as young as 3 years of age.

https://www.nasdaq.com/articles/gilead-%3A-fda-oks-epclusa-expanding-pediatric-indication-to-treat-children-with-chronic

Roche Evrysdi shows improved motor function in babies and safety in SMA

 

• Data from JEWELFISH, the first trial in a diverse population aged 1 to 60 years with SMA who received prior treatment, showed a consistent safety profile and >2-fold increase in SMN protein levels
• Pre-symptomatic babies with SMA treated with Evrysdi for at least one year were able to sit, stand and walk in preliminary data from RAINBOWFISH study
• Evrysdi has proven efficacy in adults, children and babies two months and older and is now approved in 44 countries worldwide

https://www.globenewswire.com/news-release/2021/06/11/2245634/0/en/New-Roche-data-for-Evrysdi-show-improved-motor-function-in-pre-symptomatic-babies-after-one-year-and-confirm-safety-profile-in-previously-treated-people-with-spinal-muscular-atroph.html

CytRx, Orphazyme await FDA decision

 Shares of Orphazyme A/S (ORPH) soared as much as over 1,300%, touching an intraday price of $77.77, yesterday, as the company awaits FDA word on its lead drug candidate MIPLYFFA.

MIPLYFFA, known generically as Arimoclomol, is proposed for the treatment of Niemann-Pick type C, a rare inherited lysosomal storage disorder, with the regulatory agency's decision expected on June 17, 2021.

If approved, MIPLYFFA would become the first approved therapy in the U.S. for people with Niemann-Pick disease Type C (NPC) and the drug could achieve peak global sales of $340 million, according to analysts.

Orphazyme acquired the rights to Arimoclomol from CytRx Corp. (CYTR) in 2011 by paying a one-time, upfront payment of $150,000. CytRx is also entitled to receive up to a total of $120 million in milestone payments upon the achievement of certain pre-specified regulatory and business milestones, as well as royalty payments based on a specified percentage of any net sales of products derived from Arimoclomol.

https://www.nasdaq.com/articles/did-you-ride-the-rally-in-orphazyme-2021-06-11

AbbVie: 4-year Phase 3 Venetoclax Trial Followup Continues Longer Leukemia Survival

  AbbVie (ABBV) announced Friday results from a four-year follow-up analysis of the Phase 3 CLL14 trial. The new data shows that AbbVie's VENCLYXTO/VENCLEXTA fixed duration combination demonstrates sustained progression-free survival or PFS in chronic lymphocytic leukemia or CLL patients after three years off treatment.

CLL is a slow-growing form of leukemia, or blood cancer, in which too many immature lymphocytes are found predominantly in the blood and bone marrow.

The data showed that previously untreated patients with CLL with coexisting conditions who were treated with VENCLYXTO/VENCLEXTA (venetoclax) plus obinutuzumab continued to show longer PFS and higher rates of undetectable minimal residual disease or MRD compared to patients receiving a standard of care chemoimmunotherapy regimen of obinutuzumab and chlorambucil.

The data were presented for the first time during the virtual 26th European Hematology Association Annual Congress.

https://www.nasdaq.com/articles/abbvie%3A-4-year-follow-up-analysis-of-phase-3-venetoclax-trial-continues-to-show-longer-pfs

Bayer’s two-pronged cell/gene therapy assault on Parkinson’s enters clinic

 Bayer subsidiary BlueRock Therapeutics has dosed the first Parkinson’s disease patient in a phase 1 trial of its cell-based therapy DA01, which aims to replenish the neurons lost in people with the neurodegenerative disorder.

At the same time, the German group’s Asklepios BioPharma (AskBio) unit has started recruiting patients for a phase 1b trial of its gene therapy for Parkinson’s, another treatment with the potential to stop and possibly reverse the decline in motor control caused by the disease.

The start of trials is a milestone for Bayer’s ambition to develop cell and gene-based therapies for Parkinson’s that could replace the dopamine-producing neurons in the midbrain area that are progressively lost as the disease progresses.

In the BlueRock trial, an unnamed patient has now been administered the first dose of pluripotent stem cell-derived dopaminergic neurons – MSK-DA01 – via a surgical transplant procedure into the putamen area of the brain, which atrophies in Parkinson’s.

After transplant, the 10 subjects in the study will take immunosuppressant drugs to prevent their immune system from rejecting the transplant. They will be followed up for two years, looking at safety, tolerability, the survival of the cells and the impact on Parkinson’s symptoms, with final results due in November 2023.

AskBio’s approach meanwhile will involve using an adeno-associated virus (AAV) vector to deliver a gene for human glial cell line-derived neurotrophic factor (GDNF) into the neurons in the putamen.

GDNF is a growth factor that in animal studies has been shown to stimulate the regeneration of midbrain neurons.

In the 12-patient phase 1 study, subjects with early- and late-stage Parkinson’s will receive a single dose of  the gene therapy, and then be followed up for 18 months to gauge its effects on dopaminergic neurons, as well as motor and non-motor symptoms. With a readout expected at the end of 2022. Safety will be monitored for five years.

“The potential of BlueRock and AskBio’s clinical candidates to treat Parkinsons disease could be immense,” said Wolfram Carius, head of cell and gene therapy at Bayer.

“For the first time, it might be possible to stop and reverse this degenerative disease and truly help patients with their high unmet medical need,” he added. Parkinson’s is the most common neurodegenerative movement disorder, impacting more than 10 million people worldwide.

Bayer agreed a $600 million deal in 2019 to take full control of BlueRock, which was formed in 2016 as a joint venture between the drugmaker’s Leaps by Bayer e]venture capital group and private equity firm Versant Venture.

It followed that deal with a $4 billion agreement to acquire AskBio a year later, cementing its ambition in the regenerative medicines category. Bayer has said it wants both companies to operate independently in order to preserve their entrepreneurial culture.

Other players have been looking at cell and gene therapies for Parkinson’s disease, including Voyager Therapeutics which has indicated it plans to start trials of its candidate later this year.

Voyager’s first gene therapy – for Huntington’s disease – was placed under a clinical hold by the FDA with a request for more manufacturing information but was cleared to start trials in April.

Meanwhile, Prevail Therapeutics is developing a gene therapy for a form of Parkinson’s that is caused by mutations in the GBA1 gene, and Aspen Neuroscience is working on a personalised stem cell therapy that would use a patient’s own cells, sidestepping the need for immunosuppressant drugs.

https://pharmaphorum.com/news/bayers-two-pronged-cell-gene-therapy-assault-on-parkinsons-enters-clinic/