PDA

View Full Version : $ALXN - Alexion Pharmaceuticals



hardt
29-05-2017, 02:01 AM
Alexion, the maker of the world's most expensive drug *Soliris* (Eculizumab) at $NZD 700,000 a year - "treatment of paroxysmal nocturnal hemoglobinuria (PNH), a genetic blood disorder; and atypical hemolytic uremic syndrome (aHUS), an extremely rare genetic disease"

This company is also known for employing PR firms to aide [ without disclosure of the relationship to Alexion ] the patients themselves which believe the company to be extortionist to pressure governments into subsidizing Soliris, which is required for a lifetime...or until a cure is found ( due to the low reward profile of a cure, is highly unlikely to occur )
The recurring treatment model is definitely one worth looking at.

After many years of research, development and millions of dollars this company has created an amazing drug that prevents the premature death and suffering of thousands of people worldwide... for one hell of a price.

Great upside potential here if you have a strong stomach for the short term, got my finger on the trigger...

Orders from 96.7 through to 97 this morning alongside some loading up of USD as well.

hardt
15-06-2017, 03:42 AM
Already up 20% to 117, loving how fast biopharma moves... Until of course it works against me, so far so good.

hardt
01-07-2017, 07:40 AM
Bounced off 126 early this week, topping up at 121 this morning... More legs up after consolidation and EOFY selldown in my opinion.

Now averaging 100 and 14% of my portfolio.

hardt
24-07-2017, 07:31 AM
Earnings inbound on Friday NZT, seems to be a fair bit of optimism out there as ALXN outperforms frequently.

"While not issued just yet, recent activity from the U.S. Patent and Trademark Office (USPTO) would suggest they will issue in the next few months. Of note, these patent applications - one composition of matter and one method of treatment - appear to provide additional protection for Soliris into 2027, well beyond the 2021 expiry of the U.S. '245 composition of matter patent. This comes on the heels of last month's notice of allowance for Alexion's '839 patent application, bringing further clarity to Alexion's IP extension strategy that could stretch Soliris' exclusivity beyond 2021, which seems to bode well for the Soliris/ALXN1210 franchise."

"Robust Pipeline: Alexion’s efforts to develop its pipeline are impressive. The company is currently evaluating ALXN1210 (a longeracting
anti-C5 antibody that inhibits terminal complement) in phase III studies for both PNH and aHUS. Additionally, the company is
conducting a phase I study on a new formulation of ALXN1210 when administered subcutaneously in healthy volunteers. Patients
are currently being dosed in a phase III trial comparing ALXN1210 administered intravenously every eight weeks to Soliris in
complement inhibitor treatment-naive patients with PNH. Alexion plans to initiate a phase III PNH switch study of ALXN1210
administered intravenously every eight weeks compared to patients currently treated with Soliris in the second quarter of 2017.
Enrollment is expected to be completed in both studies in 2017. Meanwhile, Alexion has completed enrollment in two ongoing
studies on ALXN1210 in patients with PNH – a phase I/II dose-escalating study and an open-label, multi-dose phase II study on
longer dosing intervals beyond 8 weeks. Patients are also being dosed in a phase III trial with ALXN1210 administered intravenously
every eight weeks in complement inhibitor treatment-naive adolescent and adult patients with aHUS. Enrollment is expected to be
complete in 2017. Alexion expects to initiate another phase III trial of ALXN1210 in pediatric patients with aHUS in the second
quarter of 2017(approval target in late 2018or 2019). The company is evaluating ALXN1007 (a complement inhibitor that targets
C5a), in a phase II study in patients with graft-versus-host disease, involving the lower gastrointestinal tract.
Meanwhile, a phase I/II study evaluating SBC-103 (a recombinant form of the NAGLU enzyme) for the treatment of patients with
mucopolysaccharidosis IIIB, is currently underway. Additional data from the study will be presented in 2017. Alexion is also enrolling
patients in a pivotal phase II/III study on ALXN1101 for treatment-na´ve neonates with molybdenum cofactor deficiency type A.
ALXN1007, ALXN1210 and SBC-103 enjoy Orphan Drug status in both the U.S. and the EU for their respective indications. Alexion
expects as many as six additional products or indication approvals through 2018 and thus the approval of additional candidates will
be a big boost for it."

9021

hardt
29-07-2017, 08:21 AM
http://ir.alexionpharm.com/secfiling.cfm?filingID=899866-17-153&CIK=899866

Another smashing outperformance, so much upside remains here... moved upwards of 38% for me in 2 months.

Large pipeline with billions of potential revenue ahead, stock is moving towards $200 with some good news out of FDA.


