BPC January 09 update

ARIAD (ARIA) acquisition - shares soar 73%; Egalet (EGLT) FDA Approval; EnteroMedics (ETRM) 3-day gain 740%; J.P. Morgan Pipeline updates

Price and Volume Movers

The J.P. Morgan conference commenced today giving companies an opportunity to update their pipelines and provide news of license deals and acquisitions. Coupled with the fact that the Biotech Showcase for smaller companies is also running concurrently, this provides a mini earnings season news flow, yet compressed into only 3-4 days.

Although over 50 companies on BioPharmCatalyst have had their pipelines updated today with a further 15-20 to be added tonight, only those with significant changes with their trial timelines will be noted below. Any important additions made tonight will be added to tomorrow’s update.

Now to Monday’s price movers.

It was mentioned in last Friday’s newsletter that EnteroMedics Inc (NASDAQ:ETRM) shares had soared 340% over two days on volume over approximately 15x its share float. The energy continued today, with shares surging another 93% on similar volume to $17.70 for a three-day cumulative rise of over 740% with day traders, rather than meaningful news, controlling the momentum behind the price move.

ARIAD Pharmaceuticals (NASDAQ:ARIA) was the major newsmaker of the day when it announced that it will be acquired by by Takeda Pharmaceutical Company (Japan) for $24 per share under which Takeda will acquire all of the outstanding shares for approximately $5.2 billion. The transaction is expected to close by the end of February 2017. Shares of Ariad closed up 73% to $23.75 on 35x average volume.

Mast Therapeutics, Inc. (NYSE MKT: MSTX) announced over the weekend that it will create a reverse merger with Savara Inc with shareholders of Savara owning 76% of the newly formed company expected to be named Savara Inc. Shares spiked up in early trading before retracing some of its gains to close up 52% to $0.15 on 12x average volume.

AMAG Pharmaceuticals, Inc (NASDAQ:AMAG) shares plummeted to close down over 35% to $23.00 on 16x average volume. The company released a number of updates today which disappointed investors. It agreed to make an upfront payment of $60m to Palatin Technologies, Inc. for (NYSEMKT:PTN) for rights to Rekynda(bremelanotide) for treatment of hypoactive sexual desire disorder (HSDD). However, the major catalyst was a downgrade by Raymond James from Market Perform to Underperform. Makena, the key revenue earner for the company, only has orphan drug status until February 2018 and today it noted that it has discontinued a recently initiated comparative pain study and it will not plan to request orphan exclusivity as part of an upcoming Supplemental New Drug Application for Makena.

Incyte Corporation (NASDAQ:INCY) shares closed up 9% to $118.53 on 2.5x average volume. The company announced that it will be advancing its development program of its IDO1 inhibitor epacadostat, with KEYTRUDA, an anti-PD-1 therapy run by Merck (NYSE:MRK). Four additional Phase 3 trials are planned. Shares of NewLink Genetics Corp (NASDAQ:NLNK) closed up 18% to $13.12 on 4x average volume on the back of the news. NewLink has its own IDO1 inhibitor , NLG919, and has currently a license agreement with Genentech.

Egalet Corporation (Nasdaq: EGLT) announced during the trading day that the FDA has approved ARYMO ER (morphine sulfate) extended-release (ER) tablets C-II for the management of severe pain. Shares initially spiked up over 20% before retracing gains once it was noted that there would not be an oral abuse deterrence on the label. Shares closed up 6% on 10x average volume but its gains have all but evaporated in the after-hours session, currently trading at $7.91.

Exelixis, Inc. (Nasdaq:EXEL) shares closed up 15% to $17.58 on 2x average volume. The company announced today that Genentech, Inc. has withdrawn its counterclaim against Exelixis regarding marketing expenses for COTELLIC (cobimetinib) plus Zelboraf. Exelixis is relieved of $18.7m of disputed costs previously charged by Genentech and Genentech will return $7.1m that Exelixis paid previously.

ProNAi Therapeutics, Inc. (NASDAQ: DNAI) announced it has changed its corporate name to Sierra Oncology, Inc. and that its shares will trade on the NASDAQ under the symbol 'SRRA', effective on January 10.

