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APOP Turns Heads, CCXI's Kitty Gets $100 Mln Boost, BOLD Breathes Easy

Jan4 up 010418 lt

The following are some of today's top gainers in the pharma/biotech sector.

1. ChemoCentryx Inc. (CCXI)

Gained 39.42% to close Thursday's (Jan.4) trading at $8.63.

News: The Company has secured up to $100 Million in new capital commitments.

The European Medicines Agency's validation of the Company's conditional marketing authorization application for Avacopan for the treatment of patients with anti-neutrophil cytoplasmic auto-antibody-associated vasculitis (ANCA vasculitis) has triggered $50 million milestone payment from Vifor Fresenius Medical Care Renal Pharma (VFMCRP), a company of the Vifor Pharma Group and Fresenius Medical Care.

ChemoCentryx, which is responsible for the discovery and development of Avacopan, owns and retains the commercial rights to the drug in the United States and China, and VFMCRP has licensed the rights to commercialize the drug in all other countries.

In addition to the $50 million milestone from VFMCRP, the Company also entered into a $50 million growth capital financing agreement with Hercules Capital, Inc. (HTGC).

The funding will be utilized to advance Avacopan, which is currently under a phase III trial, dubbed ADVOCATE, for the treatment of ANCA-associated vasculitis. The Company expects to complete enrollment in the trial in mid-2018.

2. Cellect Biotechnology Ltd. (APOP)

Gained 37.97% to close Thursday's trading at $9.81.

News: In the Company's Phase I/II clinical trial of ApoGraft, after one month follow-up, all three patients in the first cohort demonstrated complete acceptance of the stem cell transplant with no adverse events related to the study treatment.

ApoGraft is a breakthrough technology for the isolation of stem cells from any given tissue. Cellect's ApoGraft technology is aiming to turn stem cell transplants into a simple, safe and cost effective process, reducing the associated severe side effects, such as rejection and many other risks, such as Graft versus Host Disease.

Graft versus Host Disease, a condition in which the transplanted immune cells attack the recipient's cells, is a common complication associated with *allogeneic stem cell transplantation. (*Allogeneic stem cell transplantation involves transferring the stem cells to the recipient from a genetically matched relative or other donor).

The Phase I/II clinical study is designed to enroll 12 patients, and the Company plans on recruiting a further three patients for the second cohort of patients following review of the independent data and safety monitoring board.

3. Arcturus Therapeutics Ltd. (ARCT)

Gained 19.5% to close Thursday's trading at $9.32.

News: The Company is teaming up with privately-held German firm CureVac AG to develop up to four molecular therapy products for rare diseases using Curevac's optimized natural mRNA sequence (RNAoptimizer) and Arcturus' lipid-mediated nucleic acid delivery system (LUNAR).

4. Audentes Therapeutics Inc. (BOLD)

Gained 14.86% to close Thursday's trading at $40.81.

News: The Company announced positive interim data from the first dose cohort of its Phase 1/2 clinical trial of AT132 in patients with X-Linked Myotubular Myopathy, dubbed ASPIRO.

X-Linked Myotubular Myopathy is a rare monogenic disease characterized by extreme muscle weakness, respiratory failure and early death, with an estimated 50% mortality rate by 18 months of age.

There was a significant improvement in neuromuscular function and respiratory function in the patients treated with AT132 in the study, according to the Company.

Near-term Catalyst:

-- Next update on interim data from the ASPIRO trial is expected to be available in the second quarter of 2018.

5. InflaRx N.V. (IFRX)

Gained 13.55% to close Thursday's trading at $22.99.

News: The Company will make a presentation at the J.P. Morgan Annual Healthcare Conference on January 10, 2018, and at the LEERINK Partners 7th Annual Global Healthcare Conference on February 14, 2018.

Recent event:

-- The Company went public on the NASDAQ Global Select Market on November 8, 2017, offering its shares at a price of $15.00 each.


The Company's lead compound is IFX-1, under phase II clinical trial in patients suffering from moderate to severe Hidradenitis Suppurativa (HS), a painful, chronic and debilitating inflammatory skin disease. Positive top line data from an exploratory Phase IIa trial of IFX-1 in Hidradenitis Suppurativa were reported in September of 2017.

Near-term catalyst:

-- Initiation of an international, multi-center, randomized, placebo-controlled Phase IIb trial in Hidradenitis Suppurativa is being planned.

6. Intra-Cellular Therapies Inc. (ITCI)

Gained 12.09% to close Thursday's trading at $17.34.

News: No News

Upcoming event:

The Company is scheduled to make a presentation at the 36th Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 9, 2018 at 2:00 pm PST.


The Company's lead drug candidate is Lumateperone (also known as ITI-007) for the treatment of schizophrenia, bipolar disorder, behavioral disturbances in patients with dementia, including Alzheimer's disease, depression and other neuropsychiatric and neurological disorders.

Clinical Trials & Near-term Catalysts:

-- Submit NDA for Lumateperone for the treatment of schizophrenia by mid-2018.
-- Phase III trials with Lumateperone for bipolar depression are ongoing. The bipolar depression consists of three, phase III 6-week, clinical trials. Patient enrollment in the bipolar studies is expected to be completed in 2018.
-- A phase III trial for the treatment of agitation in patients with dementia, including Alzheimer's disease is underway.
-- A Phase 1/2 clinical trial of ITI-214 in patients with Parkinson's disease is ongoing. Patient enrollment in this study is expected to be completed by mid-2018.

7. Achaogen Inc. (AKAO)

Gained 11.04% to close Thursday's trading at $11.77.

News: No news

Recent event:

-- On January 2, 2018, the Company reported positive top-line results from its Phase 1 clinical study of C-Scape, an antibacterial candidate. C-Scape is being developed for MDR gram-negative infections.

Upcoming event:

-- The Company is scheduled to make a presentation at the 36th Annual J.P. Morgan Healthcare Conference on January 11, 2018 at 7:30 a.m. Pacific Time.

Near-term Catalyst:

-- The Company's New Drug Application for Plazomicin for the treatment of complicated urinary tract infections (cUTI), and bloodstream infections is under priority review by the FDA, with a decision expected to be announced by June 25, 2018.

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