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Week Ahead In Pharma: FDA Decisions, ASH Data Readouts (LPCN, SYRS, MBIO, CYAD...)

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In the just concluded week, Auris Medical Holding Ltd. (EARS) made the biggest weekly advance, gaining over 180%, thanks to positive in vitro efficacy data of AM-301 in protecting against SARS-CoV-2 infection.

A couple of stocks, say, Merrimack Pharmaceuticals Inc. (MACK), Codiak BioSciences Inc. (CDAK), Salarius Pharmaceuticals Inc. (SLRX), and Cellectar Biosciences Inc. (CLRB) registered gains in excess of 50% for the week.

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

FDA Decision

1. Lipocine Inc. (LPCN)

Lipocine will now get to know the FDA decision on its investigational testosterone replacement therapy, Tlando, during the week of December 7. The decision was originally due on August 28, 2020.

Tlando, formerly known as LPCN 1021, is an oral testosterone product candidate that is designed to help restore normal testosterone levels in hypogonadal men. The regulatory agency had turned down Tlando in June 2016, May 2018, and in November 2019.

It remains to be seen if Tlando will be able to make it to the finish line at least this time.

In March of 2019, the FDA approved Clarus Therapeutics' Jatenzo, an oral testosterone capsule to treat men with certain forms of hypogonadism.

Lipocine is locked in a patent battle with Clarus - with Lipocine claiming that Jatenzo infringes on four of its U.S. patents. In April, the U.S. Court of Appeals for the Federal Circuit had ruled in favor of Lipocine in the patent infringement lawsuit.

LPCN closed Friday's trading at $1.54, down 3.14%.

2. Pfizer Inc.(PFE)/BioNTech(BNTX)

Pfizer and BioNTech's COVID-19 mRNA vaccine BNT162b2 is all set to be reviewed by the FDA's Vaccines and Related Biological Products Advisory Committee on Dec. 10.

The vaccine secured temporary Emergency Use Authorization from the UK regulator, Medicines & Healthcare Products Regulatory Agency, on December 2.

BNT162b2 is a two-dose vaccine, administered as intramuscular injection, given 21 days apart.

In a phase III clinical study, BNT162b2 demonstrated a vaccine efficacy rate of 95% in participants without prior SARS-CoV-2 infection (first primary objective) and also in participants with and without prior SARS-CoV-2 infection (second primary objective) in each case measured from 7 days after the second dose.

PFE closed Friday's trading at $40.34, up 0.62%.

New/Updated Data Presentations

The following are some of the companies that reported new results or updated data from their ongoing clinical trials during the weekend or are scheduled to present data this week at the 62nd American Society of Hematology (ASH) Annual Meeting.

1. Allogene Therapeutics Inc. (ALLO)

The initial results from a phase I trial of ALLO-715 in relapsed/refractory multiple myeloma, dubbed UNIVERSAL, were presented at the ASH Annual meeting on December 5.

According to the trial results, of the 26 patients evaluable for efficacy, ALLO-715 in combination with lymphodepletion drug ALLO-647 demonstrated a 60% overall response rate in the higher cell dose cohort, with 40% achieving a very good partial response or better.

ALLO closed Friday's trading at $33.65, up 4.18%.

2. Celyad Oncology SA (CYAD)

The first results from the dose escalation segment of phase I trial of CYAD-02, an investigational CAR-T therapy, in Relapsed or Refractory Acute Myeloid Leukemia and Myelodysplastic Syndrome patients were presented on December 5 at the ASH Annual Meeting. The results based on 6 patients have demonstrated the safety and tolerability for CYAD-02 in patients with r/r AML and MDS with no dose-limiting toxicity.

CYAD closed Friday's trading at $9.13, up 0.33%.

3. Trillium Therapeutics Inc. (TRIL)

Updated data for TTI-622 were presented at the American Society of Hematology (ASH) Annual Meeting on Dec.5. TTI-622 is under a phase 1a/1b study in patients with advanced relapsed or refractory lymphoma or multiple myeloma.

The results based on 25 patients demonstrated that weekly doses of TTI-622 up to 4 mg/kg are well tolerated with dose-dependent increases in exposure and receptor occupancy. Objective responses were achieved in 5 patients.