Clinical Development Programs Our clinical development programs include the following:


Refractory Generalized Myasthenia Gravis (gMG)
Refractory gMG is an ultra-rare segment of Myasthenia Gravis, a debilitating, complement-mediated neuromuscular disease in which patients suffer profound muscle weakness throughout the body, resulting in slurred speech, impaired swallowing and choking, double vision, upper and lower extremity weakness, disabling fatigue, shortness of breath due to respiratory muscle weakness and episodes of respiratory failure. The FDA, EC and MHLW have granted orphan drug designation for eculizumab as a treatment for patients with refractory gMG.
In June 2016, we announced topline results of the Phase III REGAIN trial of eculizumab for the treatment of refractory gMG. The primary efficacy endpoint of change from baseline in Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score, a patient-reported assessment, at week 26, did not reach statistical significance (p=0.0698) as measured by a worst-rank analysis. The totality of data reviewed to date, including the first three secondary endpoints and a series of prospectively defined sensitivity analyses, shows early and sustained substantial improvements over 26 weeks for patients treated with eculizumab compared to placebo. The safety of eculizumab in this study was consistent with the Soliris labels. Additional data from the Phase III study was presented in July 2016. The data showed that 18 of 22 pre-defined endpoints and pre-specified analyses in the study, based on the primary and five secondary endpoints, achieved p-values below 0.05.
In January 2017, we announced that we filed for regulatory approval for eculizumab in refractory gMG in both the U.S. and Europe. These marketing applications were based on the comprehensive data from the Phase III REGAIN trial. In January, the European submission was validated by the European Medicines Agency (EMA), marking the beginning of the review process in Europe for this potential new indication for Soliris. In March 2017, the FDA accepted for review the Company's supplemental Biologics License Application to extend the indication for Soliris as a potential treatment for patients with gMG. Additionally, in March 2017, we submitted an application to the MHLW to extend the indication for Soliris as a potential treatment for patients with gMG.
In June 2017, we announced that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has adopted a positive opinion to extend the current therapeutic indication for Soliris (eculizumab) to include the treatment of refractory gMG in patients who are anti-acetylcholine receptor (AChR) antibody-positive.


Relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD)
Relapsing NMOSD is a severe and ultra-rare autoimmune disease of the central nervous system (CNS) that primarily affects the optic nerves and spinal cord. The disease leads to severe weakness, paralysis, respiratory failure, loss of bowel and bladder function, blindness and premature death. Enrollment and dosing are ongoing in a global, randomized, double-blind, placebo-controlled trial to evaluate eculizumab as a treatment for patients with relapsing NMOSD. The FDA, EC, and MHLW have each granted orphan designation for eculizumab as a treatment for patients with relapsing NMOSD.
Transplant
Antibody Mediated Rejection (AMR) in Presensitized Kidney Transplant Patients

AMR is the term used to describe a type of transplant rejection that occurs when the recipient has antibodies to the donor organ. Enrollment in a multi-national, multi-center controlled clinical trial of eculizumab in presensitized kidney transplant patients at elevated risk for AMR who received kidneys from deceased organ donors was completed in March 2013 and patient follow-up in the trial is continuing. In September 2013, researchers presented positive preliminary data from the eculizumab deceased-donor AMR kidney transplant study. In May 2015, new data from the Phase II single-arm deceased-donor transplant trial of eculizumab in prevention of acute AMR was presented and was consistent with previous positive reports.


ALXN1210
ALXN1210 is a highly innovative, longer-acting anti-C5 antibody discovered and developed by Alexion that inhibits terminal complement. In early studies, ALXN1210 demonstrated rapid, complete, and sustained reduction of free C5 levels. Alexion has completed enrollment in two ongoing clinical studies of ALXN1210 in patients with PNH-a Phase I/II dose-escalating study and an open-label, multi-dose Phase II study that is also evaluating longer dosing intervals beyond eight weeks.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
In June 2016, we announced interim data from a Phase I/II study in patients with PNH showing that once-monthly dosing of ALXN1210 achieved rapid and sustained reductions in hemolysis, as measured by mean levels of lactate dehydrogenase (LDH), in 100 percent of treated patients. Chronic hemolysis in patients with PNH may be associated with life-threatening thromboses, recurrent pain, kidney disease, disabling fatigue, impaired quality of life, severe anemia, pulmonary hypertension, shortness of breath and intermittent episodes of dark-colored urine (hemoglobinuria). Researchers also reported that, at the time of analysis, 80 percent of patients who required at least 1 blood transfusion in the 12 months prior to treatment with ALXN1210 did not require transfusions while on treatment with ALXN1210. Furthermore, in December 2016, we reported new data from this same ongoing study that showed rapid and sustained reductions in LDH in patients with PNH treated with once-monthly dosing. Patients also had improvements in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue score from baseline, with patients in the higher-dose cohort achieving a two-fold greater improvement compared with the lower-dose cohort. In addition, we have completed enrollment and treatment is ongoing in an open-label, multi-dose Phase II study of ALXN1210 in patients with PNH designed to measure reductions in hemolysis and safety in several dosing cohorts and intervals evaluating monthly and longer dosing intervals. We have initiated a Phase III open-label, multinational, active-controlled study of ALXN1210 compared to eculizumab (Soliris) in adult patients with PNH who have never been treated with a complement inhibitor. The study is evaluating ALXN1210 administered intravenously every eight weeks. Patient enrollment has been completed in this trial.
In addition, we have initiated a supportive Phase III open-label, multinational, active-controlled study of ALXN1210 in patients with PNH who had been receiving eculizumab compared to eculizumab in adult patients with PNH who have never been treated with a complement inhibitor. The study is evaluating ALXN1210 administered intravenously every eight weeks. Patient enrollment is ongoing in this trial.
In June 2016 and January 2017, the EC and the FDA, respectively, granted orphan drug designation to ALXN1210, for the treatment of patients with PNH.
Atypical Hemolytic Uremic Syndrome (aHUS)
We initiated a Phase III open-label, single arm, multicenter study of ALXN1210 in adolescent and adult patients with aHUS who have never been treated with a complement inhibitor. In patients with aHUS, complement-mediated TMA leads to life-threatening damage to the kidney, brain, heart and other vital organs. The study will evaluate ALXN1210 administered intravenously every eight weeks. Patient enrollment is ongoing in this trial.
In addition, we have initiated a supportive Phase III open-label, multinational, study of ALXN1210 in pediatric and adolescent patients with aHUS who have never been treated with a complement inhibitor. The study is evaluating ALXN1210 administered intravenously every eight weeks. Patient enrollment is ongoing in this trial.


