The Biotech stocks ETF IBB is pushing up against its 200 day moving average. The 200 day moving average has been a major resistance level in November, December, and January.
Notice the huge surge on the CMF as we test 200 day moving average resistance, nice! This suggests we have a real chance of breaking through this major resistance level.
In hindsight I should have given more respect to the Bullish Ladder Bottom at the end of December on IBB:
This upcoming week we have the WORLD Symposium where we will get lots of clinical data updates on various trials.
8 Biotech Stocks To Watch For Week of February 4 2019
ABO-102 for Sanfilippo syndrome type A (MPS IIIA). Phase 1/2 safety and tolerability data to be presented at WORLD Symposium on February 6, 2019.
Data from ongoing clinical trials for lysosomal storage diseases and novel AIM™ chimeric AAV-based vector gene therapy programs will be presented at WORLD Symposium™, being held February 4-8, 2019 in Orlando, FL.
“The data to be presented at the WORLD Symposium™ will highlight study results on biodistribution and tissue tropism of the next-generation AIM™ AAV vector platform in Pompe and Fabry diseases, as well as data from our programs in Sanfilippo syndrome type A and CLN3 disease,” said João Siffert, M.D., Interim Chief Executive Officer of Abeona. “These new supportive data underscore the potential of our novel gene therapies for people living with lysosomal storage diseases.”
“Our AIM™ program continues to demonstrate how novel AAV capsids can target specific body tissues with high efficiency,” said Timothy J. Miller, Ph.D., President and Chief Scientific Officer.
ABEO stock shows an ok setup pattern. Prices have been consolidating lately and the volatility has been reduced.
AVR-RD-01 for Fabry disease. Phase 1 study update and two-year data to be released for first patient at WORLD Symposium on February 6, 2019.
AVROBIO’s analyst and investor event will expand on the presentations at the conference and highlight AVROBIO’s progress with the Phase 1 study and Phase 2 FAB-201 study1 for its lead clinical program in Fabry disease; the Company’s optimized gene therapy platform; and other gene therapy pipeline programs in Gaucher, Cystinosis and Pompe. Featured speakers at the analyst and investor event include AVROBIO management and Dr. Mark Thomas, a specialist in Fabry disease and physician at the Department of Nephrology, Royal Perth Hospital and Clinical Professor at the University of Western Australia Medical School.
AVR‑RD‑01 is a gene therapy being investigated in patients with Fabry disease. Designed to be a one-time therapy, its mechanism of action is by inserting the GLA gene that encodes functional galactosidase A (AGA, the enzyme that is deficient in Fabry disease), with the goal of enabling continuous AGA production and distribution to tissues and organs.
AVRO shows an ok setup pattern. Prices have been consolidating lately. There is a very little resistance above the current price.
SB-318 for MPS Type 1. Phase 1/2 preliminary safety and biochemical measurements due at the WORLD Symposium on February 7, 2019.
Sangamo Therapeutics’ inherited metabolic diseases development programs will be presented at WORLD Symposium, an annual conference dedicated to lysosomal diseases being held February 4-8th, 2019 at the Hyatt Regency Orlando in Orlando, Florida.
The WORLD Symposium program includes two platform presentations from clinical trials of Sangamo’s zinc finger nuclease (ZFN)-mediated in vivo genome editing product candidates, SB-318 and SB-913, which are being evaluated for the treatment of mucopolysaccharidosis type I (MPS I) and type II (MPS II).
The SB-913 (MPS II) presentation is expected to include interim data on safety and biochemical measurements at up to 24 weeks from six subjects enrolled in the three dose cohorts of the CHAMPIONS Study. The SB-318 (MPS I) presentation is expected to describe the scientific rationale for SB-318, the clinical trial design, and preliminary safety and biochemical measurements at up to four weeks from the first three patients enrolled in the EMPOWERS Study.
Sangamo expects to provide additional SB-913 and SB-318 updates in 2019 as data accumulate and mature in these clinical development programs.
SGMO stock does not present a good setup right now. Prices have already moved higher and an entry at this level would mean chasing SGMO stock higher. It is probably better to wait for more of a consolidation pattern before taking a long entry.
Pegunigalsidase alfa (PRX-102) for Fabry disease. Phase 3 PK data from open label trial to be presented at the WORLD Symposium on February 7, 2019.
Protalix BioTherapeutics will present preliminary pharmacokinetic (PK) data from its phase III BRIGHT study of pegunigalsidase alfa for the treatment of Fabry disease at the 15th Annual WORLD Symposium™ 2019. The conference will take place February 4-8, 2019 at the Hyatt Regency Orlando, Orlando, Florida.
Pegunigalsidase alfa, or PRX-102, is the Company’s plant cell-expressed recombinant, PEGylated, cross-linked α-galactosidase-A candidate for the treatment of Fabry disease. The BRIGHT study is an open-label switchover study to assess the safety, efficacy and pharmacokinetics of pegunigalsidase alfa 2 mg/kg administered every 4 weeks in Fabry patients previously treated with an enzyme replacement therapy: Fabrazyme® or Replagal®. The data to be presented will include pharmacokinetics data from 15 patients undergoing treatment with PRX-102.
