$GERN Your Daily Pharma Scoop: Geron Update, Rigel

$GERN Your Daily Pharma Scoop: Geron Update, Rigel Setback, Bio-Path Results

Summary
Geron shares are up more than 54% in the last one month.

Rigel suffered a setback as proof-of-concept study evaluating its ostamatinib failed.

Bio-Path announced positive preliminary results from mid-stage study of prexigebersen (BP1001) and low-dose cytarabine in AML.

Note: The Total Pharma Tracker will present a discussion session with Geron author MedTechBio on April 5 at 9am EST in our members-only chatroom. Everyone can participate for free by joining our Free Trial.

Analysis focus: Geron
Geron (NASDAQ:GERN) shares have surged more than 54% in the last month. The gains have been driven by an update on clinical studies being conducted with Geron’s lead investigational candidate, Imetelstat.

Geron out-licensed Imet to Janssen (NYSE:JNJ) back in 2014. This was after a period of ups and downs that even saw the FDA placing a clinical hold on clinical studies with Imet. Since out-licensing Imet, all eyes have been on whether Janssen will move further with the investigational candidate.

Last month, Geron provided an update on Imet that has reassured longs. This was also the reason behind the surge in mid-March. Geron noted that development partner Janssen has already completed a third internal data review of the IMbark study, which is evaluating two doses of Imetelstat in intermediate-2 or high-risk myelofibrosis (MF) patients who are refractory to or have relapsed after treatment with a JAK inhibitor. The data review was based on a January 2018 data cut and it will enable a protocol amendment to allow for long-term treatment and follow up of patients. Furthermore, with the completion of the IMbark protocol-specified primary analysis, Janssen must notify Geron as to whether it is going to maintain maintain the license rights and continue the development of imetelstat in any indication. Of course, this is the big catalyst Geron longs have been waiting for. Last month, Geron provided a definite timeline. The company expects the protocol-specified primary analysts for the IMbark study to commence by the end of the second quarter of 2018. Based on this, Gerone expects the decision from Janssen on whether to maintain the license rights to occur by the end of the third quarter of 2018.

If Janssen does opt in, the upside in Geron could be significant. This is a binary event whereby the potential reward are going to be significant but the risk is also high.

Stocks in News: Analysis of RIGL, BPTH
Rigel Pharmaceuticals (NASDAQ:RIGL) announced that a phase 2 proof-of-concept study evaluating its ostamatinib, branded as Tavalisse, in patients with IgA nephropathy failed to achieve the primary endpoint.

Analysis: The phase 2 study did not show a statistically significant change in the level of protein in the urine (proteinuria) in the treatment group compared to placebo. In a subset of patients, those with greater than 1 gram/day of proteinuria at baseline showed the most separation from placebo. However, the treatment effect was not found to be statistically valid. The news has sent RIGL shares down sharply.

Bio-Path Holdings (NASDAQ:BPTH) announced positive preliminary results from a phase 2 study evaluating the combination of prexigebersen (BP1001) and low-dose cytarabine in patients with acute myeloid leukemia (AML).

Analysis: Almost half (47%) of 17 evaluable patients receiving the combination showed early anti-leukemic activity, including four complete responders, two with significantly reduced bone marrow blasts and four with stable disease. Based on recommendations from investigators, the company is amending the protocol for a new dosing schedule in which a higher dose of prexigebersen is administered prior to cytarabine at day 10 (instead of day 4). A cohort including hypomethylating agent decitabine will also be added.

In other news
Celgene (NASDAQ:CELG) announced that its COO, Scott Smith, would be leaving the company, effective immediately. The move comes as a surprise. Smith had been made COO almost a year ago.

AstraZeneca (NYSE:AZN) announced that the FDA has accepted for Priority Review its marketing application seeking approval for moxetumomab pasudotox for the treatment of adult patients with hairy cell leukemia (HCL) who have received at least two prior lines of therapy. The agency's action date is in Q3.

Amgen (NASDAQ:AMGN) announced that the European Commission (EC) has approved XGEVA (denosumab) for the prevention of skeletal-related events in adults with advanced malignancies involving bone, including patients with bone metastases from solid tumors and those with multiple myeloma.

Regeneron (NASDAQ:REGN) and its partner Sanofi (NYSE:SNY) announced that the European Medicines Agency (EMA) has accepted for review the latter's marketing application seeking approval for DUPIXENT (dupilumab) as add-on maintenance treatment in certain adults and adolescents (at least 12 years old) with inadequately controlled moderate-to-severe asthma.

Galmed Pharmaceuticals (NASDAQ:GLMD) announced that it has signed an agreement with institutional investor Biotechnology Value Fund, L.P. for the direct sale of 1 million ordinary shares at $6 per share. The investor will also receive one-year warrants to purchase up to 1M ordinary shares at $15 per share. Closing date is April 5.

AstraZeneca and Merck (NYSE:MRK) announced that the EMA has validated for review the Marketing Authorization Application (MAA) for LYNPARZA (olaparib) for use in patients with BRCA-mutated, HER2-negative metastatic breast cancer, previously treated with chemotherapy in the neoadjuvant, adjuvant or metastatic setting.

Separately, AstraZeneca also got a nod from the FDA for once-weekly BYDUREON (exenatide extended-release) injectable suspension as add-on therapy to basal insulin in adults with type 2 diabetes (T2D) with inadequate blood sugar control.

Motif Bio (NASDAQ:MTFB) announced that it has commenced a rolling submission of a New Drug Application (NDA) to the FDA for iclaprim, a targeted, Gram-positive investigational antibiotic, for the treatment of acute bacterial skin and skin structure infections. The NDA submission is expected to be completed during Q2.

Pfizer (NYSE FE) announced that it has signed an asset contribution agreement with Allogene Therapeutics, a CAR-T developer, under which it will contribute its portfolio of assets aimed at speeding up development of allogeneic CAR T therapies. Pfizer has a 25% stake in Allogene.

Puma Biotechnology (NYSE BYI) announced that it has signed an agreement with Pint Pharma International S.A. granting the latter rights to commercialize NERLYNX (neratinib) in Latin America, including Mexico.

Myovant Sciences (NYSE:MYOV) announced that it has entered into a share purchase agreement with Roivant Sciences, pursuant to which the Company will issue and sell to Roviant 1.1 million common shares at a price of $20.27 per share in a private placement for the estimated gross proceeds of approximately $22.5 million. Myovant will use the net proceeds from the offering to fund its clinical development programs and for the commercial launch of relugolix.

Geron discussion
The Total Pharma Tracker will present a discussion session with Geron author MedTechBio on April 5 at 9am EST in our members-only chatroom. Everyone can participate for free by joining our Free Trial. Simply discontinue the trial if you do not want to continue with the service, and are not interested in future discussion sessions like this, for example, of our upcoming one-on-one with a well-known NASH authority that will also take place in April.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

https://seekingalpha.com/article/4160937-dail...th-results

Article By: Avisol Capital Partners
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