Week Ahead In Pharmaceuticals - 7 Stocks To Watch


(RTTNews.com) - The clinical trial front as well as the regulatory front witnessed mostly positive news this week.

KemPharm Inc. (KMPH) reported positive data from its pivotal efficacy and safety clinical trial of KP415 in attention-deficit/hyperactivity disorder on July 9. However, KMPH didn't get a lift from the positive data, and the stock is down 23% since the announcement of the trial data.

On July 10, Celgene Corp. ( CELG ) and Acceleron Pharma Inc. (XLRN) announced that their phase III clinical study Luspatercept in adults with transfusion-dependent beta-Thalassemia, dubbed BELIEVE, achieved primary and all key secondary endpoints.

Shares of SCYNEXIS Inc. (SCYX) rose 21% on July 11, following positive results from a phase 2b, dose-finding study of SCY-078 for the treatment of vulvovaginal candidiasis, dubbed DOVE study.

Zogenix Inc. (ZGNX) reported positive top-line results from its second confirmatory phase III study of its investigational drug, ZX008 for the treatment of children and young adults with Dravet syndrome on July 12. The stock is up over 32% since the announcement of the results.

On the regulatory front, the FDA approved Bristol-Myers Squibb Co.'s ( BMY ) Opdivo in combination with Yervoy for yet another indication on July 11, and an FDA panel voted to recommend approval of GlaxoSmithKline PLC's (GSK) single-dose Tafenoquine 150mg for the radical cure (prevention of relapse) of malaria on July 12.

SIGA Technologies Inc.'s (SIGA) oral TPOXX, a small molecule antiviral treatment for smallpox, received FDA approval today (July 13), nearly one month ahead of the decision date of August 8.

Now, here are some of the pharma/biotech stocks and upcoming events to keep an ear out for in the coming week.

<b>1. Catalyst Biosciences Inc. ( CBIO )</b>

Catalyst Biosciences is focused on developing therapies for hemophilia.

The Company's lead drug candidate is Marzeptacog alfa (activated) a potent, subcutaneously administered, next-generation Factor VIIa therapy being developed for prophylaxis in hemophilia A or B with inhibitors.

Hemophilia is a rare but serious bleeding disorder. There are two main types - Hemophilia A, caused due to deficiency or dysfunction of clotting protein factor VIII, and Hemophilia B, caused due to deficiency or dysfunction of clotting protein factor IX.

The current standard of care requires recurrent intravenous infusions of either plasma-derived or recombinant factor VIII or IX to control and prevent bleeding episodes.

But some people with hemophilia will develop inhibitors, making it difficult to treat. When a person develops an inhibitor, the body stops accepting the factor treatment product as a normal part of blood, thinking that the factor is a foreign substance and tries to destroy it with an inhibitor.

Watch out for...

The interim data from phase II part of the Company's phase II/III trial of Marzeptacog alfa (activated) in individuals with hemophilia A or B with inhibitors is scheduled to be announced on July 18, 2018.

CBIO closed Friday's trading at $12.44, up 2.64%.

<b>2. Ultragenyx Pharmaceutical Inc. (RARE)</b>

Ultragenyx is a biopharmaceutical company developing novel products for the treatment of rare and ultra-rare diseases.

The Company has two approved drugs, Burosumab, known under the trade name Crysvita, for the treatment of X-Linked Hypophosphatemia, and Mepsevii, for the treatment of Mucopolysaccharidosis 7 (MPS 7).

Watch out for...

Burosumab's potential is also being explored in the indication of Tumor-Induced Osteomalacia (TIO), and 48-week data from a phase II trial in TIO, due mid-2018, is awaited.

RARE closed Friday's trading at $84.56, up 0.58%.

<b>3. Ra Pharmaceuticals Inc. (RARX)</b>

Ra Pharma is a clinical-stage biopharmaceutical company focused on developing drugs for the treatment of serious diseases that are caused by excessive or uncontrolled activation of the complement system, a critical component of the immune system.