A selection of pipeline updates below:

Pipeline Database Updates

Drug Stage Catalyst

Parkinson’s disease (PD)

Phase 3 Noted November 15, 2017 seven cases of sepsis, five of which were fatal. However, company noted November 20, 2017 that program will be discontinued.

Axalimogene filolisbac
Anal cancer (FAWCETT)

Phase 2 Noted in 10-K December 21, 2017 that it will not initiate the Stage 2 portion of the trial in order to focus its resources on other clinical priorities.

IDHIFA (enasidenib) - AG-221
Advanced hematologic malignancies with an IDH2 mutation

Approved Approval announced August 1, 2017.

IDH1m Relapsed/Refractory AML - cancer

PDUFA priority review PDUFA date under priority review August 21, 2018.

Respiratory syncytial virus (RSV)

Phase 2a Phase 2a data released February 1, 2017 - primary endpoint not met.

Spinal muscular atrophy (SMA) Type 1

Phase 3 Phase 3 trial has been initiated - noted November 9, 2017.

Intepirdine - MINDSET
Alzheimer's disease

Phase 3 Phase 3 data released September 26, 2017 - primary endpoints not met.

Namodenoson (CF102)
Non-alcoholic steatohepatitis (NASH)

Phase 2 Phase 2 enrollment has commenced - noted November 27, 2017.


Phase 2 Phase 2 trial discontinued - noted January 9, 2017.

PIXUVRI - PIX306 Trial
B-cell non-Hodgkin lymphoma

Phase 3 Phase 3 data due 1H 2018.

Abuse-Deterrent Morphine

Approved Approved January 9, 2017. Tentative approval for label expansion announced December 15, 2017. Final approval expected October 2, 2018.

Moderate to severe chronic pain

Phase 3 Phase 3 trial met primary endpoint - November 28, 2017.

Bempedoic acid - CLEAR LDL-C Lowering Program 1002-046, 1002-047 and 1002-048
Hypercholesterolemia - statin intolerance

Phase 3 Phase 3 initiation of efficacy trials announced January 8, 2017. Data due March, May and September 2018.

Pompe Disease

Phase 1/2 Phase 1/2 additional data released February 15, 2017, May 15, 2017 and October 4, 2017. Further update February 7, 2018 showed 6MWT distance of 25 meters at 9 months and 57 meters at 12 months.

Epacadostat with Keytruda
Non-small cell lung cancer (NSCLC)

Phase 3 Phase 3 planned.

Epacadostat with Keytruda
Bladder cancer

Phase 3 Phase 3 planned.

Epacadostat with Keytruda
Squamous cell carcinoma of the head and neck cancer

Phase 3 Phase 3 planned.

Cancer - HER2-negative gastric cancer

Phase 3 Acquired by Ipsen

Early onset facioscapulohumeral muscular dystrophy (FSHD)

Phase 1/2 Phase 1/2 final data presented October 5, 2017 at the International Annual Congress of the World Muscle Society (WMS).

LPCN 1107
Prevention of preterm birth ("PTB").

Phase 3 Conduct food/fat effect study 2H 2018 in preparation for Phase 3 trial.

Rekynda (Bremelanotide)
Female sexual dysfunction (FSD)

Phase 3 Phase 3 data released November 1, 2016. NDA filing due 1Q 2018.

Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)

CRL Received a Refusal to File letter for their NDA Filing - February 23 2016. Appeal rejected October 17, 2016. Decision made to resubmit NDA during 1Q 2017 under protest. New PDUFA date October 24, 2017. Advisory Committee Meeting September 28, 2017 voted that although it is possible that ataluren may be effective, the data are inconclusive, and more work would be needed to establish whether ataluren is effective. FDA issued CRL October 25, 2017.

Pantothenate kinase-associated neurodegeneration (PKAN)

Phase 3 Phase 3 dosing has commenced - noted July 25, 2017. Enrollment to be completed 2H 2018.

Closed Triple - CAPTAIN

Phase 3 Phase 3 data due mid-2019.

Recurrent platinum resistant ovarian cancer

Phase 3 Phase 3 trial initiation December 27, 2017.