Rhythm Pharmaceuticals reports positive data

Rhythm Pharmaceuticals announces preliminary setmelanotide Phase 2 study data

Rhythm Pharmaceuticals announces preliminary setmelanotide Phase 2 study data. See Stockwinners.com for details

Rhythm Pharmaceuticals (RYTM) announced the presentation of preliminary data from an ongoing Phase 2 proof-of-concept study evaluating the safety and efficacy of setmelanotide, the company’s novel melanocortin-4 receptor, or MC4R, agonist, for the treatment of Bardet-Biedl syndrome, or BBS.

BardetBiedl syndrome (BBS) is a ciliopathic human genetic disorder that produces many effects and affects many body systems. It is characterized principally by obesity, retinitis pigmentosa, polydactyly, hypogonadism, and renal failure in some cases.

Results are being presented at the ObesityWeek 2017 meeting held October 29 – November 2, 2017, at the Gaylord National Resort & Convention Center in Washington, D.C.

Rhythm is advancing clinical research programs evaluating setmelanotide as a first-in-class treatment for a number of rare genetic forms of obesity caused by deficiencies in the MC4 pathway, a key biological pathway in humans that regulates weight by increasing energy expenditure and reducing appetite.

Mutations affecting the MC4 pathway are a potential cause of early onset obesity and hyperphagia often associated with BBS, a rare genetic disorder that is also characterized by vision loss, polydactyly, kidney abnormalities, and other symptoms.

BBS is estimated to have a prevalence of approximately one in 100,000 in North America.

The Phase 2 study includes five BBS patients who presented with morbid obesity and hyperphagia at initiation.

Setmelanotide is being administered daily by subcutaneous injection for 52 weeks.

Within 6-19 weeks of initiation, four patients experienced cumulative weight loss of 12.1%, 7.9%, 9.7%, and 9.7% respectively. One patient showed no weight loss; however, achieved apparent weight stabilization.

Hunger scores improved in all patients. Treatment has been well tolerated with adverse effects including mild injection site reactions and increased skin pigmentation.

The preliminary data provide support for continued evaluation of setmelanotide in BBS patients.

Rhythm has also demonstrated proof-of-concept in Phase 2 clinical trials that evaluated setmelanotide for the treatment of two additional MC4 pathway deficiencies: pro-opiomelanocortin, or POMC, deficiency obesity and leptin receptor deficiency obesity.

The FDA granted setmelanotide Breakthrough Therapy Designation for the treatment of POMC deficiency obesity and LepR deficiency obesity, and setmelanotide is currently in Phase 3 development for both conditions.

RYTM closed at $24.42.


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Bioverativ reports positive data from its hemophilia drug

Bioverativ says ELOCTATE shows improvements in joint health in hemophilia

bioverativ reports positive data on its hemophilia drug. See Stockwinners.com for details

Bioverativ (BIVV) and Swedish Orphan Biovitrum announced the publication of interim results from a longitudinal study of joint health in patients treated prophylactically with ELOCTATE marketed as Elocta in Europe and the Middle East, for treatment of hemophilia A.

Hemophilia A, also called factor VIII (FVIII) deficiency or classic hemophilia, is a genetic disorder caused by missing or defective factor VIII, a clotting protein. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a change in a gene.

According to the US Centers for Disease Control and Prevention, hemophilia occurs in approximately 1 in 5,000 live births. There are about 20,000 people with hemophilia in the US. All races and ethnic groups are affected. Hemophilia A is four times as common as hemophilia B while more than half of patients with hemophilia A have the severe form of hemophilia.

Interim data show participants enrolled in the ASPIRE extension study demonstrated continuous improvement in joint health over a nearly three-year period with prophylactic dosing of ELOCTATE, regardless of prior treatment regimen, severity of joint damage or target joints.

Joint health improvements were most notable in hemophilia A patients with poor joint health.

These results were published online October 30 in Haemophilia.

This interim post hoc analysis evaluated joint health in adult and adolescent participants in the A-LONG and ASPIRE studies using a modified version of the Hemophilia Joint Health Score, a first-line assessment tool that grades joints by specific domains, including swelling, muscle atrophy, alignment, range of motion, joint pain, strength and global gait.

The study examined mHJHS measurements taken at A-LONG baseline and ASPIRE baseline and annually thereafter for nearly three years of treatment.

In the study, adults and adolescents treated prophylactically with ELOCTATE experienced a mean improvement in joint health score of -4.1 at ASPIRE Year 2, compared with A-LONG baseline.

