Week Ahead In Pharma: Data Readouts At WORLDSymposium, TCT Meetings & GU Cancers Symposium

(RTTNews) - In the trading week ended February 5, a couple of Alzheimer's treatment stocks staged big gains, thanks to Cassava Sciences Inc. (SAVA) reporting encouraging data of its lead Alzheimer's drug candidate Simufilam on February 2.

Cassava was up over 126% for the week while Anavex Life Sciences Corp. (AVXL), which is also developing a drug for Alzheimer's disease, delivered a return of over 97%.

With its share price rising more than 196%, Ocugen Inc. (OCGN) was the top gainer of the week as the company inked a deal with Bharat Biotech, India's leading Covid vaccine maker.

Now, let's take a look at some of the stocks that merit attention in the coming week.


AVROBIO is a clinical-stage company developing personalized gene therapies to halt or reverse disease with a single dose.

The company is evaluating the following gene therapies:

-- AVR-RD-01, an investigational gene therapy for Fabry disease, is currently under two clinical trials - an investigator-sponsored Phase 1 trial in Canada and a phase II trial in Australia, the U.S. and Canada. -- AVR-RD-02, an investigational gene therapy for Gaucher disease, is being studied in a Phase 1/2 clinical trial. -- AVR-RD-04, an investigational gene therapy for cystinosis, is under a phase I/II trial sponsored by the University of California, San Diego.

New clinical data across the company's Fabry disease, cystinosis and Gaucher disease programs are expected to be presented at the virtual 17th annual WORLDSymposium on February 8.

AVRO closed Friday's (Feb.5, 2021) trading at $17.11, up 6.77%.


REGENXBIO is a clinical-stage biotechnology company developing gene therapies utilizing NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform.

One of the lead product candidates in the pipeline is RGX-121, which is under phase I/II trial for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome. This trial currently has 2 cohorts and enrollment of patients in Cohort 3 is expected to begin this quarter.

Last December, the company announced encouraging interim data from Cohorts 1 and 2 of the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II).

Additional data from Cohorts 1 and 2 of the trial are expected to be presented at the WORLDSymposium on February 8.

RGNX closed Friday's trading at $46.33, up 3.30%.

3. Orchard Therapeutics plc (ORTX)

Orchard Therapeutics is a gene therapy company developing treatments for rare diseases.

One of the lead product candidates in the pipeline is OTL-203, an ex vivo autologous hematopoietic stem cell (HSC) gene therapy, in development for the treatment of mucopolysaccharidosis type I (MPS-I). This investigational product is under a proof-of-concept clinical trial.

Mucopolysaccharidosis type I (MPS I) is a progressively debilitating disorder characterized by neurocognitive impairment, skeletal deformity, loss of vision and hearing, hydrocephalus, and cardiovascular and pulmonary complications.

The preliminary clinical data from the proof-of-concept trial, reported last year, demonstrated sustained hematologic engraftment for all eight patients, with the first two patients treated with at least one year of follow-up showing improved motor function, stable cognitive scores and continued normal growth.

New clinical data from all eight patients treated with OTL-203 for Mucopolysaccharidosis type I (MPS I) are scheduled to be presented at the 2021 WORLDSymposium on February 9.

ORTX closed Friday's trading at $7.39, up 18.81%.

4. Gamida Cell Ltd. (GMDA)

Gamida Cell is a cell therapy company focused on finding cures for blood cancers and serious hematologic diseases.

The company's lead product candidate Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers).

A phase III study evaluated the safety and efficacy of Omidubicel in patients with hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant.

Last May, Gamida Cell reported that the study achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in a patient's recovery from a bone marrow transplant.

Results reported last October showed that the Omidubicel phase III study achieved its secondary endpoints of platelet engraftment, infections, and hospitalizations.

A Biologics License Application for Omidubicel is expected to be submitted in the second half of this year.

On February 9, the company will be presenting the full efficacy and safety results of the phase III clinical trial of Omidubicel at the Transplantation & Cellular Therapy Meetings of the American Society of Transplantation and Cellular Therapy (ASTCT) and Center for International Blood & Marrow Transplant Research (CIBMTR), which is being held virtually from February 8-12, 2021.

GMDA closed Friday's trading at $10.06, up 4.14%.

5. Protalix BioTherapeutics Inc. (PLX)

Protalix BioTherapeutics is the first and only company to gain FDA approval of a protein produced through plant-cell based expression system, ProCellEx.

