Sienna Biopharmaceuticals Inc. (SNNA) ended the week as the worst performer in the healthcare sector - with its shares down 77 percent, and there is a reason for its dismal performance. This clinical-stage biopharmaceutical company, focused on developing medicines for skin infections, filed for bankruptcy on September 17, 2019.
Another biopharmaceutical company that disappointed investors is Acceleron Pharma Inc. (XLRN), which on September 16, 2019, announced that it is pulling the plug on the clinical development of ACE-083 in facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder. This decision was taken following the failure of its phase II trial of ACE-083 in patients with FSHD to achieve statistically significant improvements in functional endpoints relative to placebo. However, the stock didn't mind this bit of bad news, and it ended the week almost unchanged.
On the bright side, for investors of Aclaris Therapeutics Inc. (ACRS) and BioCardia (BCDA), there was overall good news.
On September 16, 2019, Aclaris announced that its phase III trial of A-101 45% Topical Solution for the treatment of common warts, dubbed THWART-2, met the primary and all secondary efficacy endpoints, achieving clinically and statistically significant clearance of common warts. The same day, BioCardia announced that it has been asked by the independent Data Safety Monitoring Board to continue its phase III pivotal CardiAMP Heart Failure Trial as planned as there were no safety concerns.
Now, here are some of the pharma/biotech stocks to watch out for in the week starting September 23.
1. Anavex Life Sciences Corp. (AVXL)
Anavex is a clinical-stage biopharmaceutical company developing treatments for Alzheimer's disease, Parkinson's disease, Rett syndrome, and other central nervous system (CNS) diseases.
The Company's lead drug candidate is ANAVEX2-73, which is under a phase 2b/3 study in Alzheimer's, and phase II studies in Parkinson's disease dementia and Rett syndrome.
Watch out for…
The Company will make a presentation at the Ladenburg Thalmann 2019 Healthcare Conference on September 24, 2019.
Detailed data from the U.S. phase II study of ANAVEX2-73 in patients with Rett Syndrome are expected to be presented at the 6th European Rett Syndrome Conference in Tampere, Finland, September 27-28, 2019.
The preliminary results from the first 6-patient cohort of the study were reported on September 16, 2019. Both global efficacy endpoints, the Rett Syndrome Behaviour Questionnaire (RSBQ) and the Clinical Global Impression - Improvement (CGI-I), showed significant improvement with respect to baseline after 7 weeks of treatment with ANAVEX2-73.
AVXL Friday's (Sep.20) trading at $3.53, up 6.65%.
2. UroGen Pharma Ltd. (URGN)
UroGen is a clinical-stage biopharmaceutical company developing treatments to address unmet needs in the field of urology, with a focus on uro-oncology.
The Company's lead investigational therapy is UGN-101 (MitoGel), which is under a phase III trial for the treatment of low-grade Low-Grade Upper Tract Urothelial Carcinoma (UTUC), dubbed OLYMPUS.
Upper tract urothelial cancer, or UTUC, is "cancer affecting the upper urinary tract", which connects the bladder to the kidney and renal pelvis.
Watch out for…
Updated phase III data from the OLYMPUS trial are slated to be presented on September 24, 2019.
Initial results from the OLYMPUS trial were reported in January of this year. The data demonstrated that 57 percent of patients with low-grade upper tract urothelial cancer achieved a complete response, four to six weeks after completion of UGN-101 treatment. All evaluated patients in complete response remained disease-free at six months.
URGN closed Friday's (Sep.20) trading at $34.85, down 1.83%.
3. Rhythm Pharmaceuticals Inc. (RYTM)
Rhythm Pharma is a biopharmaceutical company developing therapies for the treatment of rare genetic disorders of obesity.
The lead drug candidate is Setmelanotide, which has successfully completed two phase III clinical trials in pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity, two ultra-rare genetic disorders. The Company is on track to complete rolling NDA submission for Setmelanotide to the FDA for both POMC and LEPR deficiency obesities in 4Q19 or 1Q20.
A pivotal phase III clinical trial evaluating Setmelanotide in Bardet-Biedl Syndrome and Alström Syndrome is also underway.
Setmelanotide is also being evaluated for the treatment of additional rare genetic disorders of obesity in its Phase 2 basket studies, including POMC and other MC4R pathway deficiency heterozygous obesities, as well as POMC epigenetic disorders.
Early this month, the Company announced its plan to expand its ongoing phase II Basket Study with four additional rare indications -- SRC1 deficiency obesity; SH2B1 deficiency obesity; MC4R deficiency obesity; and Smith-Magenis syndrome.
Rhythm Pharma also offers "Uncovering Rare Obesity", a free genetic testing program that may help determine if individuals have an underlying genetic cause of their severe obesity.
Watch out for…
Update on genetic sequencing results and the phase II Basket Study are expected to be provided on September 25, 2019.
RYTM closed Friday's (Sep.20) trading at $24.51, up 2.81%.
4. Genmab A/S (GMAB)
The FDA decision on the supplemental Biologics License Application for the use of Darzalex in combination with standard-of-care bortezomib, thalidomide, and dexamethasone (VTd) as a treatment for patients newly diagnosed with multiple myeloma who are candidates for autologous stem cell transplant is expected on September 26, 2019.
Darzalex as monotherapy and in various combinations is already approved by the FDA for the treatment of adult patients with multiple myeloma.
The drug is being developed by Johnson & Johnson subsidiary Janssen Biotech Inc. under an exclusive worldwide license from Genmab.
The worldwide net sales of Darzalex as reported by Johnson & Johnson were US$774 million in the second quarter of 2019 compared to US$511 million in the second quarter of 2018, an increase of approximately 51%.
