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Pipeline News and Updates
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13 Drugs Added to Knowledgebase

6/27/2022

 
We recently added 13 new drugs to the knowledgebase:
  • DTX401 a gene therapy for the treatment of glycogen storage disease type Ia (GSDIa) is being developed by Ultragenyx Pharmaceutical 
  • DTX301 a gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency is being developed by Ultragenyx Pharmaceutical 
  • Donidalorsen an Antisense oligonucleotide for the treatment of hereditary angioedema is being developed by Ionis Pharmaceuticals,
  • VX-548 a selective NaV1.8 inhibitor for the treatment of pain following bunionectomy, or abdominoplasty is being developed by Vertex
  • Foslevodopa and foscarbidopa a dopamine precursor for the treatment of advanced parkinson's disease is being developed by AbbVie
  • Eladocagene exuparvovec a gene therapy for the treatment of severe aromatic L-amino acid decarboxylase (AADC) deficiency is being developed by PTC Therapeutics 
  • AZD8233 an Antisense PCSK9 inhibitor for the treatment of hypercholesterolemia is being developed by Ionis Pharmaceuticals and AstraZeneca
  • Ampreloxetine a norepinephrine reuptake inhibitor for the treatment of neurogenic orthostatic hypotension is being developed by Theravance Biopharma
  • Lifileucel a tumor infiltrating lymphocyte for the treatment of metastatic melanoma is being developed by Iovance Biotherapeutics
  • Ziresovir an RSV fusion (F) protein inhibitor for the treatment of respiratory syncytial virus (RSV) is being developed by Ark Biopharmaceutical
  • JDQ443 a KRAS G12C inhibitor for the treatment of non-small cell lung cancer harboring the KRAS G12C mutation is being developed by Novartis
  • Enoblituzumab an Anti-B7-H3 monoclonal antibody for the treatment of squamous cell carcinoma of the head and neck, non-small cell lung cancer (NSCLC) and urothelial cancer is being developed by MacroGenics
  • Cilofexor a farnesoid X receptor agonist for the treatment of non-alcoholic steatohepatitis (NASH) and primary sclerosing cholangitis is reduce “Off Time” in Parkinson’s disease is being developed by Cerevance
The Prescribe Right Pharmaceutical Pipeline Tracker is the most comprehensive source of clinical information for late-stage investigational drugs. The knowledgebase allows you to track 1,034 pipeline and recently approved drugs supported by 2,611 announced and published study results.
 

Update for June 28, 2022

6/27/2022

 
Regulatory Update

Menarini and Radius submitted an NDA elacestrant for the treatment of ER+/HER2- advanced or metastatic breast cancer.

The EMA’s CHMP recommended approval of lenacapavir and valoctocogene roxaparvovec.

Announced Research Updates

Roche announced that in a 260 to 416 week, 252 patient, Phase II trial, treatment with crenezumab did not slow the decline in cognition in cognitively unimpaired individuals who have no clinical symptoms of Alzheimer’s disease and carry the PSEN1 E280A autosomal dominant mutation.

Ionis and AstraZeneca announced interim results at 35 weeks from the 66-week, 168 patient open-label, Phase III NEURO-TTRansform trial, where treatment with eplontersen reduced serum transthyretin (TTR) concentration and improved the modified Neuropathy Impairment Score +7 (mNIS+7) compared to a historical cohort of placebo patients from the inotersen NEURO-TTR trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).

PTC Therapeutics announced that in a 48-week, 359 patient, Phase III trial, treatment with ataluren the the decline in six-minute walking distance compared to placebo (53 m vs 67.4 m) in patients with Duchenne muscular dystrophy.

Galderma announced that in the 16-week, 274 patient, Phase III, OLYMPIA 2 trial, 38% of patients treated with nemolizumab achieved clearance or almost-clearance of skin lesions, when assessed using the investigator’s global assessment (IGA) score, compared to 11% with placebo in patients with moderate to severe prurigo nodularis. 56% of nemolizumab patients achieved at least a four-point reduction in itch, as measured by the peak-pruritus numerical rating scale (PP-NRS) score, compared to 21% with placebo.

Italfarmaco announced that in the 18-month, 179 patient, Phase III, EPIDYS trial, adding givinostat to corticosteroids resulted in a reduction in the decline of time required to climb four stairs compared to corticosteroids only in ambulatory DMD patients.

Gilead announced that in the 48-week, 150 patient, Phase III, MYR301 trial, undetectable levels of HDV RNA or a 2 log(10) IU/mL decrease and normalization of ALT levels was achieved in 45% of patients treated with bulevirtide 2 mg and 48% with 10 mg compared to 2% with placebo of untreated chronic HDV patients.