The company will be presenting 1 year of treatment data from a phase III switch-over clinical trial evaluating Pegunigalsidase Alfa in Fabry disease patients previously treated with Replagal, dubbed BRIDGE, at the 17th Annual WORLDSymposium on February 10.

Pegunigalsidase Alfa, proposed for the treatment of Fabry disease, is under FDA review, with a decision date set for April 27, 2021.

PLX closed Friday's trading at $4.91, up 6.74%.

6. Clovis Oncology Inc. (CLVS)

Clovis Oncology is a biopharmaceutical company focused on the development and commercialization of innovative anti-cancer therapies.

The company's lead drug is Rubraca, which is approved for the treatment of ovarian cancer and prostate cancer. The drug is also being explored as monotherapy and in combination with other anti-cancer agents in various cancers.

Data from clinical studies evaluating Rubraca in metastatic castration-resistant prostate cancer are slated to be presented virtually at the American Society of Clinical Oncology (ASCO) 2021 Genitourinary Cancers Symposium on February 11.

These data underscore the company's continued commitment to fully understanding the clinical role of Rubraca and to accelerating the delivery of transformative therapies to the advanced prostate cancer community, according to Patrick Mahaffy, President and CEO of Clovis Oncology.

CLVS closed Friday's trading at $9.36, up 17.15%.

7. Infinity Pharmaceuticals Inc. (INFI)

Infinity Pharmaceuticals is a biopharmaceutical company whose lead drug candidate is Eganelisib, a first-in-class, oral, small molecule, immuno-oncology therapeutic.

A phase II study of Eganelisib in combination with Opdivo in patients with advanced urothelial carcinoma, dubbed MARIO-275, is underway. Preliminary analysis of the study is scheduled to be presented at the American Society of Clinical Oncology (ASCO) 2021 Genitourinary Cancers Symposium on February 11.

INFI closed Friday's trading at $3.93, up 6.50%.

8. BioXcel Therapeutics Inc. (BTAI)

BioXcel Therapeutics is a clinical-stage biopharmaceutical company utilizing artificial intelligence approaches to develop transformative medicines in neuroscience and immuno-oncology.

The lead immuno-oncology compound in the pipeline is BXCL701, which is currently under two combination therapy clinical trials - a phase I/II trial of BXCL701 in combination with KEYTRUDA for advanced prostate cancer and MD Anderson-led phase II open-label basket trial designed to evaluate the response rate of BXCL701, combined with KEYTRUDA.

An update on the phase I/II trial of BXCL701 in combination with KEYTRUDA for advanced prostate cancer is expected to be presented at the virtual 2021 ASCO Genitourinary Cancers Symposium on February 11.

BTAI closed Friday's trading at $57.75, up 6.18%.

9. Equillium Inc. (EQ)

Equillium is a clinical-stage biotechnology company developing novel products to treat severe autoimmune and inflammatory disorders.

The lead drug candidate is Itolizumab, which is being developed for the treatment of Acute Graft-versus-Host Disease (aGVHD), Lupus/Lupus Nephritis and Uncontrolled Asthma.

Itolizumab is under a phase Ib/II study in acute graft-versus-host disease, dubbed EQUATE. The trial consists of 3 Cohorts, and last November, the company had reported positive interim data from the third cohort of the Phase 1b part of the trial.

The interim data from the EQUATE study are scheduled to be presented at the 2021 TCT Meetings Digital Experience on February 12, with topline data expected in the first half of this year.

EQ closed Friday's trading at $8.49, up 8.99%.

10. Denali Therapeutics Inc. (DNLI)

Denali Therapeutics is a biopharmaceutical company developing treatments for neurodegenerative diseases.

One of the product candidates in the pipeline is DNL310, under a phase I/II clinical study in Hunter Syndrome patients. The study has two cohorts - Cohort A and Cohort B.

Hunter syndrome or mucopolysaccharidosis type 2 is characterized by the accumulation of glycosaminoglycans (GAGs), causing progressive systemic organ failure.

Last November, the company announced positive biomarker proof-of-concept data from five patients enrolled in Cohort A, who received four weekly intravenous doses of DNL310, showing a statistically significant reduction in levels of glycosaminoglycans, with normal healthy levels being achieved in four of five patients.

New data from Cohort A after 12 weeks of treatment is slated to be presented at the WORLD Symposium on February 12.

DNLI closed Friday's trading at $70.57, up 1.25%.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.


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