The Company's lead drug candidate is RA101495 that is being developed for convenient self-administered subcutaneous injection for the treatment of paroxysmal nocturnal hemoglobinuria, Myasthenia Gravis, and renal indications such as atypical hemolytic uremic disorder (aHUS) and lupus nephritis.

Watch out for...

Data from a phase 1b clinical trial of RA101495 SC in atypical hemolytic uremic disorder, due mid-2018, is awaited.

RARX closed Friday's trading at $11.18, down 7.53%.

<b>4. Pulmatrix Inc. (PULM)</b>

Pulmatrix is a clinical stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary disease using its patented iSPERSE technology.

A single dose escalation phase 1/1b study of Pulmazole (PUR1900), an inhaled iSPERSE formulation of the anti-fungal drug Itraconazole, for the treatment of allergic bronchopulmonary aspergillosis (ABPA) in patients with asthma is underway.

The Company announced positive top-line results from Parts 1 and 2 of the 3-Part phase 1/1b study of Pulmazole last month.

Watch out for...

Analysis of the preliminary data of Part 3 of the phase 1/1b study of Pulmazole is underway and expected to be complete in mid-July 2018.

PULM closed Friday's trading at $0.54, down 1.07%.

<b>5. Denali Therapeutics Inc. ( DNLI )</b>

Denali is a biopharmaceutical company developing a broad portfolio of therapeutic candidates for neurodegenerative diseases.

The Company's most advanced core programs include LRRK2 inhibitor program to address Parkinson's disease and RIPK1 inhibitor program to address Alzheimer's disease and Amyotrophic lateral sclerosis (ALS).

The two most advanced product candidates in the LRRK2 program are DNL201 and DNL151.

DNL201 is currently in a Phase 1 clinical trial in healthy volunteers in the United States, and DNL151 is currently in a Phase 1 clinical trial in healthy volunteers in the Netherlands.

The most advanced product candidate in the RIPK1 inhibitor program is DNL747, which is currently in a Phase 1 clinical trial in healthy volunteers in the Netherlands.

Watch out for...

Top line data from the phase I trial of DNL201 in healthy volunteers, due in the first half of 2018, is awaited.

DNLI closed Friday's trading at $14.49, down 1.83%. In after-hours, the stock was up 1.18% at $14.66.

<b>6. Spring Bank Pharmaceuticals Inc. (SBPH)</b>

Spring Bank Pharma is a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of viral infections, inflammatory diseases and certain cancers.

The Company's lead product candidate is Inarigivir, also known as SB 9200, under Phase 2a trial, for the treatment of HBV, dubbed ACHIEVE.

The phase 2a trial has 4 monotherapy dosing regimens of SB 9200 namely, 25mg, 50mg, 100mg and 200mg followed by Viread for the treatment of chronic hepatitis B virus (HBV).

The Company has already reported results from the 24-week trial of both the 25mg and 50mg cohorts of Part A of the ongoing phase II ACHIEVE trial.

Watch out for...

Top-line results from the third monotherapy cohort (100mg) of the ACHIEVE trial due mid-2018, are awaited.

SBPH closed Friday's trading at $12.57, up 3.88%.

<b>7. Altimmune Inc. ( ALT )</b>

Altimmune is a clinical-stage immunotherapeutics company having three clinical product candidates in its pipeline namely, NasoVAX, a phase II seasonal influenza vaccine candidate, HepTcell, a phase I immunotherapeutic candidate for the potential cure of chronic hepatitis B, SPARVAX-L, a phase II Anthrax vaccine, and NasoShield, a next generation Anthrax vaccine, under phase I study.

Watch out for...

Safety and immunogenicity data from phase I study of single dose NasoShield anthrax vaccine are expected in early 3Q, 2018.

ALT closed Friday's trading at $0.50, up 12.27%.


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