In addition to the oral presentation, the following poster presentations will be made at the conference with respect to pegunigalsidase alfa:
“Once every 4 weeks – 2 mg/kg of pegunigalsidase alfa for treating Fabry disease; Preliminary results of a phase 3 study” to be presented by principal investigator Mr. Myrl D. Holida, PA, of the University of Iowa Health Care in Iowa City, Iowa, a principal investigator in the Company’s clinical trial of PRX-102. Poster presentation is scheduled from 4:30-6:30 PM ET on Tuesday, February 5, 2019 (Poster # 160).
“Pegunigalsidase alfa for the treatment of Fabry disease: Preliminary results from a phase III open label, switch over study from agalsidase alfa,” to be presented by Prof. Ales Linhart of the Všeobecná fakultní nemocnice v Praze, Prague Czech Republic, a principal investigator in the Company’s clinical trial of PRX-102. Poster presentation is scheduled from 4:30-6:30 PM ET on Tuesday, February 5, 2019 (Poster # 218).
“Analysis of the baseline characteristics of Fabry disease patients screened for the pegunigalsidase alfa phase III BALANCE study,” to be presented by Prof. David Warnock, Professor of Nephrology at the University of Alabama Birmingham, Birmingham, Alabama. Poster presentation is scheduled from 4:30-6:30 PM ET on Wednesday, February 6, 2019 (Poster # 373).
Copies of the oral presentation and the posters will be available on the Company’s website under the Presentation tab in the Investors section at the time of the presentations.
Protalix is a biopharmaceutical company focused on the development and commercialization of recombinant therapeutic proteins expressed through its proprietary plant cell-based expression system, ProCellEx®. Protalix’s unique expression system presents a proprietary method for developing recombinant proteins in a cost-effective, industrial-scale manner. Protalix’s first product manufactured by ProCellEx, taliglucerase alfa, was approved for marketing by the U.S. Food and Drug Administration (FDA) in May 2012 and, subsequently, by the regulatory authorities of other countries. Protalix has licensed to Pfizer Inc. the worldwide development and commercialization rights for taliglucerase alfa, excluding Brazil, where Protalix retains full rights. Protalix’s development pipeline includes the following product candidates: pegunigalsidase alfa, a modified version of the recombinant human alpha-GAL-A protein for the treatment of Fabry disease; OPRX-106, an orally-delivered anti-inflammatory treatment; alidornase alfa for the treatment of Cystic Fibrosis; and others. Protalix has partnered with Chiesi Farmaceutici S.p.A., both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa.
PLX stock does not present a decent entry opportunity at the moment because the stock has already been extended to the upside. For a nice entry it is better to wait for a consolidation.
Marzeptacog alfa for Hemophilia. Phase 2/3 updated interim data is due February 8, 2019 at EAHAD.
Catalyst Biosciences will have oral and poster presentations at the 12TH Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) being held in Prague from February 6-8, 2019.
Dr. Howard Levy, chief medical officer of Catalyst, will deliver an oral presentation on the most up to date data from the Company’s ongoing Phase 2/3 trial of marzeptacog alfa (activated) (MarzAA) for the treatment of hemophilia A or B with inhibitors. Only 10 oral presentations were selected from all submitted abstracts. A poster on quality of life measure improvement of subjects in the ongoing Phase 2/3 trial of MarzAA for the treatment of hemophilia A or B with inhibitors will be presented by Drs. Frank Booth and Howard Levy. Dr. Grant Blouse, vice president of translational research, will present a poster on the preclinical efficacy of CB 2679d-GT, Catalyst’s AAV-based FIX-CB 2679d gene therapy candidate in hemophilia B mice.
Catalyst Biosciences is a clinical-stage biopharmaceutical company developing novel medicines to address hematology indications. Catalyst is focused on the field of hemostasis, including the subcutaneous prophylaxis of hemophilia and facilitating surgery in individuals with hemophilia.
CBIO stock shows a good setup pattern. Prices have been consolidating lately. There is a very little resistance above the current price. We notice that large players showed an interest for CBIO stock in the last couple of days, which is a good sign.
Esketamine for the treatment of resistant depression. PDUFA date is estimated to occur around March 3, 2019 assuming priority review. FDA Advisory Committee Meeting is coming on February 12, 2019. Meeting notes likely will be released on February 8, 2019.
On February 12, 2019, a joint meeting of the Psychopharmacologic Drugs Advisory Committee (PDAC) and the Drug Safety and Risk Management (DSaRM) Advisory Committee will occur. The committees will discuss efficacy, safety and risk-benefit profile of new drug application (NDA) 211243, esketamine 28 mg single-use nasal spray device, submitted by Janssen Pharmaceuticals, Inc., for the treatment of treatment-resistant depression.
The FDA intends to make background material available to the public no later than two (2) business days before the meeting. If FDA is unable to post the background material on its web site prior to the meeting, the background material will be made publicly available at the location of the advisory committee meeting, and the background material will be posted on FDA’s Web site after the meeting.