Regardless of pre-study treatment regimen, subjects showed continuous improvement in mHJHS from A-LONG baseline through ASPIRE Year 2 and benefits were seen in subjects with target joints as well as those with severe joint destruction.

The mHJHS components with the greatest improvement at ASPIRE Year 2 were swelling, range of motion and strength.

BIVV closed at $55.84. It has a 52-weeks trading range of $41.88 – $64.41.


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UK court rules in favor of Mylan over Teva

Mylan wins UK court ruling related to Copaxone 40 mg/mL patent

UK court votes in favor of Mylan over Teva. See Stockwinners.com for details

Mylan N.V. (MYL) announced that the United Kingdom’s High Court of Justice has issued a decision in favor of Mylan and its European partner Synthon, finding all claims of Teva’s (TEVA) patent EP (UK) 2 949 335 (EP 335) relating to Copaxone 40 mg/mL invalid based on obviousness.

This victory is yet another important milestone for Mylan, and this U.K. court decision only further increases Mylan’s confidence in its ability to bring high quality, lower-cost generic versions of Copaxone to the multiple sclerosis community and patients around the world.

Over the course of the last eight years, Mylan has successfully overcome Teva’s four waves of U.S. patent litigation, eight Citizen Petitions, injunction proceedings in India, more than 15 regulatory challenges, patent litigations or commercial actions across Europe, and now the litigation in the U.K., in addition to obtaining dismissal of Teva’s suit against the FDA seeking to delay approval of the 20 mg/mL product.

Today’s positive ruling in the U.K. will further help pave the way for Mylan’s future launches of Glatiramer Acetate Injection 40 mg/mL in certain European markets.

In addition, Mylan recently learned of Teva’s latest action with the filing of an infringement action against Mylan’s Irish subsidiary Mylan Teoranta in the High Court of Ireland alleging that Mylan’s Glatiramer Acetate 40 mg/mL injection infringes two European patents.

In fact, one of those patents is the same patent that was just invalidated today by the U.K. High Court of Justice and the counterpart to a U.S. patent that was previously held invalid by both the United States District Court for the District of Delaware and the Patent Trial and Appeal Board. Mylan will support the multiple sclerosis patient population through its continued commitment to bring lower-cost generic versions of Copaxone around the world regardless of any further attempts by Teva to deny such access.

MYL closed at $39.02. TEVA closed at $13.94.


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Atossa Genetics breast cancer drug shows promise

All objectives met in Phase 1 study of oral Endoxifen

Atossa Genetics breast cancer drug shows promise. See Stockwinners.com for details

Atossa Genetics (ATOS) reported preliminary results from its Phase 1 study of its proprietary oral Endoxifen.

All objectives were successfully met.

Safety: There were no clinically significant safety signals and no clinically significant adverse events in participants receiving oral Endoxifen.

Tolerability: Oral Endoxifen was well tolerated at each dose level and for the dosing duration utilized in the study.

Pharmacokinetics: Oral Endoxifen demonstrated blood levels that have been associated with a therapeutic effect in the adjuvant setting in women with breast cancer. These data demonstrate the suitability of oral Endoxifen for further clinical development.

“Based on these positive preliminary results, we are advancing our oral Endoxifen into Phase 2 studies,” commented Dr. Steven C. Quay, CEO and President.

“We expect our initial Phase 2 study will be in women who are refractory to tamoxifen and we expect to begin that study in the first quarter of 2018,” continued Dr. Quay.

BACKGROUND

Researchers at the Mayo Clinic Cancer Center are developing a new drug to help women with estrogen receptor positive breast cancer who are unable to derive benefit from the drug tamoxifen. These women need an alternative to #tamoxifen because their bodies are unable to effectively metabolize the drug to its active agent, endoxifen.

Tamoxifen is a hormone therapy that has been used for more than 40 years to reduce the risk of breast cancer and to prevent recurrence. However, research has demonstrated that patients with very low levels of a critical enzyme called CYP2D6 and those with low endoxifen levels have a higher risk of recurrence or progression when treated with tamoxifen.

Endoxifen, also known as N-desmethyl-4-hydroxytamoxifen, is an orally active nonsteroidal selective estrogen receptor modulator (SERM) of the triphenylethylene group that is or was under development for the treatment of estrogen receptor-positive breast cancer. It is also being evaluated as an antipsychotic for treatment of mania and other psychotic disorders.

ATOS haled at $1.22.


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