Genmab receives royalties on the worldwide net sales of Darzalex.
GMAB closed Friday's (Sep.20) trading at $20.46, up 0.94%.
5. Aravive Inc. (ARAV)
Aravive is a clinical-stage biotechnology company developing therapies for solid tumors and hematologic malignancies.
The Company's lead drug candidate is AVB-500, which is under a phase 1b/2 trial in patients with platinum-resistant recurrent ovarian cancer.
Watch out for…
Detailed safety, pharmacokinetic, pharmacodynamic and preliminary efficacy data from the phase I portion of phase 1b/2 study of AVB500 in combination with standard chemotherapy in patients with platinum-resistant ovarian cancer are scheduled to be presented at the European Society for Medical Oncology (ESMO) Congress on September 27, 2019.
On July 31, 2019, the Company had announced topline efficacy data from this study showing compelling anti-tumor activity in the first 12 patients.
ARAV closed Friday's (Sep.20) trading at $8.35, up 3.34%. The stock is up 44% over the last 5 trading days.
6. Immunomedics Inc. (IMMU)
Immunomedics is a clinical-stage biopharmaceutical company developing monoclonal antibody-based products for the targeted treatment of cancer.
The Company's lead drug candidate is Sacituzumab govitecan, in development for metastatic triple-negative breast cancer (mTNBC) advanced urothelial cancer (UC) and metastatic non-small cell lung cancer.
In January of this year, the FDA refused to approve Sacituzumab govitecan proposed for the treatment of patients with metastatic triple-negative breast cancer (mTNBC) who previously received at least two prior treatments due to issues related to Chemistry, Manufacturing and Control matters. The Company plans to resubmit the Biologics License Application for Sacituzumab govitecan in mTNBC early fourth quarter 2019.
A phase II study of Sacituzumab govitecan in patients with metastatic solid tumors, including NSCLC, small cell lung cancer, head and neck cancer, and endometrial cancer, dubbed TROPICS-03, is underway.
Another phase II trial, dubbed TROPHY-u-01, is evaluating Sacituzumab govitecan in stage IV urothelial cancer after failure of a platinum-based regimen and/or anti-PD-1/PD-L1-based therapies.
Watch out for…
Interim data from the phase II TROPHY-u-01 trial of Sacituzumab govitecan in metastatic urothelial cancer patients is expected to be presented on September 28, 2019.
IMMU closed Friday's (Sep.20) trading at $16.59, down 2.70%.
7. Checkpoint Therapeutics Inc. (CKPT)
Checkpoint is a clinical-stage, immuno-oncology biopharmaceutical company.
The Company has two drug candidates in clinical development - CK-101 in Phase 1/2 clinical trial for the treatment of patients with EGFR mutation-positive non-small cell lung cancer, and Cosibelimab, in an ongoing phase I clinical trial in checkpoint therapy-naïve patients with selected recurrent or metastatic cancers.
Watch out for…
New clinical data on Cosibelimab will be presented at the European Society for Medical Oncology (ESMO) Congress on September 28, 2019.
CKPT closed Friday's (Sep.20) trading at $2.15, down 1.83%.
8. Calithera Biosciences Inc. (CALA)
Calithera is a clinical-stage biopharmaceutical company developing new therapies for people with cancer and other life-threatening diseases.
The Company's lead drug candidate is Telaglenastat, under phase II trials in combination with everolimus and in combination with cabozantinib in patients with advanced renal cell carcinoma, dubbed ENTRATA and CANTATA, respectively.
Telaglenastat is also being evaluated in multiple combination trials.
Next up in the pipeline is CB-1158 (also known as INCB001158), under phase 1/2 clinical trials in patients with advanced solid tumors. CB-1158 is being developed in collaboration with Incyte Corp.
Watch out for…
New data from the CB-1158 (also known as INCB001158) program are slated to be presented on September 29, 2019.
CALA closed Friday's (Sep.20) trading at $3.88, up 0.78%.
9. ImmunoGen Inc. (IMGN)
ImmunoGen is focused on developing the next generation of antibody-drug conjugates (ADCs) for the treatment of cancer.
The Company's lead drug candidate is Mirvetuximab soravtansine, a potential new treatment for patients with folate receptor alpha (FRa)-positive platinum-resistant ovarian cancer cancer.
In May of this year, the Company announced mature data from the FORWARD II expansion cohort evaluating Mirvetuximab soravtansine in combination with Avastin in patients with folate receptor alpha (FRa)-positive platinum-resistant ovarian cancer. The trial demonstrated that the combination of Mirvetuximab plus Avastin generates significant anti-tumor activity in patients with platinum-resistant disease.
FORWARD II is a phase I/II study of Mirvetuximab in combination with Avastin, carboplatin or Keytruda in patients with FRa-positive platinum-resistant or platinum-agnostic ovarian cancer, primary peritoneal, or fallopian tube tumors, as well as a triplet combination of Mirvetuximab plus Carboplatin and Avastin in patients with platinum-sensitive ovarian cancer.
In March of 2019, the Company announced that its phase 3 FORWARD I trial evaluating Mirvetuximab soravtansine compared to chemotherapy in patients with folate receptor alpha (FRa)-positive, platinum-resistant ovarian cancer did not meet its primary endpoint of progression-free survival (PFS).
Watch out for…
Initial safety and overall response rate data from the Phase 1b FORWARD II triplet study evaluating Mirvetuximab in combination with carboplatin and Avastin in patients with recurrent platinum-sensitive ovarian cancer will be featured in a poster at the European Society for Medical Oncology (ESMO) Congress on September 29, 2019.
IMGN closed Friday's (Sep.20) trading at $3.31, up 5.75%.
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