Subcutaneous (SC) Delivery
We have completed enrollment in a Phase I study in healthy volunteers to evaluate ALXN1210 delivered subcutaneously.


cPMP (ALXN1101)
Molybdenum Cofactor Deficiency (MoCD) Disease Type A (MoCD Type A)
MoCD Type A is an ultra-rare metabolic disorder characterized by severe and rapidly progressive neurologic damage and death in newborns. MoCD Type A results from a genetic deficiency in cyclic Pyranopterin Monophosphate (cPMP), a molecule that enables the function of certain enzymes and the absence of which allows neurotoxic sulfite to accumulate in the brain. To date, there is no approved therapy available for MoCD Type A. There has been some early clinical experience with the recombinant cPMP replacement therapy in a small number of children with MoCD Type A, and we have completed enrollment in a natural history study in patients with MoCD Type A. cPMP has received Breakthrough Therapy Designation from the FDA for the treatment of patients with MoCD Type A. Evaluation of our synthetic form of cPMP replacement therapy in a Phase I healthy volunteer study is complete. In addition, we completed enrollment in a multi-center, multinational open-label clinical trial of synthetic cPMP in patients with MoCD Type A switched from treatment with recombinant cPMP. These trials will not be expanded and no new patients will be added to the trials. Patients currently enrolled in the trials will continue to receive therapy. No additional studies are planned.
Samalizumab (ALXN6000)
Samalizumab is a first-in-class immunomodulatory humanized monoclonal antibody that blocks CD200 a key immune checkpoint protein expressed in both hematologic and solid malignancies. The safety and efficacy of samalizumab are being evaluated in patients with advanced solid tumors.
The Leukemia and Lymphoma Society is conducting a multi-arm acute myeloid leukemia (AML) study as referred to as the BEAT AML Master Trial, evaluating samalizumab as well as other potential therapies for the treatment of AML.
These trials will not be expanded and no new patients will be added to the trials. Patients currently enrolled in the trials will continue to receive therapy. No additional studies are planned.






Updated GAAP Guidance

Prior GAAP Guidance

Updated Non-GAAP Guidance

Prior Non-GAAP Guidance



Total revenues

$3,450 to $3,525 m

$3,400 to $3,500 m

$3,450 to $3,525 m

$3,400 to $3,500 m



Soliris revenues

$3,075 to $3,125 m

$3,025 to $3,100 m

$3,075 to $3,125 m

$3,025 to $3,100 m



Metabolic revenues

$375 to $400 m

$375 to $400 m

$375 to $400 m

$375 to $400 m



R&D (% total rev)

23% to 25%

24% to 26%

21% to 22%

22% to 23%



SG&A (% total rev)

29% to 30%

28% to 30%

25% to 26%

25% to 26%



Operating margin

23% to 26%

25% to 28%

43% to 44%

43% to 44%



Earnings per share

$2.82 to $3.12

$2.80 to $3.20

$5.40 to $5.55

$5.10 to $5.30

hardt
08-10-2017, 09:31 AM
Skimmed the lot I bought at 121... too much of my portfolio on little movement.




POS 1 ALXN

POS 2 ALXN








ENTRY

97.5

121



FX NZD

0.74

0.75



EXIT

142*

142



CAPITAL GAIN

45.64%*

17.36%



FX NZD

0.705

0.71



CURRENCY GAIN

4.96%

5.63%



TOTAL GAIN

50.61%*

22.99%