Published Research Updates

In the 454 patient, Phase III, PANTHER trial, adding pevonedistat to azactidine did not improve event-free survival compared to azactidine alone in patients with higher-risk myelodysplastic syndromes, chronic myelomonocytic leukemia or low-blast acute myeloid leukemia.

In a 12-day, 62 patient, Phase IIa trial, vaccination with RSVpreF demonstrated 86.7% efficacy in preventing respiratory syncytial virus (RSV) compared to placebo in a challenge study of healthy adults 18 to 50. 

Update for June 22, 2022

6/22/2022

 
Regulatory Update
 
The FDA approved vutrisiran (Amvuttra, Alnylam), on 6/13/2022, for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. Alnylam set WAC for vutrisiran at $463,500 per year.
 
The FDA extended the PDUFA date for omecamtiv mecarbi by three months to allow additional time to review newly submitted pharmacokinetic data. The new PDUFA date is 2/28/2023.
 
Mallinckrodt resubmitted an NDA for terlipressin for the treatment of hepatorenal syndrome - acute kidney injury (HRS-AKI).
 
Addex Therapeutics discontinued development of dipraglurant.
 
The FDA designated rencofilstat an Orphan Drug for treatment of hepatocellular carcinoma.
 
Canada approved the combination of sodium phenylbutyrate and taurursodiol (AMX0035) for the treatment of Amyotrophic Lateral Sclerosis (ALS).
 
Announced Research Updates
 
BridgeBio announced that in the 24-week period 3 of a Phase IIb trial, continued treatment with encaleret resulted in parathyroid hormone levels of 31.3 pg/ml and urine calcium excretion or 189 mg/24 hour in patients with Autosomal Dominant Hypocalcemia Type 1 (ADH1).
 
Roche announced that in a 260 to 416 week, 252 patient, Phase II trial, treatment with crenezumab did not slow the decline in the Alzheimer's Prevention Initiative Autosomal-Dominant Alzheimer's Disease (API ADAD) Composite Cognitive Test Total Score and Episodic Memory Measure: Free and Cued Selective Reminding Task (FCSRT) score in cognitively unimpaired individuals who have no clinical symptoms of Alzheimer’s disease and carry the PSEN1 E280A autosomal dominant mutation.
 
Published Research Updates
 
In a draft report, ICER found evidence to support efficacy for AMX0035 in the treatment of amyotrophic lateral sclerosis (ALS) to be comparable or better compared to standard of care alone. If AMX0035 is priced similar to intravenous edaravone at $171,000 a year, the drug would exceed cost effectiveness thresholds.

Update for June 8, 2022

6/7/2022

 
Regulatory Update
 
The FDA withdrew approval of umbralisib for the treatment of marginal zone lymphoma and follicular lymphoma due to increased mortality seen in the UNITY-CLL trial.
 
The FDA extended the PDFA date for AMX0035 by three months. The new date is 9/29/2022.
 
Gamida completed a rolling BLA for omidubicel.
 
The FDA designated efanesoctocog alfa a Breakthrough Therapy for the treatment of hemophilia A.
 
The EMA designated gavorestat an Orphan Drug for the treatment of galactosemia.
 
Announced Research Updates
 
Sage announced that in the 15-day, 195 patient, Phase III, SKYLARK trial, patients treated with zuranolone had a 15.6 point reduction in their HAMD-17 total score compared to an 11.6 point decrease with placebo in patients with severe postpartum depression. 
 
MaaT Pharma announced that in the 10-week, 21 patient, open-label, dose-ranging, Phase Ib, CIMON trial, treatment with MaaT033 resulted in rapid and persistent engraftment of MaaT033 in patients with acute myeloid leukemia or high-risk myelodysplastic syndrome who have received intensive chemotherapy.
 
Immunic announced that in the Phase II, CALDOSE-1 trial, treatment with vidofludimus did not improve achievement of remission compared to placebo in patients with moderate-to-severe ulcerative colitis
 
Mirati announced that in a 19 patient cohort of the 740 patient, Phase II, KRYSTAL-1 trial, treatment with adagrasib resulted in an ORR of 32% in patients with advanced NSCLC and CNS metastases harboring the KRAS G12C mutation.
 
Cullinan reported interim results from 73 patients enrolled in a Phase I/IIa trial where treatment with CLN-081 resulted in a partial response in 38% of patients with non-small cell lung cancer (NSCLC) harboring epidermal growth factor(EGFR) exon 20 insertion mutations resistant to platinum-based therapy.
 