JNJ stock is an ok setup. Price movement has been a little bit too volatile to find a nice entry and exit point. It is probably a good idea to wait for a consolidation first.
AMT-061 for Hemophilia B. Phase 2b top-line positive data released November 15, 2018 with 31% FIX level. Updated data is due at EAHAD on February 8, 2019.
uniQure N.V. a gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it will participate in the following upcoming conferences in February:
12th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) in Prague, Czech Republic
Annette von Drygalski, M.D., Pharm.D., RMSK, associate clinical professor of medicine and director of the Hemophilia and Thrombosis Treatment Center, University of California San Diego, will deliver an oral presentation on updated clinical data from the ongoing Phase IIb dose-confirmation study of AMT-061 in hemophilia B on Friday, February 8. The presentation will include at least 12 weeks of follow-up on all three patients in the study.
uniQure management will host an investor call and webcast with Dr. von Drygalski that morning at 8:30 a.m. ET. Details for the conference call and webcast will be forthcoming.
MassBio Member Forum in Cambridge, MA
Dan Leonard, director of global patient advocacy at uniQure, will participate in a panel discussion as part of the MassBio Forum “Gene Therapy Then and Now: Tales of Perseverance from its Inception to Today” on Tuesday, February 12 from 8:00 to 10:00 a.m. ET.
CHDI 14th Annual Huntington’s disease Conference in Palm Springs, California
Pavlina Konstantinova, Ph.D., senior director of new therapeutic target discovery at uniQure, will present as part of the afternoon HTT lowering session on Tuesday, February 26 between 2 p.m. and 5:35 p.m. PT. Her presentation, “Gene Therapy for Huntington’s Disease: Silencing the Villain,” will focus on uniQure’s proprietary gene-silencing technology platform, miQURETM. Following the speakers’ session, she will participate in a panel discussion.
Leerink Global Health Care Conference in New York City
uniQure will attend the Leerink Global Health Care Conference from February 27 through March 1. Matt Kapusta, chief executive officer at uniQure, will participate in a fireside chat on Friday, March 1 at 9:30 a.m. ET. The live webcast can be accessed through the link displayed in the Investor section of the uniQure website. The webcast replay will be available for at least 72 hours following the live event.
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with liver/metabolic, central nervous system and other severe genetic diseases.
QURE does not present a decent entry opportunity at the moment. Prices have been extended to the upside lately. For a nice entry it is better to wait for a consolidation.
Iclaprim – REVIVE-2 for acute bacterial skin and skin structure infections (ABSSSI). The PDUFA date is set for February 13, 2019.
Back on August 14, 2018, Motif Bio announced that the FDA accepted the New Drug Application (NDA) for iclaprim, a targeted, Gram-positive investigational antibiotic, for the treatment of acute bacterial skin and skin structure infections (ABSSSI). This means that the FDA has determined that the application is sufficiently complete to perform a substantive review. The NDA has been granted Priority Review, and the FDA has set a target decision date under the Prescription Drug User Fee Act (PDUFA) of February 13, 2019.
“The NDA acceptance by the FDA is an important milestone for Motif Bio and reflects the dedication and commitment of our team who have worked tirelessly to accomplish this,” said Graham Lumsden, Chief Executive Officer of Motif Bio. “We believe that, if approved, iclaprim could be an important new treatment option for patients with serious skin infections. We look forward to working closely with the FDA as we move through the review process with the goal of bringing iclaprim to patients as quickly as possible.”
More than 3.6 million patients with ABSSSI are hospitalised annually in the U.S. It is estimated that up to 26% of hospitalised ABSSSI patients have renal impairment. Hospitalised patients with obesity, diabetes and/or poor kidney function are particularly vulnerable to vancomycin-associated kidney injury. Many standard of care Gram-positive antibiotics are not suitable for treatment of hospitalised ABSSSI patients with these conditions due to efficacy and/or safety issues.
The NDA includes data from two Phase 3 trials (REVIVE-1 and REVIVE-2) evaluating iclaprim for the treatment of patients with ABSSSI. In both trials, iclaprim achieved the primary endpoint of non-inferiority (NI) (10% margin) compared to vancomycin, the current standard of care, at the early time point (ETP), 48 to 72 hours after the start of administration of the study drug, in the intent-to-treat (ITT) patient population. Iclaprim also achieved NI (10% margin) at the test of cure endpoint, 7 to 14 days after study drug discontinuation, in the ITT patient population.
Iclaprim has received Qualified Infectious Disease Product (QIDP) designation from the FDA. If iclaprim is approved as a new chemical entity with QIDP designation, it will be eligible for 10 years of market exclusivity in the U.S. starting from the date of approval, under the Generating Antibiotic Incentives Now Act (the GAIN Act).
MTFB stock does not present a decent entry opportunity at the moment. Price movement has been a little bit too volatile to find a nice entry and exit point. It is probably a good idea to wait for a consolidation first.
Disclosure: I do not hold any position in any stock mentioned in this article.