Ready to launch
A record 59 NME approvals in 2018 means investors will focus on launches in 2019
The proposed $74 billion takeout of Celgene Corp. by Bristol-Myers Squibb Co. will not shift buysider attention away from upcoming and maturing drug launches heading into 2019, with plenty to track following a record year for NME approvals by FDA.
Several investors expect “shorting the launch” to be an ongoing theme, particularly among small and mid-cap biotechs looking to launch products independently.
Some portfolio managers are turning their attention to companies with more mature launches that have gained momentum. “We try and look at names that aren’t necessarily launching imminently but have been on the market and they seem to be picking up sales and ticking up,” International Biotechnology Trust plc’s Ailsa Craig told BioCentury.
“Once you get to the commercial stage you sell.”
Buysiders will also be monitoring reimbursement trends among new modalities, including gene and cell therapies and RNAi, which have seen mixed commercial performance over the last six months (see “Eyes on Launches”).
As for clinical catalysts, a flurry of readouts from late-stage non-alcoholic steatohepatitis (NASH) trials and updates from Biogen Inc.’s aducanumab program in Alzheimer’s disease will take the spotlight, though the latter may be a 2020 event.
Finally, against the backdrop of a poor macro environment and down market, some investors are looking to names they view as being undervalued or unfairly punished on negative news.
“Historically, when valuations have been overblown versus fundamentals, we have stepped back our investment pace,” said Omega Funds’ Otello Stampacchia. “In this environment, we’re stepping back up our deployment pace.”
While BMS’s proposed takeout of Celgene, announced Jan. 3, takes an active acquirer of small to mid-cap biotechs out of the game, buysiders don’t expect the deal to impact their investment strategies. The proposal caps off Celgene’s 15-month decline driven by pipeline discontinuations and commercial headwinds that have made it hard to diversify revenues beyond Revlimid lenalidomide (see “Rocky Road”).
“M&A does not change my investment view for the year. I do not play for takeouts, although I am usually happy when I get them,” said ClearBridge Investments’ Marshall Gordon.
Table: 2019 milestones
Selected products with clinical or regulatory milestones expected in 2019. (A) Bristol-Myers Squibb Co. (NYSE:BMY) is acquiring Celgene Corp. (NASDAQ:CELG); Source: BCIQ: BioCentury Online Intelligence
|Acceleron Pharma Inc. (NASDAQ:XLRN) / Celgene Corp. (NASDAQ:CELG) (A)||Luspatercept||Myelodysplastic syndrome (MDS)-associated anemia and transfusion-dependent β-thalassemia||Submit BLA and MAA||1H19|
|Acer Therapeutics Inc. (NASDAQ:ACER)||ACER-001||Urea cycle disorder||Submit NDA||4Q19|
|Aimmune Therapeutics Inc. (NASDAQ:AIMT)||AR101||Peanut allergies in patients ages 4-17||Submit MAA||1H19|
|AIT Therapeutics Inc. (Pink:AITB)||AIT-PPHN||Persistent pulmonary hypertension in the newborn||Submit PMA||2Q19|
|Alder BioPharmaceuticals Inc. (NASDAQ:ALDR)||Eptinezumab||Prevent episodic and chronic migraine||Submit BLA||1Q19|
|Aldeyra Therapeutics Inc. (NASDAQ:ALDX)||Topical dermal reproxalap||Ichthyosis in patients with Sjogren-Larsson syndrome||Phase III data||2H19|
|Aldeyra Therapeutics Inc. (NASDAQ:ALDX) / Ligand Pharmaceuticals Inc. (NASDAQ:LGND)||Ocular reproxalap||Acute allergic conjunctivitis||Phase III data||Early 2019|
|Aldeyra Therapeutics Inc. (NASDAQ:ALDX) / Ligand Pharmaceuticals Inc. (NASDAQ:LGND)||Ocular reproxalap||Non-infectious anterior uveitis||Phase III data||2H19|
|Alexion Pharmaceuticals Inc. (NASDAQ:ALXN)||Soliris eculizumab||Neuromyelitis optica spectrum disorder||Submit regulatory applications in EU, Japan and U.S.||Early 2019|
|Alexion Pharmaceuticals Inc. (NASDAQ:ALXN)||Ultomiris ravulizumab||Atypical hemolytic uremic syndrome||Phase III data||Early 2019|