The company is also expected to report updated results of TTI-621 at the ASH Annual Meeting on Dec.7. TTI-621 is under a phase 1 study in patients with advanced relapsed or refractory hematologic malignancies.

TRIL closed Friday's trading at $17.04, down 5.80%.

4. Legend Biotech Corp. (LEGN)

The latest data from a phase Ib/II study of ciltacabtagene autoleucel (cilta-cel), an investigational BCMA-directed CAR-T cell therapy, being studied for the treatment of patients with relapsed or refractory multiple myeloma, dubbed CARTITUDE-1, is scheduled to be highlighted at a virtual Key Opinion Leader on December 7.

LEGN closed Friday's trading at $32.39, up 2.99%.

5. Syros Pharmaceuticals Inc. (SYRS)

New data from the ongoing, fully enrolled phase II trial cohort evaluating SY-1425 in combination with azacitidine in RARA-positive relapsed or refractory acute myeloid leukemia (AML) patients and mature data from the ongoing, fully enrolled cohort evaluating SY-1425 in combination with azacitidine in newly diagnosed unfit AML patients who are not suitable candidates for standard chemotherapy were presented on December 5 at the 62nd American Society of Hematology (ASH) Annual Meeting, taking place virtually.

The results demonstrated a 61% Composite complete response rate in RARA-positive newly diagnosed unfit AML patients, with median overall survival of 18 months among responders. The overall response rate was 19% and median overall survival was 5.9 months in RARA-positive relapsed or refractory AML patients.

SYRS closed Friday's trading at $7.76, up 1.57%.

6. Autolus Therapeutics plc (AUTL)

The updated data from phase I/II trial of AUTO1 in adult acute lymphoblastic B cell leukemia and updated data from a phase I/II study of AUTO3 in relapsed/refractory diffuse large B cell lymphoma, dubbed ALEXANDR, are slated to be presented on December 7.

AUTL closed Friday's trading at $10.50, down 0.57%.

7. Constellation Pharmaceuticals Inc. (CNST)

Data update on MANIFEST, a phase II clinical trial of CPI-0610 in patients with myelofibrosis, a rare cancer of the bone marrow that disrupts the body's normal production of blood cells, is slated to be presented on December 7.

CNST closed Friday's trading at $22.64, up 0.49%.

8. Rocket Pharmaceuticals Inc. (RCKT)

Data updates from the Fanconi Anemia, Leukocyte Adhesion Deficiency-I (LAD-I) and Pyruvate Kinase Deficiency programs are scheduled to be discussed on December 7. The data to be presented include results from ongoing global clinical studies of RP-L102 in patients with Fanconi Anemia subtype A; phase I results from phase I/II study of Lentiviral-Mediated Ex-Vivo Gene Therapy for pediatric patients with severe Leukocyte Adhesion Deficiency-I (LAD-I), and phase I study of RP-L301 for Pyruvate Kinase Deficiency.

RCKT closed Friday's trading at $32.08, down 4.95%.

9. MacroGenics Inc. (MGNX)

The clinical update on the registrational study of Flotetuzumab in patients with refractory acute myeloid leukemia (AML) is expected to be presented on December 7. The company's lead drug candidate Margetuximab is under FDA review, proposed in combination with chemotherapy as a treatment for patients with metastatic HER2-positive breast cancer, with a decision expected on December 18.

MGNX closed Friday's trading at $24.00, up 6.24%.

10. Agios Pharmaceuticals Inc. (AGIO)

The updated data from a phase I trial of Mitapivat in sickle cell disease and an overview of the company's phase III development plans for Mitapivat in patients with thalassemia are expected to be presented on December 8.

AGIO closed Friday's trading at $44.70, down 1.22%.

11.Mustang Bio Inc. (MBIO)

The interim phase I/II data on MB-106, a CD20-targeted, autologous CAR T cell therapy, for patients with relapsed or refractory B-cell non-Hodgkin lymphoma is scheduled to be discussed on Wednesday, December 9.

MBIO closed Friday's trading at $3.84, down 0.52%.

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