Biogen announced 12-month data from the VALOR trial, where patients who originally received tofersen had an improvement in clinical function, respiratory function and muscle strength compared to patients who originally received placebo, then switched to tofersen in the open label extension (six-months later).
 
Immutep announced that in the 114 patient, Phase II, open-label TACTI-002 trial, treatment with eftilagimod alpha plus pembrolizumab resulted in an overall response rate (ORR) of 38.6% in patients with non-small cell lung cancer (NSCLC). 
 
Seres announced that in the 24-week, 263 patient, open-label, Phase III, ECOSPOR IV trial, 8.4% of patients treated with SER-109 experienced a recurrence of C. difficile infection at eight weeks in patients with recurrent C. difficile infection. After 24 weeks 86% of patients treated maintained a sustained clinical response.
 
Lilly announced one-year results from ten patients enrolled in the long term extension ADjoin trial, where continued treatment with lebrikizumab resulted in clear skin in 8/10 patients who had achieved EASI-75 while participating in the Phase III, ADvocate 1 or 2 trials.
 
Published Research Updates
 
In the 88 patient, Phase II, open-label, MERECA trial, treatment with ilixadencel plus sunitinib did not improve overall survival compared to sunitinib alone in patients with metastatic renal cell carcinoma.
 
In the 37 patient advanced/metastatic urothelial carcinoma cohort of the open-label, Phase II, PIVOT-2 trial, treatment with bempegaldesleukin plus nivolumab resulted in an ORR of 35%.
 
In the 165 patient, Phase II portion of the MajesTEC-1 trial, treatment with teclistamab resulted in an ORR of 63% in patients with relapsed or refractory multiple myeloma.

Update for June 1, 2022

6/1/2022

 
Regulatory Update
 
The FDA approved tapinarof 1% cream (Vtama, Dermavant Sciences), on 5/24/2022, for the treatment of plaque psoriasis.
 
The FDA accepted the BLA for etranacogene dezaparvoveca, a one-time treatment for hemophilia B, with a potential PDUFA date of 11/24/2022. 
 
The FDA accepted the BLA for mirvetuximab soravtansine to treat folate receptor alpha-high, platinum-resistant ovarian cancer and set a PDUFA date for 11/28/2022.
 
The FDA accepted the NDA for omaveloxolone for the treatment of Friedreich’s ataxia and set a PDUFA date for 11/30/2022.
 
The FDA requested additional, unspecified information for valoctocogene roxaparvovec, so BioMarin is delaying submission of an NDA until the end of September 2022.
 
The FDA extended the PDUFA date for ublituximab, to 12/28/2022, to allow time to review new data requested by the FDA.
 
Announced Research Updates
 
Concert announced that in a 24-week,706 patients, Phase III, THRIVE-AA1 trial, treatment with CTP-543 resulted in achievement of a SALT score of 20 or less in 41.5% of patients treated with 12 mg and 29.6% who received 8 mg compared to 0.8% with placebo in patients with moderate to severe alopecia areata. 
 
Lilly announced that in the 40-week, 544 patient, Phase III, LUCENT-2 trial, patients who achieved remission in the LUCENT-1 induction trial were re-randomized to mirikizumab or placebo. 49.9% of patients treated with mirikizumab achieved or maintained remission compared to 25.1% with placebo
 
Pfizer announced that in the 52-week, 433 patient, Phase III, ELEVATE UC 52 trial, 32.1% of patients treated with etrasimod achieved remission compared to 6.7% with placebo in patients with moderately-to-severely active ulcerative colitis.
 
Elevar announced that in an 80 patient, Phase II trial, treatment with apatinib resulted in an ORR of 15.1% in patients with progressive recurrent or metastatic adenoid cystic carcinoma.
 
SpringWorks announced that in a 142 patient, Phase II trial, treatment with nirogacestat reduced PFS by 41% compared to placebo in patients with desmoid tumors.
 
Published Research Updates
 
In the 12-week, 1,162 patient, Phase III, LUCENT-1 trial, 24.2% of patients treated with mirikizumab achieved remission compared to 13.3% with placebo in patients with moderate-to-severe active ulcerative colitis. 
 
The Phase III, BRIGHTER trial was discontinued after 714 patients when an interim analysis found no improvement in the overall survival from adding napabucasin to nab-paclitaxel and gemcitabine compared to the two drugs alone in the treatment of metastatic pancreatic ductal adenocarcinoma.

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