|Alkermes plc (NASDAQ:ALKS)||ALKS 3831 (olanzapine/samidorphan)||Schizophrenia||Submit NDA||Mid-2019|
|Allergan plc (NYSE:AGN)||Bimatoprost sr||Glaucoma||Phase III additional data; submit NDA||1H19; 2H19|
|Allergan plc (NYSE:AGN)||Rapastinel||Major depressive disorder (MDD)||Phase III data||2019|
|Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY)||Givosiran||Acute hepatic porphyrias||Phase III data; submit NDA and MAA||Early 2019; mid-2019|
|Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) / Sanofi (Euronext:SAN; NASDAQ:SNY)||Fitusiran||Hemophilia A and B||Phase III data||2019|
|Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) / Sanofi (Euronext:SAN; NASDAQ:SNY)||Lumasiran||Primary hyperoxaluria type 1||Phase III data||YE19|
|Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) / The Medicines Co. (NASDAQ:MDCO)||Inclisiran||Hypercholesterolemia||Phase III data; submit NDA||Mid-2019; YE19|
|Amryt Pharma plc (LSE:AMYT; ISE:AYP)||Episalvan (AP101)||Epidermolysis bullosa||Phase III data||2Q19|
|AnaptysBio Inc. (NASDAQ:ANAB)||Etokimab||Atopic dermatitis||Phase IIb data||2H19|
|Apellis Pharmaceuticals Inc. (NASDAQ:APLS)||APL-2||Paroxysmal nocturnal hemoglobinuria||Phase III data||2019|
|Ardelyx Inc. (NASDAQ:ARDX)||Tenapanor||Hyperphosphatemia in end-stage renal disease patients on hemodialysis||Phase III data||2019|
|Ascendis Pharma A/S (NASDAQ:ASND)||TransCon Growth Hormone (ACP-001)||Growth hormone deficiency||Phase III data||1Q19|
|AstraZeneca plc (LSE:AZN; NYSE:AZN)||PT010||Moderate to very severe chronic obstructive pulmonary disease||Phase III data; submit MAA and NDA||2H19|
|AstraZeneca plc (LSE:AZN; NYSE:AZN) / Celgene Corp. (NASDAQ:CELG) (A)||Imfinzi durvalumab||First-line non-small cell lung cancer (NSCLC)||Phase III data||1H19; 2H19|
|Atara Biotherapeutics Inc. (NASDAQ:ATRA)||Tabelecleucel||Epstein-Barr virus-associated post-transplant lymphoproliferative disease||Phase III data; submit conditional MAA||1H19; 2H19|
|Aurinia Pharmaceuticals Inc. (TSX:AUP; NASDAQ:AUPH) / 3SBio Inc. (HKSE:1530)||Voclosporin||Lupus nephritis||Phase III data||YE19|
|Bayer AG (Xetra:BAYN) / Syndax Pharmaceuticals Inc. (NASDAQ:SNDX)||Entinostat||Hormone receptor-positive, HER2-negative breast cancer||Phase III interim data||2Q19|
|BeiGene Ltd. (NASDAQ:BGNE; HKSE:6160)||Zanubrutinib (BGB-3111)||Waldenstrom macroglobulinemia||Submit NDA||YE19 - early 2020|
|Bellicum Pharmaceuticals Inc. (NASDAQ:BLCM)||Rivogenlecleucel||Prevent graft-vs-host disease (GvHD) and leukemia recurrence in pediatric patients receiving hematopoietic stem cell transplantat||Submit MAA||2019|
|BeyondSpring Inc. (NASDAQ:BYSI)||Plinabulin||Prevent chemotherapy-induced neutropenia||Phase III final data; submit Chinese NDA; submit U.S. NDA||1H19; early 2019; 2H19|
|BeyondSpring Inc. (NASDAQ:BYSI)||Plinabulin||NSCLC||Submit Chinese NDA||1H19|
|BioCryst Pharmaceuticals Inc. (NASDAQ:BCRX)||BCX7353||Prevent attacks of hereditary angioedema||Phase III data; submit NDA||2Q19; 4Q19|
|BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)||Valoctocogene roxaparvovec||Hemophilia A||Phase III data; submit BLA||2019; 2H19|
|BioMarin Pharmaceutical Inc. (NASDAQ:BMRN)||Vosoritide||Achondroplasia||Phase III data||YE19|
|BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) / Catalyst Pharmaceuticals Inc. (NASDAQ:CPRX)||Firdapse amifampridine||Congenital myasthenic syndromes||Phase III data||2H19|
|BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) / Catalyst Pharmaceuticals Inc. (NASDAQ:CPRX)||Firdapse amifampridine||Myasthenia gravis in patients with anti-muscle associated receptor tyrosine kinase (MUSK) antibodies||Phase III data||2H19|
|Blueprint Medicines Corp. (NASDAQ:BPMC)||Avapritinib||Gastrointestinal stromal tumors (GIST)||Submit NDA||1H19|
|Bristol-Myers Squibb Co. (NYSE:BMY) / AstraZeneca plc (LSE:AZN; NYSE:AZN)||Anifrolumab||Systemic lupus erythematosus||Submit MAA and NDA; submit Japanese NDA||1H19; 2H19|