→ Neu­rotrope be­came the lat­est ca­su­al­ty in the high-risk Alzheimer’s R&D field. The biotech re­port­ed that their Phase II study of Bryo­statin-1 failed to show any im­prove­ment over place­bo us­ing the Se­vere Im­pair­ment Bat­tery (SIB) to­tal score at week 13. In fact, the drug arm did a lit­tle worse than a sug­ar pill, with an av­er­age in­crease of 1.3 points for the drug and 2.1 points for place­bo. Their shares $NTRP cratered in the rout that fol­lowed, plung­ing 78%. It’s cur­rent­ly a pen­ny stock.

The Alzheimer’s R&D field hasn’t record­ed a suc­cess in chang­ing the tra­jec­to­ry of the dis­ease, with re­cent piv­otal fail­ures forc­ing all the play­ers to re­con­sid­er their strate­gies.

→ Platelet Bio­Gen­e­sis (PBG) — a com­pa­ny fo­cused on stem cell-de­rived, on-de­mand hu­man platelets and platelet-based ther­a­peu­tics — has raised more than $26 mil­lion in Se­ries A-1 fi­nanc­ing. In com­bi­na­tion to this round, as well as Se­ries A fi­nanc­ing and grants, the com­pa­ny’s to­tal fund­ing is brought to about $45 mil­lion. The fi­nanc­ing was co-led by Ziff Cap­i­tal Part­ners and Qim­ing Ven­ture Part­ners USA in ad­di­tion to ex­ist­ing in­ves­tures Nest.Bio Ven­tures and eCoast An­gels.

The com­pa­ny said that it “will use the funds from the Se­ries A-1 round to ac­cel­er­ate the de­vel­op­ment of its donor-in­de­pen­dent platelet man­u­fac­tur­ing ca­pa­bil­i­ties and ad­vance its proof of con­cept work around platelet-based ther­a­peu­tics.” PBG plans to dou­ble its staff to ap­prox­i­mate­ly 50 em­ploy­ees in 2020.

→ Stu­art Levy, co-founder of Paratek Phar­ma­ceu­ti­cals, passed away last week.

“Stu­art was a dis­tin­guished physi­cian who was the fore­front of an­tibi­ot­ic de­vel­op­ment and a ded­i­cat­ed cham­pi­on for the pru­dent use of an­tibi­otics,” said Evan Loh, Paratek CEO. “More im­por­tant­ly, he was a dear friend and men­tor to so many and his pas­sion lives on in each of us as we work to con­tin­ue his mis­sion of com­bat­ing the dai­ly threat of life-threat­en­ing in­fec­tions. We will great­ly miss him.”

Levy co-found­ed Paratek more than 20 years ago along with Wal­ter Gilbert. He was in­stru­men­tal in the de­vel­op­ment of the com­pa­ny’s lead an­tibi­otics, Nuzyra and Seysara. Levy dis­cov­ered the ef­flux mech­a­nism for drug (tetra­cy­cline) re­sis­tance and was among the first sci­en­tists to doc­u­ment the trans­fer of re­sis­tant bac­te­ria from an­i­mals to farm­work­ers. His 1992 book, The An­tibi­ot­ic Para­dox: How Mir­a­cle Drugs Are De­stroy­ing the Mir­a­cle, has been cit­ed wide­ly and trans­lat­ed in­to four lan­guages.

The news comes a few weeks af­ter the com­pa­ny pub­lished pos­i­tive Phase III da­ta for Nuzyra for pa­tients with AB­SS­SI.

→ In the midst of a sur­prise in­ter partes re­view by the US Patent Tri­als Ap­peal Board (PT­AB) for Alex­ion’s Soliris, the com­pa­ny and Bridge­Bio’s sub­sidiary Ei­dos Ther­a­peu­tics have inked an agree­ment grant­i­ng Alex­ion an ex­clu­sive li­cense to de­vel­op and com­mer­cial­ize their oral­ly-ad­min­is­tered small mol­e­cule AG10 — de­signed to treat transthyretin amy­loi­do­sis (AT­TR) by bind­ing and sta­bi­liz­ing transthyretin (TTR) in the blood— in Japan.

“Ei­dos is cur­rent­ly eval­u­at­ing AG10 in a Phase III study in the US and Eu­rope for AT­TR car­diomy­opa­thy (AT­TR-CM) – a pro­gres­sive, fa­tal dis­ease caused by the ac­cu­mu­la­tion of mis­fold­ed TTR amy­loid in the heart – and plans to be­gin a Phase III study in AT­TR polyneu­ropa­thy (AT­TR-PN) – a pro­gres­sive, fa­tal dis­ease caused by the ac­cu­mu­la­tion of mis­fold­ed TTR amy­loid in the pe­riph­er­al ner­vous sys­tem.”

→ A few months af­ter Ac­celeron and its part­ner at Cel­gene se­cured pri­or­i­ty re­view for their red blood cell boost­ing drug, lus­pa­ter­cept — to man­age ane­mia re­sult­ing from myelodys­plas­tic syn­drome (MDS) as well as be­ta tha­lassemia — the com­pa­ny has an­nounced an­oth­er win. This time they’ve gained an or­phan drug des­ig­na­tion by the FDA for its in­ves­ti­ga­tion­al ther­a­py so­tater­cept for the treat­ment of pa­tients with pul­monary ar­te­r­i­al hy­per­ten­sion (PAH).

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

→ Less than a year ago Gilead’s antiviral remdesivir failed to make the cut as investigators considered a raft of potential drugs that could be used against an Ebola outbreak. But it may gain a new mission with the outbreak of the coronavirus in China, which is popping up now around the world.

Gilead put out a statement saying that they’re now in discussions with health officials in the US and China about testing their NUC against the virus. It’s the latest in a growing lineup of biopharma companies that are marshaling R&D forces to see if they can come up with a vaccine or therapy to blunt the spread of the virus, which has now sickened hundreds, killed at least 17 people and led the Chinese government to start quarantining cities.

Back at the beginning of 2015, Deerfield Management co-led a $10 million Series C for a private gene therapy startup, reshaping the company and bringing in new leaders to pave way for an IPO just a year later.

Fast forward four more years and the startup, AveXis, is now a subsidiary of Novartis marketing the second-ever gene therapy to be approved in the US.

For its part, Deerfield has also grown more comfortable and ambitious about the nascent field. And the investment firm is now putting down its biggest bet yet: a $1.1 billion contract development and manufacturing facility to produce everything one needs for cell and gene therapy — faster and better than how it’s currently done.

Different therapeutic traits brandished by the three approved therapies for sickle cell disease all extend life expectancy, but their impact on quality of life is uncertain and their long-term cost-effectiveness is not up to scratch according to the thresholds considered reasonable by ICER, the non-profit concluded in a draft guidance report on Thursday.

Sickle cell disease (SCD), which encompasses a group of inherited red blood cell disorders that typically afflict those of African ancestry, impacts hemoglobin — and is characterized by episodes of searing pain as well as organ damage.